Duchenne disease Duchenne muscular dystrophy
Conditions
Interventions
If patient and parents consented to participate to the clinical trial, the
patient will be allocated to one of the following arms:
- Weekly subcutaneous dosing with PRO044 6mg/kg
- Weekly intraveneou
Sponsors
Prosensa Therapeutics B.V.
Eligibility
Age
12 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Subjects previously treated with PRO044 in the PRO044-CLIN-01 study.;2. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticoids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor.
Exclusion criteria
Exclusion criteria: 1. Current or history of liver or renal disease. 2. Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements. 3. Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study. 4. Need for daytime mechanical ventilation. 5. Screening aPTT above the upper limit of normal (ULN). 6. Screening platelet count below the lower limit of normal (LLN). 7. Use of anticoagulants, antithrombotics or antiplatelet agents. 8. Use of any investigational product within 6 months prior to the start of Screening for the study or during participation in the study. 9. Current or history of drug and/or alcohol abuse.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety, tolerability and efficacy | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic profile | — |
Countries
Netherlands
Outcome results
None listed