NCT01239758TerminatedPhase 2
An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA4 sites in 1 country11 target enrollmentStarted: October 1, 2010Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Sponsor
- Enrollment
- 11
- Locations
- 4
- Primary Endpoint
- Number of patients with adverse events.
Study Overview
Brief Summary
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 Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 4 Years to — (Child, Adult, Older Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Completion of participation in Study A031-03 and Investigator approval
- •Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
Exclusion Criteria
- •Participation in any other therapeutic clinical trial
- •Plans to have surgery during the course of the study
Outcomes
Primary Outcomes
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.
Secondary Outcomes
- Change in pulmonary function tests.(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- 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.)
- 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.)
Investigators
Study Sites (4)
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