Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-04-30

Study Completion Date

2011-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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. \[Note: This study was terminated based on safety data\]

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT01239758

Duchenne Muscular Dystrophy

TERMINATED PHASE2

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209

Duchenne Muscular Dystrophy

RECRUITING PHASE1/PHASE2

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

NCT02515669

Muscular Dystrophy (DMD)

TERMINATED PHASE1/PHASE2

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02927080

Facioscapulohumeral Muscular Dystrophy

TERMINATED PHASE2

Rimeporide in Patients With Duchenne Muscular Dystrophy

NCT02710591

Muscular Dystrophy, Duchenne

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Duchenne Muscular Dystrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ACE-031 0.5 mg/kg q4wk

Group Type EXPERIMENTAL

ACE-031 0.5 mg/kg q4wk

Intervention Type BIOLOGICAL

ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.

ACE-031 1.0 mg/kg q2wk

Group Type EXPERIMENTAL

ACE-031 1.0 mg/kg q2wk

Intervention Type BIOLOGICAL

ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ACE-031 0.5 mg/kg q4wk

ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.

Intervention Type BIOLOGICAL

ACE-031 1.0 mg/kg q2wk

ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.

Intervention Type BIOLOGICAL

Placebo

Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

4 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No