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

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terminated

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

key information

study id #: NCT01239758

condition: Duchenne Muscular Dystrophy

status: terminated

purpose:

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.]

intervention:
ACE-031 (Extension of cohort 1 from core study, A031-03),
ACE-031 (Extension of cohort 2 from core study, A031-03),
ACE-031 (Extension of cohort 3 from core study, A031-03)

mechanism of action: Myostatin inhibitor to promote muscle growth

results: https://clinicaltrials.gov/ct2/show/results/NCT01239758

study details

start date: October 2010

estimated completion: May 2011

phase of development: Phase 2

size / enrollment: 11

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.] 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.]

secondary outcomes:

  • 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.]

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

study contacts

sponsor: Acceleron Pharma, Inc.

locations: Canada