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A phase I/IItrial of MYO‐029 in adult subjects with muscular dystrophy
Open accessAbstract
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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“However, myostatin inhibitors display important side effects when tested in humans93 or display no significant improvement of survival in cancer patients94.”
From Mechanisms of muscle atrophy and hypertrophy: implications in health and disease (2021) · cites this paper as [93]
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Cites
(1992) · PubMed · Cited by 33,716
The MOS 36-ltem Short-Form Health Survey (SF-36): I. Conceptual Framework and Item Selection
Ware, J. E., & Sherbourne, C. D. (1992) · Medical Care · doi:10.1097/00005650-199206000-00002 · Cited by 30,033
(1994) · Medical Entomology and Zoology · Cited by 4,156
Regulation of skeletal muscle mass in mice by a new TGF-p superfamily member
McPherron, A. C., Lawler, A. M., & Lee, S.-J. (1997) · Nature · doi:10.1038/387083a0 · Cited by 4,013
Double muscling in cattle due to mutations in the myostatin gene
McPherron, A. C., & Lee, S.-J. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.23.12457 · Cited by 2,039
Jaretzki, A., Barohn, R. J., Ernstoff, R. M., Kaminski, H. J., Keesey, J., Penn, A. S., & Sanders, D. B. (2000) · Neurology · doi:10.1212/wnl.55.1.16 · Cited by 1,660
Self-citationA deletion in the bovine myostatin gene causes the double–muscled phenotype in cattle
Grobet, L., Royo Martin, L. J., Poncelet, D., Pirottin, D., Brouwers, B., Riquet, J., Schoeberlein, A., Dunner, S., Ménissier, F., Massabanda, J., et al. (1997) · Nature Genetics · doi:10.1038/ng0997-71 · Cited by 1,556
Myostatin Mutation Associated with Gross Muscle Hypertrophy in a Child
Schuelke, M., Wagner, K. R., Stolz, L. E., Hübner, C., Riebel, T., Kömen, W., Braun, T., Tobin, J. F., & Lee, S.-J. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa040933 · Cited by 1,399
Self-citationClop, A., Marcq, F., Takeda, H., Pirottin, D., Tordoir, X., Bibé, B., Bouix, J., Caiment, F., Elsen, J.-M., Eychenne, F., et al. (2006) · Nature Genetics · doi:10.1038/ng1810 · Cited by 1,260
Review of MR image segmentation techniques using pattern recognition
Bezdek, J. C., Hall, L., & Clarke, L. P. (1993) · Medical Physics · doi:10.1118/1.597000 · Cited by 956
Functional improvement of dystrophic muscle by myostatin blockade
Bogdanovich, S., Krag, T. O. B., Barton, E. R., Morris, L. D., Whittemore, L.-A., Ahima, R. S., & Khurana, T. S. (2002) · Nature · doi:10.1038/nature01154 · Cited by 831
Mosher, D. S., Quignon, P., Bustamante, C. D., Sutter, N. B., Mellersh, C. S., Parker, H. G., & Ostrander, E. A. (2007) · PLoS Genetics · doi:10.1371/journal.pgen.0030079 · Cited by 826
Myasthenia gravis activities of daily living profile
Wolfe, G. I., Herbelin, L. L., Nations, S., Foster, B., Bryan, W. W., & Barohn, R. J. (1999) · Neurology · doi:10.1212/wnl.52.7.1487 · Cited by 577
Self-citationRandomized, Double-Blind Six-Month Trial of Prednisone in Duchenne's Muscular Dystrophy
Mendell, J. R., Moxley, R. T., Griggs, R. C., Brooke, M. H., Fenichel, G. M., Miller, J. P., King, W. M., Signore, L., Pandya, S., Florence, J. M., et al. (1989) · New England Journal of Medicine · doi:10.1056/nejm198906153202405 · Cited by 520
Self-citationGuidelines for treatment of autoimmune neuromuscular transmission disorders
SKEIE, G. O., Apostolski, S., Evoli, A., Gilhus, N. E., Illa, I., Harms, L., Hilton‐Jones, D., Melms, A., Verschuuren, J. J. G. M., & Horge, H. W. (2010) · European Journal of Neurology · doi:10.1111/j.1468-1331.2010.03019.x · Cited by 513
Inhibition of myostatin in adult mice increases skeletal muscle mass and strength
Whittemore, L.-A., Song, K., Li, X., Aghajanian, J. D., Davies, M. V., Girgenrath, S., Hill, J. J., Jalenak, M., Kelley, P. E., Knight, A., et al. (2003) · Biochemical and Biophysical Research Communications · doi:10.1016/s0006-291x(02)02953-4 · Cited by 512
A randomized double‐blind trial of prednisolone alone or with azathioprine in myasthenia gravis
Palace, J. A., Newsom–Davis, J., Lecky, B. R. F., & Myasthenia Gravis Study Group (1998) · Neurology · doi:10.1212/wnl.50.6.1778 · Cited by 471
Reliability Testing of the Quantitative Myasthenia Gravis Score a
Barohn, R. J., McIntire, D. D., Herbelin, L. L., Wolfe, G. I., Nations, S., & Bryan, W. W. (1998) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.1998.tb11015.x · Cited by 417
Self-citationLoss of myostatin attenuates severity of muscular dystrophy in mdx mice
Wagner, K. R., McPherron, A. C., Winik, N., & Lee, S. (2002) · Annals of Neurology · doi:10.1002/ana.10385 · Cited by 378
Self-citationBrooke, M. H., Fenichel, G. M., Griggs, R. C., Mendell, J. R., Moxley, R. T., Miller, J. P., & Province, M. A. (1983) · Muscle & Nerve · doi:10.1002/mus.880060204 · Cited by 353
Self-citationAdverse Gastrointestinal Effects of Mycophenolate Mofetil
Behrend, M. (2001) · Drug Safety · doi:10.2165/00002018-200124090-00002 · Cited by 325
Mycophenolate mofetil: A safe and promising immunosuppressant in neuromuscular diseases
Chaudhry, V., Cornblath, D. R., Griffin, J. W., O’Brien, R., & Drachman, D. B. (2001) · Neurology · doi:10.1212/wnl.56.1.94 · Cited by 258
Girgenrath, S., Song, K., & Whittemore, L.-A. (2004) · Muscle & Nerve · doi:10.1002/mus.20175 · Cited by 238
Immunosuppressive agents for myasthenia gravis
Hart, I. K., Sathasivam, S., & Sharshar, T. (2007) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd005224.pub2 · Cited by 212
Muscle regeneration in the prolonged absence of myostatin
Wagner, K. R., Liu, X., Chang, X., & Allen, R. E. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0408729102 · Cited by 193
Self-citationRandomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy
Kissel, J. T., McDermott, M. P., Mendell, J. R., King, W. M., Pandya, S. K., Griggs, R. C., Tawil, R., & the FSH-DY Group (2001) · Neurology · doi:10.1212/wnl.57.8.1434 · Cited by 190
Self-citationMycophenolate mofetil for myasthenia gravis: An open-label pilot study
Ciafaloni, E., Massey, J. M., Lipscomb, B., & Sanders, D. B. (2001) · Neurology · doi:10.1212/wnl.56.1.97 · Cited by 183
Limb-Girdle Muscular Dystrophy in the United States
Moore, S. A., Shilling, C. J., Westra, S., Wall, C., Wicklund, M., Stolle, C. A., Brown, C. A., Michele, D. E., Piccolo, F., Winder, T. L., et al. (2006) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/01.jnen.0000235854.77716.6c · Cited by 179
Self-citationCorticosteroids for myasthenia gravis
Schneider‐Gold, C., Gajdos, P., Toyka, K. V., & Hohlfeld, R. (2005) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd002828.pub2 · Cited by 174
Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study
Walter, M. C., Lochmüller, H., Reilich, P., Klopstock, T., Huber, R., Hartard, M., Hennig, M., Pongratz, D. E., & Müller‐Felber, W. (2000) · Neurology · doi:10.1212/wnl.54.9.1848 · Cited by 169
Phase I Study of Dystrophin Plasmid-Based Gene Therapy in Duchenne/Becker Muscular Dystrophy
Romero, N. B., Braun, S., Benvéniste, O., Leturcq, F., Hogrel, J., Morris, G. E., Barois, A., Eymard, B., Payan, C., Ortega, V., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1065 · Cited by 153
Mycophenolate mofetil for myasthenia gravis
Meriggioli, M. N., Ciafaloni, E., Al‐Hayk, K., Rowin, J., Lipscomb, B., Massey, J. M., & Sanders, D. B. (2003) · Neurology · doi:10.1212/01.wnl.0000094122.88929.0b · Cited by 146
Martinez Elizondo, P. (1952) · PubMed · Cited by 135
(1997) · Neurology · doi:10.1212/wnl.48.1.38 · Cited by 131
Mowzoon, N., Sussman, A. T., & Bradley, W. G. (2001) · Journal of the Neurological Sciences · doi:10.1016/s0022-510x(01)00478-6 · Cited by 124
Mycophenolate Mofetil for Myasthenia Gravis A Double‐Blind, Placebo‐Controlled Pilot Study
Meriggioli, M. N., Rowin, J., RICHMAN, J. G., & Leurgans, S. E. (2003) · Annals of the New York Academy of Sciences · doi:10.1196/annals.1254.064 · Cited by 110
Myostatin inhibition slows muscle atrophy in rodent models of amyotrophic lateral sclerosis
Holzbaur, E. L. F., Howland, D., Weber, N., Wallace, K. E., She, Y., Kwak, S., Tchistiakova, L. A., Murphy, E. K., Hinson, J., Karim, R., et al. (2006) · Neurobiology of Disease · doi:10.1016/j.nbd.2006.05.009 · Cited by 104
A Pilot Trial of Prednisone in Facioscapulohumeral Muscular Dystrophy
Tawil, R., McDermott, M., Pandya, S. K., King, W. M., Kissel, J. T., Mendell, J. R., & Griggs, R. C. (1997) · Neurology · doi:10.1212/wnl.48.1.46 · Cited by 103
Self-citationKami, K., & Senba, E. (1998) · Muscle & Nerve · doi:10.1002/(sici)1097-4598(199806)21:6 · Cited by 99
Facioscapulohumeral muscular dystrophy (FSHD)
Tawil, R., McDermott, M. P., Mendell, J. R., Kissel, J. T., Griggs, R. C., & FSH-DY Group (1994) · Neurology · doi:10.1212/wnl.44.3_part_1.442 · Cited by 98
Self-citationInflammation and response to steroid treatment in limb-girdle muscular dystrophy 2I
Darín, N., Kroksmark, A., Ahlander, A., Moslemi, A. ‐., Oldfors, A., & Tulinius, M. (2007) · European Journal of Paediatric Neurology · doi:10.1016/j.ejpn.2007.02.018 · Cited by 96
Pilot trial of albuterol in facioscapulohumeral muscular dystrophy
Kissel, J. T., McDermott, M. P., Natarajan, R., Mendell, J. R., Pandya, S., King, W. M., Griggs, R. C., Tawil, R., & The FSH-DY Group* (1998) · Neurology · doi:10.1212/wnl.50.5.1402 · Cited by 86
Self-citationPublic health impact of adverse bone effects of oral corticosteroids
van Staa, T. P., Abenhaim, L., Cooper, C. C., Zhang, B., & Leufkens, H. G. M. (2001) · British Journal of Clinical Pharmacology · doi:10.1046/j.0306-5251.2001.1385.x · Cited by 74
Hauser, R. A., Malek, A. R., & Rosen, R. (1998) · Neurology · doi:10.1212/wnl.51.3.912-a · Cited by 71
Libelius, R., & Johansson, F. (2000) · Muscle & Nerve · doi:10.1002/1097-4598(200008)23:8 · Cited by 62
Creatine monohydrate in myotonic dystrophy
Walter, M. C., Reilich, P., Lochmüller, H., Kohnen, R., Schlotter, B., Hautmann, H., Dunkl, E., Pongratz, D. E., & M�ller-Felber, W. (2002) · Journal of Neurology · doi:10.1007/s00415-002-0923-x · Cited by 61
Mycophenolate Mofetil in the Therapy of Severe Myasthenia gravis
Schneider‐Gold, C., Gold, R., Reiners, K., & Toyka, K. V. (2001) · European Neurology · doi:10.1159/000050768 · Cited by 48
Prevalence of HTLV-I-associated T-cell lymphoma
Poiesz, B. J., Papsidero, L. D., Ehrlich, G. D., Sherman, M., Dube, S., Poiesz, M. J., Dillon, K. M., Ruscetti, F. W., Slamon, D., Fang, C., et al. (2000) · American Journal of Hematology · doi:10.1002/1096-8652(200101)66:1 · Cited by 39
Meriggioli, M. N., & Rowin, J. (2003) · Journal of Clinical Neurophysiology · doi:10.1097/00004691-200309000-00011 · Cited by 36
Rózsa, C., Lovas, G., Fornádi, L., Szabó, G., & Komoly, S. (2006) · European Journal of Neurology · doi:10.1111/j.1468-1331.2006.01382.x · Cited by 28
PILOT TRIAL OF DILTIAZEM IN FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
Elsheikh, B., Bollman, E., Peruggia, M., King, W. M., Galloway, G., & Kissel, J. T. (2007) · Neurology · doi:10.1212/01.wnl.0000264017.08217.39 · Cited by 28
Self-citationEpidemiology of allergic drug reactions.
Hoigné, R., Schlumberger, H. P., VERVLOET, D., & Zoppi, M. (1993) · PubMed · Cited by 25
Unsupervised statistical segmentation of multispectral volumetric MRI images
Tamez‐Peña, J. G., Totterman, S. M. S., & Parker, K. J. (1999) · Proceedings of SPIE, the International Society for Optical Engineering/Proceedings of SPIE · doi:10.1117/12.348585 · Cited by 23
The natural course of myasthenia gravis.
Fonseca, V., & Havard, C. W. H. (1990) · BMJ · doi:10.1136/bmj.300.6737.1409 · Cited by 22
Caponnetto, C., Rossi, E. G., & Primavera, A. (2001) · European Neurology · doi:10.1159/000050759 · Cited by 19
Mycophenolate mofetil and myasthenia gravis
Ciafaloni, E. (2005) · Lupus · doi:10.1191/0961203305lu2118oa · Cited by 19
Kotani, A., Takahashi, A., Koga, H., Morita, R., Fukuyama, H., Ichinohe, T., Ishikawa, T., Hori, T., & Uchiyama, T. (2002) · European Journal Of Haematology · doi:10.1034/j.1600-0609.2002.02789.x · Cited by 14
Prakash, K. M., Ratnagopal, P., Puvanendran, K., & Lo, Y. L. (2006) · Journal of Clinical Neuroscience · doi:10.1016/j.jocn.2005.12.008 · Cited by 13
Motor unit discharge behaviors in stroke patients
Yan, K., Fang, J., & Shahani, B. T. (1998) · Muscle & Nerve · doi:10.1002/(sici)1097-4598(199811)21:11 · Cited by 12
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- Ware JE, 2000, How to score version two of the SF‐36 Health Survey
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Exercise is the real polypill.
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Metadata
- 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
- Venue
- Annals of Neurology
- Publisher
- Wiley
- Published
- 2008-05
- Volume
- 63
- Issue
- 5
- Pages
- 561-571
- ISSN
- 0364-5134 (ISSN-L)
- 1531-8249
- 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
- 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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