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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PRO044 SC 6 mg/kg | Experimental | Weekly subcutaneous (SC) dosing with 6 mg/kg |
|
| PRO044 IV 6 mg/kg | Experimental | Weekly intravenous (IV) dosing with 6 mg/kg |
|
| PRO044 SC 9 mg/kg | Experimental | Weekly intravenous (IV) dosing with 9 mg/kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PRO044 SC 6 mg/kg | Drug |
| ||
| PRO044 IV 6 mg/kg |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of PRO044 (composite of several measures) | Efficacy parameters: Muscle Function
Muscle strength
Exploratory:
| 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 | After 48 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Assess the pharmacokinetics of PRO044 (composite of several measures) | Pharmacokinetic parameters:
| After 48 weeks of treatment |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UZ Leuven | Leuven | Belgium | ||||
| S.Anna Hospital |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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|
| PRO044 IV 9 mg/kg | Drug |
|
| Ferrara |
| Italy |
| Policlinico Universitario Agostino Gemelli | Roma | Italy |
| Leids Universitair Medisch Centrum | Leiden | Netherlands |
| Drottning Silvias Barn- ochungdomssjukhus | Gothenburg | Sweden |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |