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Study · Randomized controlled trial · 2017

7 results

Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.

Campbell C, McMillan HJ, Mah JK, Tarnopolsky M, Selby K, McClure T, Wilson DM, Sherman ML, Escolar D, Attie KM

Muscle & nerve23 Dec 2016Cited by 190 (Europe PMC)

PMID27462804 (opens in a new tab)DOI10.1002/mus.25268 (opens in a new tab)PepBankPUB-27462804
Publisher pagePubMedAsk about this study
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

  1. 01HumanRandomized controlled trialSafety outcome

    In boys with Duchenne muscular dystrophy, ACE‑031 was not associated with serious or severe adverse events.

    In plain wordsIn boys with Duchenne muscular dystrophy, ACE‑031 was not associated with serious or severe adverse events.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedSerious or severe adverse events
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    ACE-031 was not associated with serious or severe adverse events.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  2. 02HumanRandomized controlled trialSafety outcome

    In boys with Duchenne muscular dystrophy, potential epistaxis concerns contributed to stopping the ACE‑031 trial after the second dosing regimen.

    In plain wordsIn boys with Duchenne muscular dystrophy, possible nosebleed concerns contributed to stopping the ACE‑031 trial.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedEpistaxis safety concern
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Respiratory
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  3. 03HumanRandomized controlled trialSafety outcome

    In boys with Duchenne muscular dystrophy, potential telangiectasia concerns contributed to stopping the ACE‑031 trial.

    In plain wordsIn boys with Duchenne muscular dystrophy, possible widened small blood vessels contributed to stopping the ACE‑031 trial.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedTelangiectasia safety concern
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  4. 04HumanRandomized controlled trialEfficacy outcome

    In boys with Duchenne muscular dystrophy, ACE‑031 showed a nonsignificant trend toward maintaining walking distance versus a decline with placebo.

    In plain wordsIn boys with Duchenne muscular dystrophy, ACE‑031 tended to preserve walking distance versus placebo, but the difference was not significant.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    No significant changeSix-minute walk distance
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    A trend for maintenance of the 6-minute walk test (6MWT) distance in the ACE-031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  5. 05HumanRandomized controlled trialEfficacy outcome

    In boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward increased lean body mass.

    In plain wordsIn boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward increased lean body mass.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedLean body mass
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Muscle injury and repair · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    A trend for maintenance of the 6-minute walk test (6MWT) distance in the ACE-031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  6. 06HumanRandomized controlled trialEfficacy outcome

    In boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward increased bone mineral density.

    In plain wordsIn boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward increased bone density.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedBone mineral density
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    A trend for maintenance of the 6-minute walk test (6MWT) distance in the ACE-031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.
  7. 07HumanRandomized controlled trialEfficacy outcome

    In boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward reduced fat mass.

    In plain wordsIn boys with Duchenne muscular dystrophy, ACE‑031 showed a trend toward reduced body fat.

    Peptide
    ACE-031 (ramatercept)
    Outcome
    Direction not reportedFat mass
    Study

    n: not reported in source

    Analysis
    Secondary
    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    A trend for maintenance of the 6-minute walk test (6MWT) distance in the ACE-031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.
    1 context sentence
    Summary · context
    ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    21 Oct 2026, 15:58 UTC

    Study results updated

  2. Version 1

    21 Oct 2026, 12:50 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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