Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)

Phase 1

• Conditions: Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping; MedDRA version: 19.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2016-005002-19-Outside-EU/EEA
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-03-28
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: A
• Sex: Male
• Target Recruitment: 160

Inclusion Criteria

1. Male 7-16 years old
2. Diagnosed with DMD, genotypically confirmed
3. Stable dose of corticosteroids for at least 24 weeks
4. Have intact right and left alternative upper muscle groups
5. Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
6. Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
Are the trial subjects under 18? yes
Number of subjects for this age range: 160
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months
2. Participation in any other DMD interventional clinical study within 12 weeks
3. Major surgery within 3 months
4. Presence of other clinically significant illness
5. Major change in the physical therapy regime within 3 months

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified