NCT01099761

Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA · Industry
• Sex: Male
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys 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. \[Note: This study was terminated based on safety data\]

Detailed description

ACE-031, a soluble form of the human activin receptor type IIB, was administered once every 2 to 4 weeks by subcutaneous (SC) injection to boys with DMD. Dose levels and regimens for this multiple-dose study were based on data from the initial clinical studies in healthy subjects in which doses of 0.02 to 3 mg/kg SC were evaluated. A total of 24 subjects were enrolled into the study; 18 received ACE-031 and 6 placebo. All subjects were treated for a period of 12 weeks.The pharmacodynamic effects of ACE-031 treatment were assessed by a battery of motor function test that included the 6-Minute Walk Test, the 10-Minute Walk/Run Test, the 4-Stair Climb Test and the Gower Maneuver (GW). Muscle strength was assessed by hand-held myometry and fixed system testing. Body composition (i.e., spine BMD, lean mass, and fat mass) was assessed by whole body and lumbar spine DXA scans. Pulmonary function was assessed by forced vital capacity (FVC), maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP). ACE-031 safety was evaluated through observation of the incidence and severity of adverse events.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
BIOLOGICAL	ACE-031 0.5 mg/kg q4wk	ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.
BIOLOGICAL	ACE-031 1.0 mg/kg q2wk	ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.
OTHER	Placebo	Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.

Timeline

• Start date: 2010-04-01
• Primary completion: 2011-06-01
• Completion: 2011-06-01
• First posted: 2010-04-08
• Last updated: 2022-10-13
• Results posted: 2016-10-14

Locations

5 sites across 1 country: Canada