The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks. Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
15
UZ Leuven
Leuven, Belgium
S.Anna Hospital
Ferrara, Italy
Policlinico Universitario Agostino Gemelli
Roma, Italy
Leids Universitair Medisch Centrum
Leiden, Netherlands
Drottning Silvias Barn- ochungdomssjukhus
Efficacy of PRO044 (composite of several measures)
Efficacy parameters: Muscle Function * 6 Minute Walk Distance (6MWD) * North Star Ambulatory Assessment * Timed tests (10-meter walk/run, rising from floor, stair climb) * DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only * Egen Klassification - for non-ambulant subjects. Muscle strength * Pulmonary Function (Spirometry) * Handheld myometry. Exploratory: * Performance Upper Limb (PUL). * Patient Reported Outcome measure (PROM).
Time frame: After 48 weeks of treatment
Safety and tolerability of PRO044 (treatement emergent adverse events)
Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing
Time frame: After 48 weeks of treatment
Assess the pharmacokinetics of PRO044 (composite of several measures)
Pharmacokinetic parameters: * t ½ * AUC: 0-24h, 0-∞ (where applicable) * Cmax * tmax * CL (for IV subjects) or CL/F (for SC subjects) * PRO044 concentrations in muscle tissue.
Time frame: After 48 weeks of treatment
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Gothenburg, Sweden