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This study was terminated based on preliminary safety data.
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To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.]
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ACE-031 (Extension of cohort 1 from core study, A031-03) | Experimental |
| |
| ACE-031 (Extension of cohort 2 from core study, A031-03) | Experimental |
| |
| ACE-031 (Extension of cohort 3 from core study, A031-03) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACE-031 (Extension of cohort 1 from core study, A031-03) | Biological | ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients with adverse events. | From treatment initiation to End-of-Study Visit, approximately 24 weeks later. | |
| Change in laboratory parameters and vital signs. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Measure | Description | Time Frame |
|---|---|---|
| Percent change in total lean body mass by DXA scan. | Baseline to End-of-Study Visit, approximately 24 weeks later. | |
| Percent change in total body and lumbar spine bone mineral density by DXA scan. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Acceleron Investigative Site | Calgary | Alberta | Canada | |||
| Acceleron Investigative Site |
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| ACE-031 (Extension of cohort 2 from core study, A031-03) | Biological | Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks. |
|
| ACE-031 (Extension of cohort 3 from core study, A031-03) | Biological | Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks. |
|
| Percent change in muscle strength score by hand-held myometry. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test). | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test). | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in pulmonary function tests. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Hamilton |
| Ontario |
| Canada |
| Acceleron Investigative Site | London | Ontario | Canada |
| Acceleron Investigative Site | Ottawa | Ontario | Canada |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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