Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

Phase 1

• Conditions: Patients with Duchenne Muscular Dystropy 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: EUCTR2016-005023-92-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: 40

Inclusion Criteria

1. Male 4-6 years of age
2. Diagnosis of DMD, genotypically confirmed
3. Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks
4. Intact right and left biceps muscles or two alternative upper arm muscle groups

Are the trial subjects under 18? yes
Number of subjects for this age range: 40
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. 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)
2. Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months
3. 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
4. Presence of other clinically significant illness

Study & Design

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