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
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- EUCTR2009-013762-63-BE
- Lead Sponsor
- Prosensa Therapeutics B.V
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- Male
- Target Recruitment
- 24
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
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
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
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