Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Sarepta Therapeutics

Status and phase

Completed

Phase 2

Conditions

Muscular Dystrophy, Duchenne

Treatments

Drug: Eteplirsen

Study type

Interventional

Funder types

Industry

Identifiers

NCT02286947

4658-204

Details and patient eligibility

About

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.

Full description

This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.

Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).

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.

Enrollment

24 patients

Sex

Male

Ages

7 to 21 years old

Volunteers

No Healthy Volunteers

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.