Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy - Full Text View

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 Biological: ACE-031 2.5 mg/kg q4wk 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:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy

U.S. FDA Resources

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:	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.
Experimental: ACE-031 ACE-03 2.5 mg/kg q4wk	Biological: ACE-031 2.5 mg/kg q4wk ACE-031 2.5 mg/kg subcutaneously once every 4 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
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

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

No publications provided

Responsible Party:	Acceleron Pharma, Inc.
ClinicalTrials.gov Identifier:	NCT01099761 History of Changes
Other Study ID Numbers:	A031-03
Study First Received:	April 2, 2010
Last Updated:	January 31, 2013
Health Authority:	Canada: Health Canada United States: Food and Drug Administration

Additional relevant MeSH terms:

Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases

Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 22, 2013