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Duchenne Muscular DystrophyPhase 2NCT01099761

Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

A Phase 2 study testing ACE-031 0.5 mg/kg q4wk and ACE-031 1.0 mg/kg q2wk in people with Duchenne Muscular Dystrophy, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA at 5 sites in Canada.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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] Read more about this study

Who is it for?
Ages 4 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
ACE-031 0.5 mg/kg q4wk and ACE-031 1.0 mg/kg q2wk
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
5 sitesCanada
Study dates
Apr 2010to Jun 2011
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

Canada5 sites

  • Acceleron Investigative Site

    Calgary, Alberta

  • Acceleron Investigative Site

    Hamilton, Ontario

  • Acceleron Investigative Site

    London, Ontario

  • Acceleron Investigative Site

    Ottawa, Ontario

  • Acceleron Investigative Site

    Vancouver, British Columbia

Other trials in Canada

About this study

Official 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

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]

Read the 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.

Being tested
ACE-031 0.5 mg/kg q4wk, ACE-031 1.0 mg/kg q2wk, Placebo
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

Study groups in NCT01099761
Study groupRoleWhat they receive
ACE-031 0.5 mg/kg q4wkExperimentalNot described
Receives: Biological: ACE-031 0.5 mg/kg q4wk
ACE-031 1.0 mg/kg q2wkExperimentalNot described
Receives: Biological: ACE-031 1.0 mg/kg q2wk
PlaceboPlacebo comparatorNot described
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

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

  2. 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

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

Secondary outcomes (8)

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

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

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

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

  3. 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.

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

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

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

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

  5. Change From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test).

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

  6. Change in Pulmonary Function Tests (FVC)

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

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

  7. Change in Pulmonary Function Test (MIP)

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

    Measured: Baseline to End-of-Study Visit. approximately 24 weeks

  8. Change in Pulmonary Function Test (MEP)

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

    Measured: Baseline to End-of-Stuidy Visit, approximately 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Apr 2010 (estimated)
  2. Study startApr 2010
  3. Primary completionJun 2011
  4. Study completionJun 2011

Why it stopped: Based on preliminary safety data.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01099761
Sponsor's protocol ID
A031-03
Status last verified
Sep 2022
First submitted
2 Apr 2010
Last updated
13 Oct 2022
Data monitoring committee
Yes
Individual data sharing
No: Abstract summarizing trial data has been published online in Muscle \& Nerve 23-Dec-2016 https://www.ncbi.nlm.nih.gov/pubmed/27462804 The Sponor currently has no plans to make IPD available for a number of reasons; (i) the study was terminated by the Sponsor prematurely based upon concerns for potential adverse drug reactions following long-term use, which were identified in chronic toxicity studies in animals, (ii) the study population was one with a rare disease, in a groups of subjects with a relatively narrow age range, across a small number of study sites. The Sponsor believes that these factors, taken together, make patient anonymity a significant challenge.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ACE-031 0.5 mg/kg q4wk and ACE-031 1.0 mg/kg q2wk in people with Duchenne Muscular Dystrophy, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA at 5 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 13 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Subjects With Adverse Reactions. (time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01099761.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2022.

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