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

This study has been terminated.
(This study was terminated based on preliminary safety data.)
Sponsor:
Information provided by (Responsible Party):
Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT01239758
First received: November 1, 2010
Last updated: January 30, 2013
Last verified: January 2013
  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.]


Condition Intervention 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 Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Acceleron Pharma, Inc.:

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

Secondary Outcome Measures:
  • Percent change in total lean body mass by DXA scan. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
  • Percent change in muscle strength score by hand-held myometry. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ] [ Designated as safety issue: No ]
  • Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test). [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
  • Change in pulmonary function tests. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ] [ Designated as safety issue: No ]

Enrollment: 11
Study Start Date: October 2010
Study Completion Date: May 2011
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01239758

Locations
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

No publications provided

Responsible Party: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01239758     History of Changes
Other Study ID Numbers: A031-06
Study First Received: November 1, 2010
Last Updated: January 30, 2013
Health Authority: Canada: Health Canada
United States: Food and Drug Administration

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on October 30, 2014