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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: NCT01099761
Recruitment Status : Terminated (Based on preliminary safety data.)
First Posted : April 8, 2010
Results First Posted : October 14, 2016
Last Update Posted : April 5, 2017
Sponsor:
Information provided by (Responsible Party):
Acceleron Pharma, Inc.

Tracking Information
First Submitted Date  ICMJE April 2, 2010
First Posted Date  ICMJE April 8, 2010
Results First Submitted Date  ICMJE May 10, 2016
Results First Posted Date  ICMJE October 14, 2016
Last Update Posted Date April 5, 2017
Study Start Date  ICMJE April 2010
Actual Primary Completion Date June 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 22, 2016)
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: April 6, 2010)
  • 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 22, 2016)
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: April 6, 2010)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
Official Title  ICMJE 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
Brief Summary 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]
Detailed Description 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • 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.
Study Arms  ICMJE
  • 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
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)
24
Original Estimated Enrollment  ICMJE
 (submitted: April 6, 2010)
76
Actual Study Completion Date  ICMJE June 2011
Actual Primary Completion Date June 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
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 NCT01099761
Other Study ID Numbers  ICMJE A031-03
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
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.
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 March 2017

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