Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

January 30, 2013 updated by: Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)

An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy

To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [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.]

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

11

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Acceleron Investigative Site
    • Ontario
      • Hamilton, Ontario, Canada
        • Acceleron Investigative Site
      • London, Ontario, Canada
        • Acceleron Investigative Site
      • Ottawa, Ontario, Canada
        • Acceleron Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Completion of participation in Study A031-03 and Investigator approval
  • Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03

Exclusion Criteria:

  • Participation in any other therapeutic clinical trial
  • Plans to have surgery during the course of the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: ACE-031 (Extension of cohort 1 from core study, A031-03)
Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
EXPERIMENTAL: ACE-031 (Extension of cohort 2 from core study, A031-03)
Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
EXPERIMENTAL: ACE-031 (Extension of cohort 3 from core study, A031-03)
Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of patients with adverse events.
Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Baseline to End-of-Study Visit, approximately 24 weeks later.

Secondary Outcome Measures

Outcome Measure
Time Frame
Percent change in total lean body mass by DXA scan.
Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Baseline to End-of-Study Visit, approximately 24 weeks later.
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.
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.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Baseline to End-of-Study Visit, approximately 24 weeks later.
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.
Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in pulmonary function tests.
Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Baseline to End-of-Study Visit, approximately 24 weeks later.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2010

Primary Completion (ACTUAL)

May 1, 2011

Study Completion (ACTUAL)

May 1, 2011

Study Registration Dates

First Submitted

November 1, 2010

First Submitted That Met QC Criteria

November 10, 2010

First Posted (ESTIMATE)

November 11, 2010

Study Record Updates

Last Update Posted (ESTIMATE)

February 1, 2013

Last Update Submitted That Met QC Criteria

January 30, 2013

Last Verified

January 1, 2013

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A031-06

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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