Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 4658-204
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 9

Brief Summary

The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Conditions

Muscular Dystrophy, Duchenne

Keywords

DMD; exon 51; dystrophin; dystrophy; eteplirsen; Duchenne

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Emergent Adverse Events

  An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A 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 AEs 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 pretreatment state. AEs included both serious and non-serious adverse events.

  From first dose of drug up to 100 weeks

Secondary Outcomes (5)

• Number of Participants With Potentially Clinically Significant Laboratory Abnormalities

  Baseline up to 100 weeks

• Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs

  Baseline up to 100 weeks

• Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations

  Baseline up to 100 weeks

• Number of Participants With Abnormalities in Electrocardiograms (ECGs)

  Baseline up to 100 weeks

• Number of Participants With Abnormalities in Echocardiograms (ECHO)

  Baseline up to 100 weeks

Study Arms (1)

Eteplirsen 30 mg/kg

EXPERIMENTAL

Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks.

Drug: Eteplirsen

Interventions

Eteplirsen DRUG

Eteplirsen solution for IV infusion

Also known as: AVI-4658, EXONDYS 51®

Eteplirsen 30 mg/kg

Eligibility Criteria

• Age: 7 Years - 21 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male 7 - 21 years of age
• Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
• Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
• Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
• Score of ≤4 on the Brooke Score for Arms and Shoulders.
• Stable cardiac and pulmonary function
• Use of contraceptives for sexually active males throughout the study
• Willing to provide consent and comply with the study

You may not qualify if:

• 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 treatment with SMT C1100/BMN 195 at any time.
• Previous treatment with drisapersen (PRO051) within the last 6 months.
• Participation in any other DMD interventional clinical study within 12 weeks
• Major change in physiotherapy regimen within the past 3 months
• Major surgery within 3 months
• Presence of other clinically significant illness
• Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
• Forced vital capacity % predicted \[FVC % predicted\] \<40%, or requiring daytime ventilation.
• Require antiarrhythmic and/or antidiuretic therapy for heart failure.
• Have a left ventricular ejection fraction (LVEF) of \<40%.
• Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (9)

Ronald Reagan UCLA Medical Center

Los Angeles, California, 90095, United States

Location

University of California, Davis Medical Center

Sacramento, California, 95817, United States

Location

University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

eteplirsen

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Medical Director
• Organization: Sarepta Therapeutics, Inc.

clinicaltrials@sarepta.com

+1-888-727-3782

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2014
• First Posted: November 10, 2014
• Study Start: November 1, 2014
• Primary Completion: April 21, 2017
• Study Completion: March 23, 2018
• Last Updated: March 30, 2020
• Results First Posted: February 20, 2019

Record last verified: 2020-03

Locations

US (9)