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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:
ClinicalTrials.gov Identifier:
NCT01099761
First Posted: April 8, 2010
Last Update Posted: April 5, 2017
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.
Information provided by (Responsible Party):
Acceleron Pharma, Inc.
April 2, 2010
April 8, 2010
May 10, 2016
October 14, 2016
April 5, 2017
April 2010
June 2011   (Final data collection date for primary outcome measure)
  • Number of Subjects With Adverse Reactions. [ Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later ]
    Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug
  • Number of Subjects With Clinical Laboratory Adverse Reactions. [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
    Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug
  • 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 ]
  • Percent change from baseline in total body lean mass by DXA [ Time Frame: 4 months ]
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. ]
  • Percent Change in 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. ]
    Manual Muscle Testing (MMT) is a procedure to measure the function and strength of individual muscles and muscle groups. Hand-held myometry, using a device known as a dynamometer, is one method used for MMT. The dynamometer is held against the patient's limb by the examiner and the patient is asked to resist the force applied by the examiner. The dynamometer measures the force applied by the patient, providing a quantitative and objective assessment of strength of the particular muscle or muscle group. The effectiveness of a therapeutic intervention on muscle strength, as measured by hand-held myometry, can be assessed by comparing post-treatment to pre-treatment (baseline) measurements.
  • 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 distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (<10 years vs. >=10 years)
  • Change From Baseline 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 (FVC) [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ]
    Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study
  • Change in Pulmonary Function Test (MIP) [ Time Frame: Baseline to End-of-Study Visit. approximately 24 weeks ]
    Maximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study
  • Change in Pulmonary Function Test (MEP) [ Time Frame: Baseline to End-of-Stuidy Visit, approximately 24 weeks ]
    Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study
  • Change from baseline in timed function tests [ Time Frame: 6 months ]
  • Change from baseline in muscle strength tests [ Time Frame: 6 months ]
  • Change from baseline in pulmonary function tests [ Time Frame: 6 months ]
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 boys 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 safety data]
ACE-031, a soluble form of the human activin receptor type IIB, was administered once every 2 to 4 weeks by subcutaneous (SC) injection to boys with DMD. Dose levels and regimens for this multiple-dose study were based on data from the initial clinical studies in healthy subjects in which doses of 0.02 to 3 mg/kg SC were evaluated. A total of 24 subjects were enrolled into the study; 18 received ACE-031 and 6 placebo. All subjects were treated for a period of 12 weeks.The pharmacodynamic effects of ACE-031 treatment were assessed by a battery of motor function test that included the 6-Minute Walk Test, the 10-Minute Walk/Run Test, the 4-Stair Climb Test and the Gower Maneuver (GW). Muscle strength was assessed by hand-held myometry and fixed system testing. Body composition (i.e., spine BMD, lean mass, and fat mass) was assessed by whole body and lumbar spine DXA scans. Pulmonary function was assessed by forced vital capacity (FVC), maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP). ACE-031 safety was evaluated through observation of the incidence and severity of adverse events.
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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.
  • 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
  • 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
Sexes Eligible for Study: Male
4 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Canada
 
 
NCT01099761
A031-03
Yes
Not Provided
Plan to Share IPD: No
Plan Description: Abstract summarizing trial data has been published online in Muscle & Nerve 23-Dec-2016 https://www.ncbi.nlm.nih.gov/pubmed/27462804 The Sponor currently has no plans to make IPD available for a number of reasons; (i) the study was terminated by the Sponsor prematurely based upon concerns for potential adverse drug reactions following long-term use, which were identified in chronic toxicity studies in animals, (ii) the study population was one with a rare disease, in a groups of subjects with a relatively narrow age range, across a small number of study sites. The Sponsor believes that these factors, taken together, make patient anonymity a significant challenge.
Acceleron Pharma, Inc.
Acceleron Pharma, Inc.
Not Provided
Not Provided
Acceleron Pharma, Inc.
March 2017

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