Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

Phase 2TerminatedNCT01099761

Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA24 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

ACE-031 0.5 mg/kg q4wkBIOLOGICAL

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

ACE-031 1.0 mg/kg q2wkBIOLOGICAL

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

PlaceboOTHER

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

Eligibility

Sex: MALEMin age: 4 Years

Medical Language ↔ Plain English

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

Locations (5)

Acceleron Investigative Site

Calgary, Alberta, Canada

Acceleron Investigative Site

Vancouver, British Columbia, Canada

Acceleron Investigative Site

Hamilton, Ontario, Canada

Acceleron Investigative Site

London, Ontario, Canada

Outcomes

Primary Outcomes

Number of Subjects With Adverse Reactions.

Number of subjects in each cohort with a treatment-emergent adverse event considered at least possibly related to study drug

Time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later

Number of Subjects With Clinical Laboratory Adverse Reactions.

Number of subjects in each cohort with treatment-emergent adverse laboratory values judged to be at least possibly related to study drug

Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.

Secondary Outcomes

Percent Change in Total Lean Body Mass by DXA Scan.

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.

Percent Change in Muscle Strength Score by Hand-held Myometry.

Manual Muscle Testing (MMT) is a procedure to measure the function and strength of individual muscles and muscle groups. Hand-held myometry, using a device known as a dynamometer, is one method used for MMT. The dynamometer is held against the patient's limb by the examiner and the patient is asked to resist the force applied by the examiner. The dynamometer measures the force applied by the patient, providing a quantitative and objective assessment of strength of the particular muscle or muscle group. The effectiveness of a therapeutic intervention on muscle strength, as measured by hand-held myometry, can be assessed by comparing post-treatment to pre-treatment (baseline) measurements.

Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.

Change in Distance Traveled in 6 Minutes (Standardized 6-Minute-Walk Test).

Data from ClinicalTrials.gov

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