Conditions: Duchenne Muscular Dystrophy
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 18
Healthy volunteers: no

Brief summary:
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.

Detailed description:
To assess the effect of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To assess the safety and tolerability of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous and intravenous administration in subjects with Duchenne muscular dystrophy.

Arms:
arm 1: Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29. arm 2: Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29. arm 3: Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29. arm 4: Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29. arm 5: Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29 arm 6: Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29 arm 7: Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29 arm 8: Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29 arm 9: Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29

Interventions: intervention 1: PRO044 SC intervention 2: PRO044 IV
Intervention descriptions: intervention 1: Subcutaneous injection, once a week, for five weeks intervention 2: Intravenous injection, once a week, for five weeks

Protocol document: