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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: November 22, 2016
Last verified: November 2016
  Purpose
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]

Condition Intervention Phase
Duchenne Muscular Dystrophy
Biological: ACE-031 0.5 mg/kg q4wk
Biological: ACE-031 1.0 mg/kg q2wk
Other: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Number of Subjects With Adverse Reactions. [ Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later ] [ Designated as safety issue: Yes ]
    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. ] [ Designated as safety issue: Yes ]
    Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug


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 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 ]
    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. ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
  • Change in Pulmonary Function Tests (FVC) [ Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later. ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study


Enrollment: 24
Study Start Date: April 2010
Study Completion Date: June 2011
Primary Completion Date: June 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ACE-031 0.5 mg/kg q4wk Biological: ACE-031 0.5 mg/kg q4wk
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
Experimental: ACE-031 1.0 mg/kg q2wk Biological: ACE-031 1.0 mg/kg q2wk
ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
Placebo Comparator: Placebo Other: Placebo
Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.

  Eligibility

Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  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: NCT01099761

Locations
Canada, Alberta
Acceleron Investigative Site
Calgary, Alberta, Canada
Canada, British Columbia
Acceleron Investigative Site
Vancouver, British Columbia, 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

Responsible Party: Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01099761     History of Changes
Other Study ID Numbers: A031-03 
Study First Received: April 2, 2010
Results First Received: May 10, 2016
Last Updated: November 22, 2016
Health Authority: Canada: Health Canada
United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on December 09, 2016