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Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

This study has been terminated.
(Based on preliminary safety data.)
Sponsor:
Information provided by (Responsible Party):
Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT01099761
First received: April 2, 2010
Last updated: January 31, 2013
Last verified: January 2013

April 2, 2010
January 31, 2013
April 2010
June 2011   (final data collection date for primary outcome measure)
  • 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 ]
  • Safety and tolerability by monitoring adverse events, clinical laboratory tests, electrocardiogram (ECG), echocardiogram (ECHO), physical examinations, vital signs, and anti-drug antibodies [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Percent change from baseline in total body lean mass by DXA [ Time Frame: 4 months ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01099761 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Change from baseline in timed function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Change from baseline in muscle strength tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Change from baseline in pulmonary function tests [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy

The purpose of this study is to determine if ACE-031 is safe and well-tolerated in children with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies. [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]

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
  • Biological: ACE-031 0.5 mg/kg q4wk
    ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
  • Biological: ACE-031 1.0 mg/kg q2wk
    ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
  • Biological: ACE-031 2.5 mg/kg q4wk
    ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
  • Other: Placebo
    Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.
  • Experimental: ACE-031 0.5 mg/kg q4wk
    Intervention: Biological: ACE-031 0.5 mg/kg q4wk
  • Experimental: ACE-031 1.0 mg/kg q2wk
    Intervention: Biological: ACE-031 1.0 mg/kg q2wk
  • Experimental: ACE-031 ACE-03 2.5 mg/kg q4wk
    Intervention: Biological: ACE-031 2.5 mg/kg q4wk
  • Placebo Comparator: Placebo
    Intervention: Other: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
24
June 2011
June 2011   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of DMD confirmed
  • Ambulant
  • Corticosteroid therapy for at least one year prior to study day 1 and on a stable dose and schedule for at least 6 months prior to study day 1
  • Evidence of muscle weakness by clinical assessment

Exclusion Criteria:

  • Any previous treatment with another investigational product within 6 months prior to study day 1
  • Any clinically significant cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease that is not related to DMD
  • Inability to perform a whole body dual x-ray absorptiometry (DXA) scan
Male
4 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Canada
 
NCT01099761
A031-03
Yes
Acceleron Pharma, Inc.
Acceleron Pharma, Inc.
Not Provided
Not Provided
Acceleron Pharma, Inc.
January 2013

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