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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.

Study Description
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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.]

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy 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) Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Study Start Date : October 2010
Actual Primary Completion Date : May 2011
Actual Study Completion Date : May 2011

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Arms and Interventions
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Arm Intervention/treatment
Experimental: ACE-031 (Extension of cohort 1 from core study, A031-03) 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.
Experimental: ACE-031 (Extension of cohort 2 from core study, A031-03) 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.
Experimental: ACE-031 (Extension of cohort 3 from core study, A031-03) 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.


Outcome Measures
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Primary Outcome Measures :
  1. Number of patients with adverse events. [ Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later. ]
  2. Change in laboratory parameters and vital signs. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]

Secondary Outcome Measures :
  1. Percent change in total lean body mass by DXA scan. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  2. 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. ]
  3. Percent change in muscle strength score by hand-held myometry. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  4. Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test). [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
  5. 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. ]
  6. Change in pulmonary function tests. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]

Eligibility Criteria
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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01239758


Locations
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Canada, Alberta
Acceleron Investigative Site
Calgary, Alberta, Canada
Canada, Ontario
Acceleron Investigative Site
Hamilton, Ontario, Canada
Acceleron Investigative Site
London, Ontario, Canada
Acceleron Investigative Site
Ottawa, Ontario, Canada
Sponsors and Collaborators
Acceleron Pharma Inc.
More Information
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Responsible Party: Acceleron Pharma Inc.
ClinicalTrials.gov Identifier: NCT01239758    
Other Study ID Numbers: A031-06
First Posted: November 11, 2010    Key Record Dates
Last Update Posted: February 1, 2013
Last Verified: January 2013
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked