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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 2
Locations
Canada
Study Type
Interventional
Intervention
ACE-031 (Extension of cohort 1 from core study, A031-03)
ACE-031 (Extension of cohort 2 from core study, A031-03)
ACE-031 (Extension of cohort 3 from core study, A031-03)
Sponsored by
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Acceleron Investigative Site
  • Acceleron Investigative Site
  • Acceleron Investigative Site
  • Acceleron Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

ACE-031 (Extension of cohort 1 from core study, A031-03)

ACE-031 (Extension of cohort 2 from core study, A031-03)

ACE-031 (Extension of cohort 3 from core study, A031-03)

Arm Description

Outcomes

Primary Outcome Measures

Number of patients with adverse events.
Change in laboratory parameters and vital signs.

Secondary Outcome Measures

Percent change in total lean body mass by DXA scan.
Percent change in total body and lumbar spine bone mineral density by DXA scan.
Percent change in muscle strength score by hand-held myometry.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).
Change in pulmonary function tests.

Full Information

First Posted
November 1, 2010
Last Updated
January 30, 2013
Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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1. Study Identification

Unique Protocol Identification Number
NCT01239758
Brief Title
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
Official Title
An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
January 2013
Overall Recruitment Status
Terminated
Why Stopped
This study was terminated based on preliminary safety data.
Study Start Date
October 2010 (undefined)
Primary Completion Date
May 2011 (Actual)
Study Completion Date
May 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.]

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ACE-031 (Extension of cohort 1 from core study, A031-03)
Arm Type
Experimental
Arm Title
ACE-031 (Extension of cohort 2 from core study, A031-03)
Arm Type
Experimental
Arm Title
ACE-031 (Extension of cohort 3 from core study, A031-03)
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
ACE-031 (Extension of cohort 1 from core study, A031-03)
Intervention Description
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Intervention Type
Biological
Intervention Name(s)
ACE-031 (Extension of cohort 2 from core study, A031-03)
Intervention Description
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
Intervention Type
Biological
Intervention Name(s)
ACE-031 (Extension of cohort 3 from core study, A031-03)
Intervention Description
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Primary Outcome Measure Information:
Title
Number of patients with adverse events.
Time Frame
From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Title
Change in laboratory parameters and vital signs.
Time Frame
Baseline to End-of-Study Visit, approximately 24 weeks later.
Secondary Outcome Measure Information:
Title
Percent change in total lean body mass by DXA scan.
Time Frame
Baseline to End-of-Study Visit, approximately 24 weeks later.
Title
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.
Title
Percent change in muscle strength score by hand-held myometry.
Time Frame
Baseline to End-of-Study Visit, approximately 24 weeks later.
Title
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Time Frame
Baseline to End-of-Study Visit, approximately 24 weeks later.
Title
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.
Title
Change in pulmonary function tests.
Time Frame
Baseline to End-of-Study Visit, approximately 24 weeks later.

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Acceleron Investigative Site
City
Calgary
State/Province
Alberta
Country
Canada
Facility Name
Acceleron Investigative Site
City
Hamilton
State/Province
Ontario
Country
Canada
Facility Name
Acceleron Investigative Site
City
London
State/Province
Ontario
Country
Canada
Facility Name
Acceleron Investigative Site
City
Ottawa
State/Province
Ontario
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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