Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01239758
• Date of registration: 01/11/2010
• Prospective Registration: No
• Primary sponsor: Acceleron Pharma, Inc.
• Public title: Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
• Scientific title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
• Date of first enrolment: October 2010
• Target sample size: 11
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT01239758
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Canada

Key inclusion & exclusion criteria

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



Age minimum: 4 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)

Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)

Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)

Primary Outcome(s)

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 Outcome(s)

Percent change in total lean body mass by DXA scan. [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.]

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.]

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.]

Percent change in muscle strength score by hand-held myometry. [Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.]

Secondary ID(s)

A031-06

Source(s) of Monetary Support

Please refer to primary and secondary sponsors