Cited by 481Muscle Physiology and DisordersNeurogenetic and Muscular Disorders ResearchInflammatory Myopathies and Dermatomyositis

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A phase I/IItrial of MYO‐029 in adult subjects with muscular dystrophy

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Wagner, Kathryn R., Fleckenstein, James L., Amato, Anthony A., Barohn, Richard J., Bushby, Katharine, Escolar, Diana M., Flanigan, Kevin M., Pestronk, Alan, Tawil, Rabi, Wolfe, Gil I., Eagle, Michelle, Florence, Julaine M., King, Wendy M., Pandya, Shree, Straub, Volker, Juneau, Paul, Meyers, Kathleen, Csimma, Cristina, Araujo, Tracey, Allen, Robert, Parsons, Stephanie A., Wozney, John M., LaVallie, Edward R., Mendell, Jerry R.·2008·Annals of Neurology

DOI 10.1002/ana.21338

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Abstract

Objective: Myostatin is an endogenous negative regulator of muscle growth and a novel target for muscle diseases. We conducted a safety trial of a neutralizing antibody to myostatin, MYO‐029, in adult muscular dystrophies (Becker muscular dystrophy, facioscapulohumeral dystrophy, and limb‐girdle muscular dystrophy). Methods: This double‐blind, placebo‐controlled, multinational, randomized study included 116 subjects divided into sequential dose‐escalation cohorts, each receiving MYO‐029 or placebo (Cohort 1 at 1mg/kg; Cohort 2 at 3mg/kg; Cohort 3 at 10mg/kg; Cohort 4 at 30mg/kg). Safety and adverse events were assessed by reported signs and symptoms, as well as by physical examinations, laboratory results, echocardiograms, electrocardiograms, and in subjects with facioscapulohumeral dystrophy, funduscopic and audiometry examinations. Biological activity of MYO‐029 was assessed through manual muscle testing, quantitative muscle testing, timed function tests, subject‐reported outcomes, magnetic resonance imaging studies, dual‐energy radiographic absorptiometry studies, and muscle biopsy. Results: MYO‐029 had good safety and tolerability with the exception of cutaneous hypersensitivity at the 10 and 30mg/kg doses. There were no improvements noted in exploratory end points of muscle strength or function, but the study was not powered to look for efficacy. Importantly, bioactivity of MYO‐029 was supported by a trend in a limited number of subjects toward increased muscle size using dual‐energy radiographic absorptiometry and muscle histology. Interpretation: This trial supports the hypothesis that systemic administration of myostatin inhibitors provides an adequate safety margin for clinical studies. Further evaluation of more potent myostatin inhibitors for stimulating muscle growth in muscular dystrophy should be considered. Ann Neurol 2008

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Authors: Wagner, K. R., Fleckenstein, J. L., Amato, A. A., Barohn, R. J., Bushby, K., Escolar, D. M., Flanigan, K. M., Pestronk, A., Tawil, R., Wolfe, G. I., Eagle, M., Florence, J. M., King, W. M., Pandya, S., Straub, V., Juneau, P., Meyers, K., Csimma, C., Araujo, T., Allen, R., Parsons, S. A., Wozney, J. M., LaVallie, E. R., & Mendell, J. R.
Affiliations: Johns Hopkins University (US)
University of Oklahoma - Tulsa (US)
Brigham and Women's Hospital (US)
University of Kansas Medical Center (US)
Newcastle University (GB)
Children's National (US)
University of Utah (US)
Washington University in St. Louis (US)
University of Rochester Medicine (US)
The University of Texas Southwestern Medical Center (US)
Abigail Wexner Research Institute (US)
Wyeth (US)
Corresponding author: Wagner, Kathryn R.
ORCID: Wagner, Kathryn R.
Amato, Anthony A.
Escolar, Diana M.
Flanigan, Kevin M.
Pestronk, Alan
Tawil, Rabi
Wolfe, Gil I.
Eagle, Michelle
Straub, Volker
Juneau, Paul
Allen, Robert
Wozney, John M.
Mendell, Jerry R.
Venue: Annals of Neurology
Publisher: Wiley
Published: 2008-05
Volume: 63
Issue: 5
Pages: 561-571
ISSN: 0364-5134 (ISSN-L)
1531-8249
DOI: 10.1002/ana.21338
OpenAlex: W2166485013
Cited by: 481
References: 59
Citations by year: 2026: 21 · 2025: 17 · 2024: 15 · 2023: 12 · 2022: 22 · 2021: 25 · 2020: 26 · 2019: 17 · 2018: 26 · 2017: 29 · 2016: 28 · 2015: 29 · 2014: 32 · 2013: 25 · 2012: 42
FWCI: 15.51
Type: journal article
Language: en
Open access: bronze
doi.org
Licence: onlinelibrary.wiley.com/termsAndConditions#vor (vor)
Locations: Annals of Neurology (publishedVersion)
PubMed (publishedVersion)
Newcastle University ePrints (Newcastle Univesity)
Funders: Medical Research Council
National Center for Research Resources (M01RR000052, M01RR023940)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Muscle Physiology and Disorders
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Health Sciences › Medicine › Rheumatology › Inflammatory Myopathies and Dermatomyositis
Keywords: myostatin inhibition, muscular dystrophy, Becker muscular dystrophy, facioscapulohumeral muscular dystrophy, limb-girdle muscular dystrophy, neutralizing antibodies, dose escalation, drug safety, skin hypersensitivity, muscle growth, muscle strength
MeSH terms: Adult, Antibodies, Drug Eruptions, Double-Blind Method, Female, Humans, Male, Muscular Dystrophies, Risk Factors, Cohort Studies, Comorbidity, Placebo Effect, Incidence, Treatment Outcome, Risk Assessment, Internationality

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A phase I/IItrial of MYO‐029 in adult subjects with muscular dystrophy

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