A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne Disease

• Conditions: Duchenne Muscular Dystrophy; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]; MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2009-013762-63-SE
• Lead Sponsor: Prosensa Therapeutics B.V

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-11-03
• Last Update Posted: 5 August 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

1.Age at least 5 and not older than 16 years on the day of first drug administration
2.Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044
3.Life expectancy at least 6 months after inclusion in the trial

Are the trial subjects under 18? yes
Number of subjects for this age range: 24
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. Ventilator dependency
2. Previous treatment with investigational medicinal treatment within 6 months prior to the start of the pre-screening for the study
3. Previous treatment with idebenone within 6 months prior to the start of the pre-screening for the study.

Study & Design

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