## Protocol Section
### Identification Module
**NCT ID:** NCT00483379

**Brief Title:** High Dose or High Dose Frequency Study of Alglucosidase Alfa

**Official Title:** An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

#### Organization Study ID Info
**ID:** AGLU03306
#### Organization
**Class:** INDUSTRY
**Full Name:** Sanofi
### Status Module
#### Completion Date
**Date:** 2010-07

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-03-07

**Type:** ESTIMATED

**Last Update Submit Date:** 2014-02-04

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2009-12

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2011-04-22

**Type:** ESTIMATED

**Results First Submit Date:** 2011-02-07

**Results First Submit QC Date:** 2011-03-23

#### Start Date
**Date:** 2007-05

**Status Verified Date:** 2014-02

#### Study First Post Date
**Date:** 2007-06-07
**Type:** ESTIMATED
**Study First Submit Date:** 2007-06-06
**Study First Submit QC Date:** 2007-06-06
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Genzyme, a Sanofi Company

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.

### Conditions Module

**Conditions:**

- Pompe Disease

- Glycogen Storage Disease Type II (GSD-II)

- Glycogenesis 2 Acid Maltase Deficiency

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 13

**Type:** ACTUAL

**Phases:**

- PHASE4

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.

**Intervention Names:**

- Biological: alglucosidase alfa

**Label:** alglucosidase alfa 20 mg/kg every week

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.

**Intervention Names:**

- Biological: alglucosidase alfa

**Label:** alglucosidase alfa 40 mg/kg every other week

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- alglucosidase alfa 20 mg/kg every week

- alglucosidase alfa 40 mg/kg every other week

**Description:** intravenous infusion

**Name:** alglucosidase alfa

**Other Names:**

- Recombinant human acid glucosidase

- Myozyme

**Type:** BIOLOGICAL

### Outcomes Module

#### Primary Outcomes

**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each participant served as his or her own control.

**Measure:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment

**Time Frame:** Baseline, Week 52

**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Participants could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each participant served as his or her own control.

**Measure:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment

**Time Frame:** Baseline, Week 52

**Description:** Overall safety summary of participants experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment.

**Measure:** Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period

**Time Frame:** Day 1 up to Week 52

#### Secondary Outcomes

**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.

**Measure:** Baseline Values for Left Ventricular Mass (LVM) Z-Scores

**Time Frame:** Day 0

**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.

**Measure:** Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52

**Time Frame:** Baseline, Week 52

**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.

**Measure:** Baseline Values for Left Ventricular Mass Index (LVMI)

**Time Frame:** Day 0

**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.

**Measure:** Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52

**Time Frame:** Baseline, Week 52

**Description:** The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support.

**Measure:** Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)

**Time Frame:** Baseline, approximately Week 52

**Description:** Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength.

**Measure:** Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52

**Time Frame:** Baseline, Week 52

**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.

**Measure:** Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results

**Time Frame:** Day 0

**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.

**Measure:** Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52

**Time Frame:** Baseline, Week 52

**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.

**Measure:** Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)

**Time Frame:** Day 0

**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.

**Measure:** Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52

**Time Frame:** Baseline, Week 52

**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.

**Measure:** Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)

**Time Frame:** Day 0

**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.

**Measure:** Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52

**Time Frame:** Baseline, Week 52

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures;
* The patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency in skin fibroblasts or blood;
* The patient must have been compliant with the standard dosing regimen of alglucosidase alfa (20 mg/kg every other week) for a minimum of 6 months immediately prior to study entry
* The patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to the beginning alglucosidase alfa treatment:

  1. Cardiac: Left Ventricular Mass (LVM) Z-score ≥6 or LVM index ≥150 g/m2 after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
  2. Respiratory: New development of respiratory failure requiring the use of ventilatory assistance (invasive or non-invasive) after a minimum of 6 months of regular treatment with alglucosidase alfa. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment; OR
  3. Motor Skills:
* For patients ≤ 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
* For patients \> 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through loss of functional use of the upper extremities after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients \> 8 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through longitudinal assessments of manual muscle testing after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients previously ambulatory, progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of regular treatment with alglucosidase alfa.

Exclusion Criteria:

* For patients \< 18 years of age, negative Cross-Reactive Immunologic Material (CRIM) assay result (added in protocol amendment #2);
* Any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of alglucosidase alfa;
* The patient is not currently receiving alglucosidase alfa;
* The patient has major congenital abnormality;
* The patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment;
* The patient is pregnant or lactating.

**Minimum Age:** 6 Months

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Birmingham

**Country:** United States

**State:** Alabama

**Location 2:**

**City:** Stanford

**Country:** United States

**State:** California

**Location 3:**

**City:** Washington D.C.

**Country:** United States

**State:** District of Columbia

**Location 4:**

**City:** Chicago

**Country:** United States

**State:** Illinois

**Location 5:**

**City:** Kansas City

**Country:** United States

**State:** Kansas

**Location 6:**

**City:** Boston

**Country:** United States

**State:** Massachusetts

**Location 7:**

**City:** Grand Rapids

**Country:** United States

**State:** Michigan

**Location 8:**

**City:** Glenn Falls

**Country:** United States

**State:** New York

**Location 9:**

**City:** Durham

**Country:** United States

**State:** North Carolina

**Location 10:**

**City:** Parkville Victoria

**Country:** Australia

**Location 11:**

**City:** Calgary

**Country:** Canada

**State:** Alberta

#### Overall Officials

**Official 1:**

**Affiliation:** Genzyme, a Sanofi Company

**Name:** Medical Monitor

**Role:** STUDY_DIRECTOR

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000020140

- Term: Lysosomal Storage Diseases, Nervous System

- ID: D000020739

- Term: Brain Diseases, Metabolic, Inborn

- ID: D000001928

- Term: Brain Diseases, Metabolic

- ID: D000001927

- Term: Brain Diseases

- ID: D000002493

- Term: Central Nervous System Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000008661

- Term: Metabolism, Inborn Errors

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000002239

- Term: Carbohydrate Metabolism, Inborn Errors

- ID: D000016464

- Term: Lysosomal Storage Diseases

- ID: D000008659

- Term: Metabolic Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M9114

- Name: Glycogen Storage Disease

- Relevance: HIGH

- As Found: Glycogen Storage Disease

- ID: M9115

- Name: Glycogen Storage Disease Type II

- Relevance: HIGH

- As Found: Glycogen Storage Disease Type II

- ID: M18871

- Name: Lysosomal Storage Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5204

- Name: Brain Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5205

- Name: Brain Diseases, Metabolic

- Relevance: LOW

- As Found: Unknown

- ID: M22498

- Name: Brain Diseases, Metabolic, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M5742

- Name: Central Nervous System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M11641

- Name: Metabolism, Inborn Errors

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M5498

- Name: Carbohydrate Metabolism, Inborn Errors

- Relevance: LOW

- As Found: Unknown

- ID: M11639

- Name: Metabolic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T2562

- Name: Glycogen Storage Disease Type 2

- Relevance: HIGH

- As Found: Pompe Disease

### Condition Browse Module - Meshes

- ID: D000006009

- Term: Glycogen Storage Disease Type II

- ID: D000006008

- Term: Glycogen Storage Disease

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

**Description:** In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.

Events are listed independent of relationship to treatment reported.

#### Event Groups

**Group ID:** EG000

**Title:** Treatment: Alglucosidase Alfa 20 mg/kg Every Week

**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.

**ID:** EG000

**Other Num Affected:** 6

**Other Num at Risk:** 6

**Serious Number Affected:** 2

**Serious Number At Risk:** 6

**Title:** Treatment: Alglucosidase Alfa 20 mg/kg Every Week

**Group ID:** EG001

**Title:** Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week

**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.

**ID:** EG001

**Other Num Affected:** 7

**Other Num at Risk:** 7

**Serious Number Affected:** 1

**Serious Number At Risk:** 7

**Title:** Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week

**Group ID:** EG002

**Title:** Extension: Alglucosidase Alfa 20 mg/kg Every Week

**Description:** The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available.

**ID:** EG002

**Other Num Affected:** 1

**Other Num at Risk:** 1

**Serious Number Affected:** 1

**Serious Number At Risk:** 1

**Title:** Extension: Alglucosidase Alfa 20 mg/kg Every Week

**Group ID:** EG003

**Title:** Extension: Alglucosidase Alfa 40 mg/kg Every Other Week

**Description:** The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available.

**ID:** EG003

**Other Num Affected:** 1

**Other Num at Risk:** 2

**Serious Number At Risk:** 2

**Title:** Extension: Alglucosidase Alfa 40 mg/kg Every Other Week

**Frequency Threshold:** 0

#### Other Events

**Term:** Lymphadenitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Lymphadenopathy

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Right ventricular hypertrophy

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Cardiac disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Tachycardia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Cardiac disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypoacusis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Ear and labyrinth disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Dry eye

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Eye disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Eyelid ptosis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Eye disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Abdominal discomfort

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Abdominal pain upper

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Amalgam tattoo

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Anal fissure

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Aphthous stomatitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Constipation

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Diarrhoea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Haematochezia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Nausea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Vomiting

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Adverse drug reaction

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Asthenia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Fatigue

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Generalised oedema

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Pyrexia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Abscess limb

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Acute sinusitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Candidiasis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Cellulitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Device related infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Ear infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Fungal skin infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Gastroenteritis viral

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Gastrointestinal viral infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Hordeolum

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Influenza

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Nasopharyngitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Otitis media

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Otitis media acute

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Pneumococcal infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Pneumonia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Rash pustular

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Respiratory tract infection bacterial

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Respiratory tract infection viral

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Sinusitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Streptococcal infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Subcutaneous abscess

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Tonsillitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Tooth abscess

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Upper respiratory tract infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Urinary tract infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Varicella

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Viral infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

**Term:** Arthropod bite

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Contusion

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Epicondylitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Excoriation

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Fall

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Muscle strain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Vaccination complication

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

**Term:** Aspiration tracheal abnormal

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Blood calcium increased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Breath sounds abnormal

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Heart rate irregular

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Prostatic specific antigen increased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Protein urine present

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** White blood cells urine positive

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

**Term:** Dehydration

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Gout

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypocalcaemia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypoglycaemia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypokalaemia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypomagnesaemia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Back pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Muscle spasms

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Myalgia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Pain in extremity

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Musculoskeletal and connective tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Dizziness

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Headache

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypotonia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Presyncope

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Restless legs syndrome

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Somnolence

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Tremor

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Pollakiuria

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Renal and urinary disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Asthma

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Atelectasis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Cough

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Dyspnoea

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Increased bronchial secretion

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Oropharyngeal pain

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Vasomotor rhinitis

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Dermatitis diaper

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Eczema

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Erythema

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Rash

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Rash erythematous

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Rash macular

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Urticaria

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Blood pressure fluctuation

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Vascular disorders

**Source Vocabulary:** MedDRA 13.0

**Term:** Hypotension

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Vascular disorders

**Source Vocabulary:** MedDRA 13.0

#### Serious Events

**Term:** Supraventricular tachycardia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Cardiac disorders

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num At Risk:** 6

**Group ID:** EG001

**Num Affected:** 1

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Dysphagia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num At Risk:** 6

**Group ID:** EG001

**Num Affected:** 1

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Device related infection

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num At Risk:** 6

**Group ID:** EG001

**Num Affected:** 1

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Pneumonia

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num Affected:** 2

**Num At Risk:** 6

**Group ID:** EG001

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Fibula fracture

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Injury, poisoning and procedural complications

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num At Risk:** 6

**Group ID:** EG001

**Num At Risk:** 7

**Group ID:** EG002

**Num Affected:** 1

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Weight decreased

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num At Risk:** 6

**Group ID:** EG001

**Num Affected:** 1

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Term:** Respiratory failure

**Assessment Type:** SYSTEMATIC_ASSESSMENT

**Organ System:** Respiratory, thoracic and mediastinal disorders

**Source Vocabulary:** MedDRA 13.0

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 6

**Group ID:** EG001

**Num At Risk:** 7

**Group ID:** EG002

**Num At Risk:** 1

**Group ID:** EG003

**Num At Risk:** 2

**Time Frame:** Treatment period AEs were collected up to week 52. Extension period AEs were collected following completion of the treatment period until the product was commercially available (up to week 118).

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 6

**Group ID:** BG001

**Value:** 7

**Group ID:** BG002

**Value:** 13

**Units:** Participants

### Group

**ID:** BG000

**Title:** Alglucosidase Alfa 20 mg/kg Every Week

**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.

### Group

**ID:** BG001

**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week

**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.

### Group

**ID:** BG002

**Title:** Total

**Description:** Total of all reporting groups

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 27.75

**Value:** 23.3

#### Measurement

**Group ID:** BG001

**Spread:** 15.56

**Value:** 16.8

#### Measurement

**Group ID:** BG002

**Spread:** 21.29

**Value:** 19.8

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 4

#### Measurement

**Group ID:** BG001

**Value:** 5

#### Measurement

**Group ID:** BG002

**Value:** 9

**Class Title:** <18 years

#### Measurement

**Group ID:** BG000

**Value:** 2

#### Measurement

**Group ID:** BG001

**Value:** 2

#### Measurement

**Group ID:** BG002

**Value:** 4

**Class Title:** >= 18 and <=65 years

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Class Title:** >65 years

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 2

#### Measurement

**Group ID:** BG001

**Value:** 3

#### Measurement

**Group ID:** BG002

**Value:** 5

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 4

#### Measurement

**Group ID:** BG001

**Value:** 4

#### Measurement

**Group ID:** BG002

**Value:** 8

**Category Title:** Male

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** American Indian or Alaska Native

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 1

#### Measurement

**Group ID:** BG002

**Value:** 1

**Category Title:** Asian

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** Native Hawaiian or Other Pacific Islander

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** Black or African American

#### Measurement

**Group ID:** BG000

**Value:** 6

#### Measurement

**Group ID:** BG001

**Value:** 6

#### Measurement

**Group ID:** BG002

**Value:** 12

**Category Title:** White

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** More than one race

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** Unknown or Not Reported

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 4

#### Measurement

**Group ID:** BG001

**Value:** 5

#### Measurement

**Group ID:** BG002

**Value:** 9

**Class Title:** Infantile-onset Pompe Disease

#### Measurement

**Group ID:** BG000

**Value:** 2

#### Measurement

**Group ID:** BG001

**Value:** 2

#### Measurement

**Group ID:** BG002

**Value:** 4

**Class Title:** Late-onset Pompe Disease

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Class Title:** Cardiac

#### Measurement

**Group ID:** BG000

**Value:** 1

#### Measurement

**Group ID:** BG001

**Value:** 1

#### Measurement

**Group ID:** BG002

**Value:** 2

**Class Title:** Respiratory

#### Measurement

**Group ID:** BG000

**Value:** 5

#### Measurement

**Group ID:** BG001

**Value:** 6

#### Measurement

**Group ID:** BG002

**Value:** 11

**Class Title:** Motor Skills

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 3

#### Measurement

**Group ID:** BG002

**Value:** 3

**Class Title:** Positive

#### Measurement

**Group ID:** BG000

**Value:** 1

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 1

**Class Title:** Negative

#### Measurement

**Group ID:** BG000

**Value:** 5

#### Measurement

**Group ID:** BG001

**Value:** 4

#### Measurement

**Group ID:** BG002

**Value:** 9

**Class Title:** Unknown

**Denomination Units Selected:** 

## Results Section - Baseline Characteristics Module

### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** NUMBER
**Title:** Age, Customized
**Unit of Measure:** participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 5
**Parameter Type:** NUMBER
**Title:** Life-stage of Disease Onset
**Unit of Measure:** participants
### Measure 6
**Description:** Participant counts of the parameter in clinical decline (cardiac, respiratory or motor skills as compared to their condition prior to the beginning alglucosidase alfa treatment) for which participants were included in the study.
**Parameter Type:** NUMBER
**Title:** Parameter in Clinical Decline
**Unit of Measure:** participants
### Measure 7
**Parameter Type:** NUMBER
**Title:** Cross-Reactive Immunologic Material (CRIM) Assay Result
**Unit of Measure:** participants
## Results Section - More Information Module
### Certain Agreement
**Other Details:** In multi-site studies, PI will delay submission of a publication until the earlier of; (i) publication of the multi-center data by Genzyme (ii)12-24 months after study completion or termination of the Study at all sites, or (iii) Genzyme notification that a publication will not occur. PI will send Genzyme a draft 60 days before publication. Genzyme can defer publication 90-120 days upon notifying PI that it will file a patent application on inventions contained in the draft.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Limitations and Caveats
**Description:** This small exploratory study lacked a parallel control arm at the standard dose for a longer period; decline in respiratory or motor function prior to study was not collected systematically, thus change from baseline observations are inconclusive.
### Point of Contact
**Organization:** Genzyme Corporation
**Phone:** 800-745-4447
**Title:** Genzyme Medical Information
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
### Outcome Measure 6
### Outcome Measure 7
### Outcome Measure 8
### Outcome Measure 9
### Outcome Measure 10
### Outcome Measure 11
### Outcome Measure 12
### Outcome Measure 13
### Outcome Measure 14
### Outcome Measure 15
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -1.2
- **Spread:** 
- **Upper Limit:** 6.3
- **Value:** 0.3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** -1.2
- **Spread:** 
- **Upper Limit:** 2.2
- **Value:** -0.3
**Title:** 
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -0.8
- **Spread:** 1.04
- **Upper Limit:** 1.2
- **Value:** 0.3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** -0.1
- **Spread:** 0.33
- **Upper Limit:** 0.5
- **Value:** 0.4
**Title:** 
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 49.0
- **Spread:** 
- **Upper Limit:** 187.3
- **Value:** 62.1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 45.4
- **Spread:** 
- **Upper Limit:** 73.1
- **Value:** 56.5
**Title:** 
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -10.9
- **Spread:** 14.44
- **Upper Limit:** 15.5
- **Value:** 12.5
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** -5.3
- **Spread:** 5.95
- **Upper Limit:** 5.8
- **Value:** 4.0
**Title:** 
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 3
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 4
**Title:** 
#### Outcome Measure 7
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 60.52
- **Upper Limit:** 
- **Value:** 65.0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 84.00
- **Upper Limit:** 
- **Value:** 82.8
**Title:** 
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 8.49
- **Upper Limit:** 
- **Value:** 6.0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 6.12
- **Upper Limit:** 
- **Value:** 6.7
**Title:** 
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 20.94
- **Upper Limit:** 
- **Value:** 38.3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 21.26
- **Upper Limit:** 
- **Value:** 46.8
**Title:** 
#### Outcome Measure 11
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 4.28
- **Upper Limit:** 
- **Value:** 0.6
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 3.84
- **Upper Limit:** 
- **Value:** 3.5
**Title:** 
#### Outcome Measure 12
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 2.84
- **Upper Limit:** 
- **Value:** 31.3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 9.33
- **Upper Limit:** 
- **Value:** 36.0
**Title:** 
#### Outcome Measure 13
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2.5
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 11.24
- **Upper Limit:** 
- **Value:** 4.4
**Title:** 
#### Outcome Measure 14
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 4
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
#### Outcome Measure 15
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 7
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 7
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 6
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 3
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 0
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 2
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 1
**Title:** 
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each participant served as his or her own control.
**Parameter Type:** NUMBER
**Population Description:** All participants who enrolled due to decline in respiratory function while on standard treatment.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 2
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVM data
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Left Ventricular Mass (LVM) Z-Scores
**Type:** SECONDARY
**Unit of Measure:** Z-score
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 3
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVM data at both timepoints
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
**Type:** SECONDARY
**Unit of Measure:** Z-score
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 4
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVMI data
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Left Ventricular Mass Index (LVMI)
**Type:** SECONDARY
**Unit of Measure:** g/m^2
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 5
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVMI data at both timepoints
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
**Type:** SECONDARY
**Unit of Measure:** g/m^2
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 6
**Description:** The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support.
**Parameter Type:** NUMBER
**Population Description:** Full analysis population. The participant in the worsened category died after week 52.
**Reporting Status:** POSTED
**Time Frame:** Baseline, approximately Week 52
**Title:** Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
**Type:** SECONDARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 7
**Description:** Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength.
**Population Description:** Full analysis population of participants \>= 8 years old. Due to the age restriction and small study population, the number of participants analyzed is too small for results to be meaningful.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
**Type:** SECONDARY
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 8
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 9
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 10
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 11
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 12
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population of participants \>= 14 years old.
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 13
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population of participants \>= 14 years old.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 14
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Participants could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each participant served as his or her own control.
**Parameter Type:** NUMBER
**Population Description:** All participants who enrolled due to decline in motor function while on standard treatment.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 15
**Description:** Overall safety summary of participants experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment.
**Parameter Type:** NUMBER
**Population Description:** Safety population comprised of all participants who received intervention.
**Reporting Status:** POSTED
**Time Frame:** Day 1 up to Week 52
**Title:** Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
### Participant Flow Module
#### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** FG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
#### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** FG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Period
**Title:** Treatment Period
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 6
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 4
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
#### Period
**Title:** Extension Period
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
**Pre-Assignment Details:** Fourteen participants were screened and enrolled; however, one withdrew before receiving any study infusions due to the burden of weekly trips to the medical center.
**Has Results:** True