This trial is active, not recruiting.

Condition muscular dystrophy, duchenne
Treatment eteplirsen
Phase phase 2
Sponsor Sarepta Therapeutics
Start date November 2014
End date May 2017
Trial size 20 participants
Trial identifier NCT02286947, 4658-204


The primary objective of this study is to explore safety and tolerability of eteplirsen in patients with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The exploratory objectives are to evaluate the effect of eteplirsen on pulmonary function tests (PFTs) and other functional clinical measures.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Approximately 20 patients with advanced stage Duchenne muscular dystrophy (DMD) who have genotypically confirmed mutations amenable to treatment by exon 51 skipping, will receive weekly infusions of eteplirsen 30 mg/kg for 96 weeks.
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks.

Primary Outcomes

Number of patients with treatment emergent adverse events.
time frame: 96 weeks

Secondary Outcomes

Number of patients with clinical laboratory abnormalities
time frame: 96 weeks
Number of patients with abnormalities in vital signs and ECG
time frame: 96 weeks
Mean Change from baseline in maximum inspiratory pressure (MIP) % predicted
time frame: Baseline to Week 96
Mean Change from baseline in maximum expiratory pressure (MEP) % predicted
time frame: Baseline to Week 96

Eligibility Criteria

Male participants from 7 years up to 21 years old.

Inclusion Criteria: - 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 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 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.

Additional Information

Official title An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
Description This is an open-label, multi-center, 96-week study to explore the safety and tolerability of eteplirsen injection in patients with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping. Patients will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible patients will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for up to 96 weeks. An extension to the dosing period may be considered prior to the end of the 96-week planned dosing period. Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory efficacy assessments, including PFTs, upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by Sarepta Therapeutics.