## Protocol Section
### Identification Module
**NCT ID:** NCT01650779

**Brief Title:** A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

**Official Title:** Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study)

#### Organization Study ID Info
**ID:** AGAL19412
#### Organization
**Class:** INDUSTRY
**Full Name:** Sanofi
### Status Module
#### Completion Date
**Date:** 2013-03

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-07-10

**Type:** ESTIMATED

**Last Update Submit Date:** 2014-06-09

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2013-03

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2014-07-10

**Type:** ESTIMATED

**Results First Submit Date:** 2014-06-09

**Results First Submit QC Date:** 2014-06-09

#### Start Date
**Date:** 2012-04

**Status Verified Date:** 2014-06

#### Study First Post Date
**Date:** 2012-07-26
**Type:** ESTIMATED
**Study First Submit Date:** 2012-07-24
**Study First Submit QC Date:** 2012-07-25
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Genzyme, a Sanofi Company

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2 milligram per kilogram (mg/kg) every two weeks (q2w) and who are being switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w.

### Conditions Module

**Conditions:**

- Fabry Disease

**Keywords:**

- alpha-galactosidase A

- a-GAL

- Fabry

- GL-3

- Fabrazyme

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 15

**Type:** ACTUAL

**Phases:**

- PHASE4

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Intervention Names:**

- Biological: Agalsidase beta

**Label:** Agalsidase beta

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Agalsidase beta

**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.

**Name:** Agalsidase beta

**Other Names:**

- Fabrazyme®

**Type:** BIOLOGICAL

### Outcomes Module

#### Primary Outcomes

**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as below quantitative limit (BQL), the lower limit of quantitation (LLOQ) value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma lyso-GL-3 was 5.0 nanogram per milliliter (ng/mL). This study is exploratory because little is known about the dose-response of these biomarkers to enzyme replacement therapy (ERT) or about the clinical significance of the biomarkers.

**Measure:** Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6

**Time Frame:** Baseline, Month 2, 4, 6

#### Secondary Outcomes

**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma GL-3 was 2.0 microgram per milliliter (mcg/mL). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.

**Measure:** Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6

**Time Frame:** Baseline, Month 2, 4, 6

**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for urine GL-3 was 0.2 mcg/mL. The absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing GL-3 (mcg/mL) by creatinine (mg/mL) and multiplying by 113.13 (mg/mmol), the molecular weight of creatinine. For levels reported BQL, the absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing 0.1 (mcg/mL) by creatinine (mg/mL) and multiplying by 133.13 (mg/mmol). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.

**Measure:** Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6

**Time Frame:** Baseline, Month 2, 4, 6

**Description:** Gastrointestinal symptoms (abdominal pain, abdominal distention, and irregular bowel movements) were to be assessed by a modified version of the Irritable Bowel Syndrome (IBS) Severity Scoring System. The modified IBS Severity Scoring System is a 7-item questionnaire. The severity score calculated by summing the scores of 5 of the 7 questions. Each of the 5 questions were scored on a scale of 0 to 100, leading to a total possible score range of 0 to 500, where higher scores indicate more severe gastrointestinal symptoms. The data for this outcome measure was exploratory and to be collected in individual participant listing only.

**Measure:** Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6

**Time Frame:** Baseline, Month 2, 4, 6

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* The participant and/or his parent/legal guardian is willing and able to provide signed informed consent, and the participant, if less than (\<) 18 years of age, is willing to provide assent if deemed able to do so
* Participant is male and has been treated with agalsidase alfa at 0.2 mg/kg q2w for the 12 months prior to switching to agalsidase beta
* The participant has a confirmed diagnosis of Fabry disease by alfa-galactosidase A (alfa-GAL) activity and/or genotyping per local standards
* The participant when switched to agalsidase beta receives the labeled dose, that is, 0.9 to 1.1 mg/kg (1 mg/kg) q2w, and must be willing to maintain the labeled dose for the duration of the study

Exclusion Criteria:

* The participant is on dialysis or is post renal transplantation
* The participant is in end-stage cardiac failure
* The participant and/or his parent or legal guardian, in the opinion of the investigator, is unable to adhere to the requirements of the study
* The participant has been switched from agalsidase alfa to agalsidase beta and does not have historical blood and urine samples

**Sex:** MALE

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Coral Springs

**Country:** United States

**State:** Florida

**Location 2:**

**City:** Decatur

**Country:** United States

**State:** Georgia

**Location 3:**

**City:** Baltimore

**Country:** United States

**State:** Maryland

**Location 4:**

**City:** Grand Rapids

**Country:** United States

**State:** Michigan

**Location 5:**

**City:** Hellertown

**Country:** United States

**State:** Pennsylvania

**Location 6:**

**City:** Fairfax

**Country:** United States

**State:** Virginia

#### Overall Officials

**Official 1:**

**Affiliation:** Genzyme, a Sanofi Company

**Name:** Medical Monitor

**Role:** STUDY_DIRECTOR

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000013106

- Term: Sphingolipidoses

- 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: D000059345

- Term: Cerebral Small Vessel Diseases

- ID: D000002561

- Term: Cerebrovascular Disorders

- ID: D000014652

- Term: Vascular Diseases

- ID: D000002318

- Term: Cardiovascular Diseases

- ID: D000040181

- Term: Genetic Diseases, X-Linked

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000008661

- Term: Metabolism, Inborn Errors

- ID: D000008064

- Term: Lipidoses

- ID: D000008052

- Term: Lipid Metabolism, Inborn Errors

- ID: D000016464

- Term: Lysosomal Storage Diseases

- ID: D000008659

- Term: Metabolic Diseases

- ID: D000052439

- Term: Lipid Metabolism Disorders

### Condition Browse Module - Browse Branches

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC14

- Name: Heart and Blood Diseases

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4124

- Name: Fabry Disease

- Relevance: HIGH

- As Found: Fabry Disease

- ID: M15904

- Name: Sphingolipidoses

- Relevance: LOW

- As Found: Unknown

- 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: M29437

- Name: Cerebral Small Vessel Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5810

- Name: Cerebrovascular Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M17400

- Name: Vascular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M24877

- Name: Genetic Diseases, X-Linked

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M11641

- Name: Metabolism, Inborn Errors

- Relevance: LOW

- As Found: Unknown

- ID: M11064

- Name: Lipidoses

- Relevance: LOW

- As Found: Unknown

- ID: M11054

- Name: Lipid Metabolism, Inborn Errors

- Relevance: LOW

- As Found: Unknown

- ID: M11639

- Name: Metabolic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M27029

- Name: Lipid Metabolism Disorders

- Relevance: LOW

- As Found: Unknown

- ID: T2169

- Name: Fabry Disease

- Relevance: HIGH

- As Found: Fabry Disease

- ID: T5335

- Name: Sphingolipidosis

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000000795

- Term: Fabry Disease

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

**Description:** All enrolled participants were included in the analysis.

#### Event Groups

**Group ID:** EG000

**Title:** Agalsidase Beta

**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion every 2 weeks up to Month 6.

**ID:** EG000

**Other Num Affected:** 1

**Other Num at Risk:** 15

**Serious Number At Risk:** 15

**Title:** Agalsidase Beta

**Frequency Threshold:** 0

#### Other Events

**Term:** Infusion-associated reactions

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA 16.0

**Time Frame:** Baseline up to end of study (Month 6) or early withdrawal

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 15

**Units:** Participants

### Group

**ID:** BG000

**Title:** Agalsidase Beta

**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 16.11

**Value:** 28.5

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 15

**Category Title:** Male

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 13

**Class Title:** Caucasian

#### Measurement

**Group ID:** BG000

**Value:** 1

**Class Title:** Hispanic

#### Measurement

**Group ID:** BG000

**Value:** 1

**Class Title:** Other

### Measure

#### Measurement

**Group ID:** BG000

**Lower Limit:** 2

**Spread:** 4.73

**Upper Limit:** 19

**Value:** 9.3

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 14

**Class Title:** Genotype

#### Measurement

**Group ID:** BG000

**Value:** 11

**Class Title:** Leukocyte alfa-galactosidase A (GAL) activity

#### Measurement

**Group ID:** BG000

**Value:** 9

**Class Title:** Plasma alfa-GAL activity

**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:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** NUMBER
**Title:** Race/Ethnicity, Customized
**Unit of Measure:** participants
### Measure 4
**Description:** Duration of Fabry disease was calculated from date of initial diagnosis of Fabry disease to date of the first study infusion. Number of participants analyzed for this baseline measure were 14, as one participant did not have information on date of initial diagnosis of Fabry disease.
**Dispersion Type:** FULL_RANGE
**Parameter Type:** MEDIAN
**Title:** Duration of Fabry Disease
**Unit of Measure:** years
### Measure 5
**Description:** Number of participants with each method (genotype, leukocyte alfa-galactosidase A \[GAL\] activity, and plasma alfa-GAL activity) are reported. Some participants were diagnosed by more than 1 method, hence reported under more than 1 category.
**Parameter Type:** NUMBER
**Title:** Method of Diagnosis of Fabry Disease
**Unit of Measure:** participants
**Population Description:** All enrolled participants were included in the analysis.
## Results Section - More Information Module
### Certain Agreement
**Other Details:** If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Limitations and Caveats
**Description:** This is considered to be an exploratory study for the following reasons: it was based on a small number of participants and has been designed as an open-label, single-arm study as opposed to a two-arm crossover design.
### Point of Contact
**Email:** Contact-us@sanofi.com
**Organization:** Sanofi
**Title:** Trial Transparency Team
## Results Section - Outcome Measures Module
### Outcome Measure 1
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 2: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.0002
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 4: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** <0.0001
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 6: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** <0.0001
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
### Outcome Measure 2
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 2: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.1178
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 4: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.1176
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 6: Analysis was performed using one sample t-test.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.0322
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample t-test
**Tested Non-Inferiority:** False
### Outcome Measure 3
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 2: Analysis was performed using one sample test of median (sign test). The percent change and absolute change from baseline in urine GL-3 levels were not normally distributed and thus medians were evaluated.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.5811
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample test of median (sign test)
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 4: Analysis was performed using one sample test of median (sign test). The percent change and absolute change from baseline in urine GL-3 levels were not normally distributed and thus medians were evaluated.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.7744
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample test of median (sign test)
**Tested Non-Inferiority:** False
#### Analysis
**CI Lower Limit:** 
**CI Number of Sides:** 
**CI Percentage Value:** 
**CI Upper Limit:** 
**CI Upper Limit Comment:** 
**Dispersion Type:** 
**Dispersion Value:** 
**Estimate Comment:** 
**Group Description:** Baseline versus Month 6: Analysis was performed using one sample test of median (sign test). The percent change and absolute change from baseline in urine GL-3 levels were not normally distributed and thus medians were evaluated.
**Non-Inferiority Comment:** 
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:** 
**P-Value:** 0.3877
**P-Value Comment:** 
**Parameter Type:** 
**Parameter Value:** 
**Statistical Comment:** 
**Statistical Method:** One sample test of median (sign test)
**Tested Non-Inferiority:** False
### Outcome Measure 4
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 22.540
- **Upper Limit:** 
- **Value:** -31.71
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 22.280
- **Upper Limit:** 
- **Value:** -39.04
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 23.567
- **Upper Limit:** 
- **Value:** -39.54
**Title:** 
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 21.087
- **Upper Limit:** 
- **Value:** -10.33
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 23.388
- **Upper Limit:** 
- **Value:** -12.80
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 25.291
- **Upper Limit:** 
- **Value:** -17.89
**Title:** 
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -98.4
- **Spread:** 
- **Upper Limit:** 1068.2
- **Value:** -44.71
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -96.5
- **Spread:** 
- **Upper Limit:** 464.9
- **Value:** -41.49
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** -97.1
- **Spread:** 
- **Upper Limit:** 1116.7
- **Value:** -33.75
**Title:** 
#### Outcome Measure 4
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as below quantitative limit (BQL), the lower limit of quantitation (LLOQ) value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma lyso-GL-3 was 5.0 nanogram per milliliter (ng/mL). This study is exploratory because little is known about the dose-response of these biomarkers to enzyme replacement therapy (ERT) or about the clinical significance of the biomarkers.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All enrolled participants were included in the analysis. Here, 'n' signifies participants with plasma Lyso-GL-3 assessment at the specified time point.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 2, 4, 6
**Title:** Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6
**Type:** PRIMARY
**Unit of Measure:** percent change
##### Group
**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.
**ID:** OG000
**Title:** Agalsidase Beta
#### Outcome Measure 2
**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma GL-3 was 2.0 microgram per milliliter (mcg/mL). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All enrolled participants were included in the analysis. Here, 'n' signifies participants with plasma GL-3 assessment at the specified time point.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 2, 4, 6
**Title:** Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6
**Type:** SECONDARY
**Unit of Measure:** percent change
##### Group
**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.
**ID:** OG000
**Title:** Agalsidase Beta
#### Outcome Measure 3
**Description:** Percent change from baseline = (\[post-baseline value minus baseline value\] divided by \[baseline value\]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for urine GL-3 was 0.2 mcg/mL. The absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing GL-3 (mcg/mL) by creatinine (mg/mL) and multiplying by 113.13 (mg/mmol), the molecular weight of creatinine. For levels reported BQL, the absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing 0.1 (mcg/mL) by creatinine (mg/mL) and multiplying by 133.13 (mg/mmol). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** All enrolled participants were included in the analysis. Here, 'n' signifies participants with urine GL-3 assessment at the specified time point.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 2, 4, 6
**Title:** Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6
**Type:** SECONDARY
**Unit of Measure:** percent change
##### Group
**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.
**ID:** OG000
**Title:** Agalsidase Beta
#### Outcome Measure 4
**Description:** Gastrointestinal symptoms (abdominal pain, abdominal distention, and irregular bowel movements) were to be assessed by a modified version of the Irritable Bowel Syndrome (IBS) Severity Scoring System. The modified IBS Severity Scoring System is a 7-item questionnaire. The severity score calculated by summing the scores of 5 of the 7 questions. Each of the 5 questions were scored on a scale of 0 to 100, leading to a total possible score range of 0 to 500, where higher scores indicate more severe gastrointestinal symptoms. The data for this outcome measure was exploratory and to be collected in individual participant listing only.
**Population Description:** The data for this outcome measure was exploratory and to be collected in individual participant listing only. Analysis of this data was planned only if baseline data was collected on a large number of enrolled participants. This was not the case and therefore interpretation of these results were not possible.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 2, 4, 6
**Title:** Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6
**Type:** SECONDARY
##### Group
**Description:** Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.
**ID:** OG000
**Title:** Agalsidase Beta
### Participant Flow Module
#### Group
**Description:** Commercially available agalsidase beta (Fabrazyme ®) 1.0 milligram per kilogram (mg/kg) administered as an intravenous infusion every 2 weeks (q2w) up to Month 6.
**ID:** FG000
**Title:** Agalsidase Beta
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 15
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 14
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
**Pre-Assignment Details:** A total of 16 participants were screened of which 1 participant was screen failure. A total of 15 participants were enrolled in this study.
**Recruitment Details:** The study was conducted at 6 centers in the United States of America between April 30, 2012 and March 15, 2013.
**Has Results:** True