## Protocol Section
### Identification Module
**NCT ID:** NCT02420379

**Brief Title:** Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

**Official Title:** An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy

#### Organization Study ID Info
**ID:** 4658-203
#### Organization
**Class:** INDUSTRY
**Full Name:** Sarepta Therapeutics, Inc.
### Status Module
#### Completion Date
**Date:** 2018-12-17

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2021-01-25

**Type:** ACTUAL

**Last Update Submit Date:** 2020-12-31

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2018-12-17

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2020-07-22

**Type:** ACTUAL

**Results First Submit Date:** 2020-07-06

**Results First Submit QC Date:** 2020-07-06

#### Start Date
**Date:** 2015-06-30

**Type:** ACTUAL

**Status Verified Date:** 2020-12

#### Study First Post Date
**Date:** 2015-04-17
**Type:** ESTIMATED
**Study First Submit Date:** 2015-04-10
**Study First Submit QC Date:** 2015-04-14
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Sarepta Therapeutics, Inc.

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

**Detailed Description:** Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.

Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy.

Population and serial PK will be collected.

### Conditions Module

**Conditions:**

- Duchenne Muscular Dystrophy (DMD)

**Keywords:**

- DMD

- Duchenne muscular dystrophy

- eteplirsen

- Dystrophin

- exon 51

### Design Module

#### Design Info

**Allocation:** NON_RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 33

**Type:** ACTUAL

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg .

**Intervention Names:**

- Drug: eteplirsen

**Label:** Open-Label

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Approximately 20 patients with DMD not amenable to exon 51 skipping will be observed for 96 weeks.

**Label:** Control Group

**Type:** NO_INTERVENTION

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Open-Label

**Description:** Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.

**Name:** eteplirsen

**Other Names:**

- AVI-4658

- EXONDYS 51®

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events.

**Measure:** Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation

**Time Frame:** Baseline up to 100 weeks

**Description:** Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.

**Measure:** Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs

**Time Frame:** Baseline up to 100 weeks

**Description:** Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.

**Measure:** Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs

**Time Frame:** Baseline up to 100 weeks

**Description:** Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of participants with at least one abnormal physical examination findings were reported. Abnormality in physical examinations was based on Investigator's discretion.

**Measure:** Number of Participants With at Least One Abnormal Physical Examination Finding

**Time Frame:** Baseline up to 100 weeks

**Description:** Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECGs were reported as TEAEs.

**Measure:** Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs

**Time Frame:** Baseline up to 96 weeks

**Description:** Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECHO were reported as TEAEs.

**Measure:** Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs

**Time Frame:** Baseline up to 96 weeks

#### Secondary Outcomes

**Description:** Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value.

**Measure:** Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96

**Time Frame:** Baseline, Week 48 and 96

**Description:** Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported.

**Measure:** Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96

**Time Frame:** Baseline, Week 48 and 96

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Male 4-6 years of age.
* Diagnosis of DMD, genotypically confirmed.
* Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
* Intact right and left biceps muscles or two alternative upper arm muscle groups.
* Parent that is willing to provide consent and comply with study procedures.

Exclusion Criteria:

* Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
* Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
* Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
* Presence of other clinically significant illness.

**Maximum Age:** 6 Years

**Minimum Age:** 4 Years

**Sex:** MALE

**Standard Ages:**

- CHILD

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Phoenix

**Country:** United States

**Facility:** Neuromuscular Research Center of Arizona

**State:** Arizona

**Zip:** 85028

**Location 2:**

**City:** Los Angeles

**Country:** United States

**Facility:** Ronald Reagan UCLA Medical Center

**State:** California

**Zip:** 90095

**Location 3:**

**City:** Sacramento

**Country:** United States

**Facility:** University of California, Davis Medical Center

**State:** California

**Zip:** 95817

**Location 4:**

**City:** Stanford

**Country:** United States

**Facility:** Stanford University Medical Center

**State:** California

**Zip:** 94305

**Location 5:**

**City:** Gainesville

**Country:** United States

**Facility:** University of Florida, Shands Hospital

**State:** Florida

**Zip:** 32610

**Location 6:**

**City:** Atlanta

**Country:** United States

**Facility:** Rare Disease Research Center

**State:** Georgia

**Zip:** 30318

**Location 7:**

**City:** Atlanta

**Country:** United States

**Facility:** Children's Hospital of Atlanta

**State:** Georgia

**Zip:** 30324

**Location 8:**

**City:** Iowa City

**Country:** United States

**Facility:** University of Iowa Children's Hospital

**State:** Iowa

**Zip:** 52242

**Location 9:**

**City:** Saint Louis

**Country:** United States

**Facility:** St. Louis Children's Hospital

**State:** Missouri

**Zip:** 63110

**Location 10:**

**City:** Columbus

**Country:** United States

**Facility:** Nationwide Children's Hospital

**State:** Ohio

**Zip:** 43205

**Location 11:**

**City:** Portland

**Country:** United States

**Facility:** Shriners Hospital for Children

**State:** Oregon

**Zip:** 97239

**Location 12:**

**City:** Philadelphia

**Country:** United States

**Facility:** Children's Hospital of Philadelphia

**State:** Pennsylvania

**Zip:** 19104

**Location 13:**

**City:** Seattle

**Country:** United States

**Facility:** Seattle Children's Hospital

**State:** Washington

**Zip:** 98105

#### Overall Officials

**Official 1:**

**Affiliation:** Sarepta Therapeutics, Inc.

**Name:** Medical Director

**Role:** STUDY_DIRECTOR

## Document Section

### Large Document Module

#### Large Docs

- Date: 2019-02-11

- Filename: SAP_000.pdf

- Has ICF: False

- Has Protocol: False

- Has SAP: True

- Label: Statistical Analysis Plan

- Size: 913431

- Type Abbrev: SAP

- Upload Date: 2020-07-03T08:46

- Date: 2017-06-08

- Filename: Prot_001.pdf

- Has ICF: False

- Has Protocol: True

- Has SAP: False

- Label: Study Protocol

- Size: 1820998

- Type Abbrev: Prot

- Upload Date: 2020-07-03T08:49

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000020966

- Term: Muscular Disorders, Atrophic

- ID: D000009135

- Term: Muscular Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

- ID: D000009468

- Term: Neuromuscular Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000040181

- Term: Genetic Diseases, X-Linked

### Condition Browse Module - Browse Branches

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: All

- Name: All Conditions

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M12093

- Name: Muscular Dystrophies

- Relevance: HIGH

- As Found: Muscular Dystrophy

- ID: M22185

- Name: Muscular Dystrophy, Duchenne

- Relevance: HIGH

- As Found: Duchenne Muscular Dystrophy

- ID: M4589

- Name: Atrophy

- Relevance: LOW

- As Found: Unknown

- ID: M12092

- Name: Muscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22697

- Name: Muscular Disorders, Atrophic

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12411

- Name: Neuromuscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M24877

- Name: Genetic Diseases, X-Linked

- Relevance: LOW

- As Found: Unknown

- ID: T3963

- Name: Muscular Dystrophy

- Relevance: HIGH

- As Found: Muscular Dystrophy

- ID: T698

- Name: Becker Muscular Dystrophy

- Relevance: HIGH

- As Found: Duchenne Muscular Dystrophy

- ID: T1945

- Name: Duchenne Muscular Dystrophy

- Relevance: HIGH

- As Found: Duchenne Muscular Dystrophy

### Condition Browse Module - Meshes

- ID: D000009136

- Term: Muscular Dystrophies

- ID: D000020388

- Term: Muscular Dystrophy, Duchenne

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

#### Event Groups

**Group ID:** EG000

**Title:** Eteplirsen 30 mg/kg

**Deaths Num At Risk:** 26

**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.

**ID:** EG000

**Other Num Affected:** 26

**Other Num at Risk:** 26

**Serious Number Affected:** 4

**Serious Number At Risk:** 26

**Title:** Eteplirsen 30 mg/kg

**Group ID:** EG001

**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)

**Deaths Num At Risk:** 7

**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.

**ID:** EG001

**Other Num Affected:** 5

**Other Num at Risk:** 7

**Serious Number At Risk:** 7

**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)

**Frequency Threshold:** 5

#### Other Events

**Term:** Nasopharyngitis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Upper respiratory tract infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Ear infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Influenza

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Otitis media

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Pharyngitis streptococcal

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Rhinitis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Conjunctivitis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Gastroenteritis viral

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Staphylococcal infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Impetigo

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Cough

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Rhinorrhoea

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Nasal congestion

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Epistaxis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Oropharyngeal pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Productive cough

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Upper respiratory tract congestion

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Vomiting

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Diarrhoea

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Abdominal pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Abdominal pain upper

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Constipation

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Nausea

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Dental caries

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Fall

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Contusion

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Procedural pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Skin abrasion

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Scratch

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Head injury

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Sunburn

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Ligament sprain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Pain in extremity

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Arthralgia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Back pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Muscle spasms

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Muscular weakness

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Musculoskeletal pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Scoliosis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Rash

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Dermatitis contact

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Keloid scar

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Rash pruritic

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

**Term:** Pyrexia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Catheter site pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Catheter site bruise

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Headache

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Migraine

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Nervous system disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Ear pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Ear and labyrinth disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Cardiac murmur

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Investigations

**Source Vocabulary:** MedDRA (17.1)

**Term:** Aggression

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Psychiatric disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Hypersensitivity

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Immune system disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Chromaturia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Renal and urinary disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Decreased appetite

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Metabolism and nutrition disorders

**Source Vocabulary:** MedDRA (17.1)

**Term:** Skin papilloma

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)

**Source Vocabulary:** MedDRA (17.1)

**Term:** Adenoidectomy

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Surgical and medical procedures

**Source Vocabulary:** MedDRA (17.1)

**Term:** Myringotomy

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Surgical and medical procedures

**Source Vocabulary:** MedDRA (17.1)

**Term:** Orchidopexy

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Surgical and medical procedures

**Source Vocabulary:** MedDRA (17.1)

#### Serious Events

**Term:** Foreign body

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 26

**Group ID:** EG001

**Num At Risk:** 7

**Term:** Coxsackie viral infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 26

**Group ID:** EG001

**Num At Risk:** 7

**Term:** Influenza

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 26

**Group ID:** EG001

**Num At Risk:** 7

**Term:** Rhinovirus infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** MedDRA (17.1)

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 26

**Group ID:** EG001

**Num At Risk:** 7

**Term:** Rhabdomyolysis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

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

**Source Vocabulary:** MedDRA (17.1)

##### Stats

**Group ID:** EG000

**Num Affected:** 1

**Num At Risk:** 26

**Group ID:** EG001

**Num At Risk:** 7

**Time Frame:** Baseline up to 100 weeks

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 26

**Group ID:** BG001

**Value:** 7

**Group ID:** BG002

**Value:** 33

**Units:** Participants

### Group

**ID:** BG000

**Title:** Eteplirsen 30 mg/kg

**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.

### Group

**ID:** BG001

**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)

**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.

### Group

**ID:** BG002

**Title:** Total

**Description:** Total of all reporting groups

### Measure

#### Measurement

**Group ID:** BG000

**Spread:** 0.82

**Value:** 5.0

#### Measurement

**Group ID:** BG001

**Spread:** 1.00

**Value:** 5.0

#### Measurement

**Group ID:** BG002

**Spread:** 0.85

**Value:** 5.0

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 0

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 26

#### Measurement

**Group ID:** BG001

**Value:** 7

#### Measurement

**Group ID:** BG002

**Value:** 33

**Category Title:** Male

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 5

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 5

**Category Title:** Hispanic or Latino

#### Measurement

**Group ID:** BG000

**Value:** 21

#### Measurement

**Group ID:** BG001

**Value:** 7

#### Measurement

**Group ID:** BG002

**Value:** 28

**Category Title:** Not Hispanic or Latino

#### 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:** 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:** 2

#### Measurement

**Group ID:** BG001

**Value:** 1

#### Measurement

**Group ID:** BG002

**Value:** 3

**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:** 1

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 1

**Category Title:** Black or African American

#### Measurement

**Group ID:** BG000

**Value:** 22

#### Measurement

**Group ID:** BG001

**Value:** 6

#### Measurement

**Group ID:** BG002

**Value:** 28

**Category Title:** White

#### Measurement

**Group ID:** BG000

**Value:** 1

#### Measurement

**Group ID:** BG001

**Value:** 0

#### Measurement

**Group ID:** BG002

**Value:** 1

**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:** 26

#### Measurement

**Group ID:** BG001

**Value:** 7

#### Measurement

**Group ID:** BG002

**Value:** 33

**Class Title:** United States

**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:** COUNT_OF_PARTICIPANTS
**Title:** Ethnicity (NIH/OMB)
**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:** Region of Enrollment
**Unit of Measure:** participants
**Population Description:** Full set included all participants who were enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all participants who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment.
## Results Section - More Information Module
### Certain Agreement
**Other Details:** The most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Point of Contact
**Email:** clinicaltrials@sarepta.com
**Organization:** Sarepta Therapeutics, Inc.
**Phone:** +1-888-727-3782
**Title:** Medical Director
## 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
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 26
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 5
**Title:** 
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 4
- **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:** 0
**Title:** 
#### Outcome Measure 2
**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:** 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:** 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:** 0
**Title:** 
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 11
- **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:** 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 4
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 23
- **Comment:** 
- **Group ID:** OG001
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 4
**Title:** 
#### Outcome Measure 5
**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 6
**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 7
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 0.0896
- **Upper Limit:** 
- **Value:** 0.102
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 0.4863
- **Upper Limit:** 
- **Value:** 0.321
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 0.0096
- **Upper Limit:** 
- **Value:** 0.004
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 14
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 0.0175
- **Upper Limit:** 
- **Value:** 0.015
**Title:** 
**Denomination:**
- **Group ID:** OG000
- **Value:** 11
**Units:** Participants
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who were enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all participants who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 100 weeks
**Title:** Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 2
**Description:** Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 100 weeks
**Title:** Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 3
**Description:** Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who were enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 100 weeks
**Title:** Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 4
**Description:** Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of participants with at least one abnormal physical examination findings were reported. Abnormality in physical examinations was based on Investigator's discretion.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment postenrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 100 weeks
**Title:** Number of Participants With at Least One Abnormal Physical Examination Finding
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 5
**Description:** Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECGs were reported as TEAEs.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at 1 assessment post-enrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 96 weeks
**Title:** Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 6
**Description:** Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECHO were reported as TEAEs.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment.
**Reporting Status:** POSTED
**Time Frame:** Baseline up to 96 weeks
**Title:** Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
##### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** OG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Outcome Measure 7
**Description:** Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 48 and 96
**Title:** Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96
**Type:** SECONDARY
**Unit of Measure:** Percent Normal Dystrophin Protein Level
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
#### Outcome Measure 8
**Description:** Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 48 and 96
**Title:** Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96
**Type:** SECONDARY
**Unit of Measure:** Percent dystrophin positive fibers
##### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** OG000
**Title:** Eteplirsen 30 mg/kg
### Participant Flow Module
#### Group
**Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks.
**ID:** FG000
**Title:** Eteplirsen 30 mg/kg
#### Group
**Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks.
**ID:** FG001
**Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants)
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 3
##### Withdraw
**Type:** Unable to complete due to schooling
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Transitioned to commercial drug
###### 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:** 26
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 25
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 3
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 4
**Pre-Assignment Details:** A total of 33 participants were enrolled in the study treatment.
**Recruitment Details:** The study was conducted at 13 sites in the United States.
**Has Results:** True