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Trial · Interventional

TerminatedPhase 2Results postedRegistry data

Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

NCTNCT01099761 (opens ClinicalTrials.gov in a new tab)Other idA031-03
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Terminated
Phase
Phase 2
Enrollment
24 participants (actual)
Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA · Industry
Design
Randomized · Quadruple masking
Countries
1 country

Canada

Start
Apr 2010
Primary completion
Jun 2011
Completion
Jun 2011
First posted
8 Apr 2010 (estimated)
Last update posted
13 Oct 2022
Conditions
Duchenne Muscular Dystrophy

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201020112012
  1. Ph 2

    Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

    NCT01099761

    Apr 2010 to Jun 2011

    Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

  • ACE-031 0.5 mg/kg q4wkBiological
  • ACE-031 1.0 mg/kg q2wkBiological
  • PlaceboOther

Arms3

  • Experimental

    ACE-031 0.5 mg/kg q4wk

  • Experimental

    ACE-031 1.0 mg/kg q2wk

  • Placebo comparator

    Placebo

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Number of Subjects With Adverse Reactions.

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

    Registry description

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

  2. Number of Subjects With Clinical Laboratory Adverse Reactions.

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

    Registry description

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

Secondary outcomes(8)

  1. Percent Change in Total Lean Body Mass by DXA Scan.

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

  2. Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.

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

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

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

    Registry description

    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.

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

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

    Registry description

    Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test); stratified by baseline age (\<10 years vs. \>=10 years)

  5. 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.

  6. Change in Pulmonary Function Tests (FVC)

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

    Registry description

    Forced Vital Capacity (FVC); 1 of 3 separate tests employed to assess pulmonary function in this study

  7. Change in Pulmonary Function Test (MIP)

    Time frame · Baseline to End-of-Study Visit. approximately 24 weeks

    Registry description

    Maximum Inspiratory Pressure (MIP); 2 of 3 separate tests employed to assess pulmonary function in this study

  8. Change in Pulmonary Function Test (MEP)

    Time frame · Baseline to End-of-Stuidy Visit, approximately 24 weeks

    Registry description

    Maximum Expiratory Pressure (MEP); 3 of 3 separate tests employed to assess pulmonary function in this study

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· From treatment initiation to End-of-Study Visit, approximately 24 weeks later

Number of Subjects With Adverse Reactions.

GroupValue (Number of subjects)
ACE-031 0.5 mg/kg q4wk1
ACE-031 1.0 mg/kg q2wk6
Placebo0
Primary outcomeNumber· Baseline to End-of-Study Visit, approximately 24 weeks later.

Number of Subjects With Clinical Laboratory Adverse Reactions.

GroupValue (Number of subjects)
ACE-031 0.5 mg/kg q4wk0
ACE-031 1.0 mg/kg q2wk0
Placebo0
Secondary and other outcome results(6)
Secondary outcomeMean· Baseline to End-of-Study Visit, approximately 24 weeks later.

Percent Change in Total Lean Body Mass by DXA Scan.

GroupValue (percentage change in lean body mass)Spread
ACE-031 0.5 mg/kg q4wk3.61.3
ACE-031 1.0 mg/kg q2wk4.11.5
Placebo2.61.8
Secondary outcomeMean· Baseline to End-of-Study Visit, approximately 24 weeks later.

Percent Change in Lumbar Spine Bone Mineral Density by DXA Scan.

GroupValue (percentage change from baseline)Spread
ACE-031 0.5 mg/kg q4wk1.61.8
ACE-031 1.0 mg/kg q2wk4.41.5
Placebo0.34.4
Secondary outcomeMean· Baseline to End-of-Study Visit, approximately 24 weeks later.

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

GroupValue (percentage change from baseline)Spread
ACE-031 0.5 mg/kg q4wk0.33.2
ACE-031 1.0 mg/kg q2wk0.12.3
Placebo-0.12.1
Secondary outcomeMean· Baseline to End-of-Study Visit, approximately 24 weeks later.

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

GroupValue (change (m) in 6MWT from baseline)Spread
ACE-031 0.5 mg/kg q4wk43.861.5
ACE-031 1.0 mg/kg q2wk2.535.6
Placebo5.223.6
Secondary outcomeMean· 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).

GroupValue (Change (sec) in 10MWT from baseline)Spread
ACE-031 0.5 mg/kg q4wk0.70.9
ACE-031 1.0 mg/kg q2wk0.70.9
Placebo1.00.8
Secondary outcomeMean· Baseline to End-of-Study Visit, approximately 24 weeks later.

Change in Pulmonary Function Tests (FVC)

GroupValue (Liters)Spread
ACE-031 0.5 mg/kg q4wk0.10.2
ACE-031 1.0 mg/kg q2wk0.10.1
Placebo0.10.2

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
ACE-031 0.5 mg/kg q4wkACE-031 0.5 mg/kg q4wk: ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.–0 / 93 / 9
ACE-031 1.0 mg/kg q2wkACE-031 1.0 mg/kg q2wk: ACE-031 1.0 mg/kg subcutaneously once every 2 weeks for 12 weeks.–0 / 96 / 9
ACE-031 ACE-03 2.5 mg/kg q4wkACE-031 2.5 mg/kg q4wk: ACE-031 2.5 mg/kg subcutaneously once every 4 weeks for 12 weeks.–0 / 00 / 0
PlaceboPlacebo: Matching volume placebo subcutaneously every 2 or 4 weeks for 12 weeks.–0 / 63 / 6

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
Male
Age
from 4 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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