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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01239758
Recruitment Status : Terminated (This study was terminated based on preliminary safety data.)
First Posted : November 11, 2010
Last Update Posted : February 1, 2013
Sponsor:
Information provided by (Responsible Party):
Acceleron Pharma Inc.

Tracking Information
First Submitted Date  ICMJE November 1, 2010
First Posted Date  ICMJE November 11, 2010
Last Update Posted Date February 1, 2013
Study Start Date  ICMJE October 2010
Actual Primary Completion Date May 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 22, 2013)
  • 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. ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 10, 2010)
Safety and tolerability as measured by number of adverse events and serious adverse events [ Time Frame: 7 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2013)
  • 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. ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 10, 2010)
  • Change from baseline in timed function tests [ Time Frame: 7 months ]
  • Change from baseline in muscle strength tests [ Time Frame: 7 months ]
  • Change from baseline in pulmonary function tests [ Time Frame: 7 months ]
  • Change from baseline in body composition [ Time Frame: 7 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
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.]
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)
    ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
  • Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)
    Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
  • Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)
    Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Study Arms  ICMJE
  • Experimental: ACE-031 (Extension of cohort 1 from core study, A031-03)
    Intervention: Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)
  • Experimental: ACE-031 (Extension of cohort 2 from core study, A031-03)
    Intervention: Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)
  • Experimental: ACE-031 (Extension of cohort 3 from core study, A031-03)
    Intervention: Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 19, 2011)
11
Original Estimated Enrollment  ICMJE
 (submitted: November 10, 2010)
76
Actual Study Completion Date  ICMJE May 2011
Actual Primary Completion Date May 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01239758
Other Study ID Numbers  ICMJE A031-06
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Acceleron Pharma Inc.
Study Sponsor  ICMJE Acceleron Pharma Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Acceleron Pharma Inc.
Verification Date January 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP