Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01099761
• Date of registration: 02/04/2010
• Prospective Registration: No
• Primary sponsor: Acceleron Pharma, Inc.
• Public title: Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
• Scientific 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
• Date of first enrolment: April 2010
• Target sample size: 24
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01099761
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada

Key inclusion & exclusion 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



Age minimum: 4 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Other: Placebo

Biological: ACE-031 0.5 mg/kg q4wk

Biological: ACE-031 1.0 mg/kg q2wk

Primary Outcome(s)

Number of Subjects With Adverse Reactions. [Time Frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later]

Number of Subjects With Clinical Laboratory Adverse Reactions. [Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.]

Secondary Outcome(s)

Change in Pulmonary Function Test (MEP) [Time Frame: Baseline to End-of-Stuidy Visit, approximately 24 weeks]

Change in Pulmonary Function Test (MIP) [Time Frame: Baseline to End-of-Study Visit. approximately 24 weeks]

Change in Pulmonary Function Tests (FVC) [Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.]

Percent Change in Total Lean Body Mass by DXA Scan. [Time Frame: 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.]

Percent Change in Muscle Strength Score by Hand-held Myometry. [Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.]

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.]

Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan. [Time Frame: Baseline to End-of-Study Visit, approximately 24 weeks later.]

Secondary ID(s)

A031-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/10/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01099761