A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 9.1Level: LLTClassification code 10013801

• Registration Number: EUCTR2009-013762-63-IT
• Lead Sponsor: PROSENSA THERAPEUTICS BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04-27
• Last Update Posted: 19 August 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 18

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:
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 of 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