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. \[Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.\]
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
11
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Acceleron Investigative Site
Calgary, Alberta, Canada
Acceleron Investigative Site
Hamilton, Ontario, Canada
Acceleron Investigative Site
London, Ontario, Canada
Acceleron Investigative Site
Ottawa, Ontario, Canada
Number of patients with adverse events.
Time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total lean body mass by DXA scan.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total body and lumbar spine bone mineral density by DXA scan.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in muscle strength score by hand-held myometry.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in pulmonary function tests.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
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