A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy

Annals of Neurology

Volumn 63, Issue 5, 2008, Pages 561-571

  Wagner, Kathryn R ^a   Fleckenstein, James L ^b   Amato, Anthony A ^c   Barohn, Richard J ^d   Bushby, Katharine ^e   Escolar, Diana M ^f   Flanigan, Kevin M ^g   Pestronk, Alan ^h   Tawil, Rabi ⁱ   Wolfe, Gil I ^j   Eagle, Michelle ^e   Florence, Julaine M ^h   King, Wendy M ^k   Pandya, Shree ⁱ   Straub, Volker ^e   Juneau, Paul ^l   Meyers, Kathleen ^m   Csimma, Cristina ^m,n   Araujo, Tracey ^m   Allen, Robert ^m   Parsons, Stephanie A ^m   Wozney, John M ^m   LaVallie, Edward R ^m   Mendell, Jerry R ^k   more..

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b University of Oklahoma Tulsa College of Medicine   (United States)

^c MASSACHUSETTS GENERAL HOSPITAL   (United States)

^d UNIVERSITY OF KANSAS SCHOOL OF MEDICINE   (United States)

^e NEWCASTLE UNIVERSITY   (United Kingdom)

^f CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^g UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^h WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

ⁱ UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^j UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^k OHIO STATE UNIVERSITY   (United States)

^l MMS HOLDINGS INC   (United States)

^m PFIZER INC   (United States)

ⁿ Clarus Ventures   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

MYO 029; MYOSTATIN; NEUTRALIZING ANTIBODY; PLACEBO; UNCLASSIFIED DRUG; ANTIBODY; STAMULUMAB;

ABDOMINAL PAIN; ACCIDENTAL INJURY; ADULT; AGEUSIA; ALLERGIC REACTION; ARTHRALGIA; ARTICLE; ASEPTIC MENINGITIS; AUDIOMETRY; BACKACHE; CLINICAL TRIAL; CONSTIPATION; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; COUGHING; DEPRESSION; DIARRHEA; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG ACTIVITY; DRUG SAFETY; DRUG TOLERABILITY; DYSPNEA; ELECTROCARDIOGRAPHY; FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY; FEMALE; FEVER; FLU LIKE SYNDROME; HERNIA; HISTOPATHOLOGY; HUMAN; HYPERLIPIDEMIA; HYPERSENSITIVITY; HYPOKALEMIA; INFECTION; INJECTION SITE PAIN; INSOMNIA; MALE; MIGRAINE; MUSCLE BIOPSY; MUSCLE CRAMP; MUSCLE HYPOTONIA; MUSCLE SPASM; MUSCULAR DYSTROPHY; MUSCULOSKELETAL PAIN; MYALGIA; NAUSEA; NECK PAIN; PAIN; PERIPHERAL EDEMA; PHARYNGITIS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PRURITUS; RANDOMIZED CONTROLLED TRIAL; RASH; SIDE EFFECT; SINUS CONGESTION; SINUSITIS; TACHYCARDIA; THORAX PAIN; URINE; URTICARIA; VASCULITIS; COHORT ANALYSIS; COMORBIDITY; DRUG ERUPTION; INCIDENCE; INTERNATIONAL COOPERATION; METHODOLOGY; MULTICENTER STUDY; PLACEBO EFFECT; RISK ASSESSMENT; RISK FACTOR; TREATMENT OUTCOME;

ADULT; ANTIBODIES; COHORT STUDIES; COMORBIDITY; DOUBLE-BLIND METHOD; DRUG ERUPTIONS; FEMALE; HUMANS; INCIDENCE; INTERNATIONALITY; MALE; MUSCULAR DYSTROPHIES; PLACEBO EFFECT; RISK ASSESSMENT; RISK FACTORS; TREATMENT OUTCOME;

EID: 44849140764     PISSN: 03645134     EISSN: None     Source Type: Journal    
DOI: 10.1002/ana.21338     Document Type: Article

References (34)

1. Mendell JR, Moxley RT, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989;320:1592-1599.
2. Elsheikh B, Bollman E, Peruggia M, et al. Pilot trial of diltiazem in facioscapulohumeral muscular dystrophy. Neurology 2007;68:1428-1429.
3. Kissel JT, McDermott M, Mendell JR, et al. Randomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy. Neurology 2001;57:1434-1440.
4. Tawil R, McDermott M, Pandya S, et al. A pilot trial of prednisone in facioscapulohumeral muscular dystrophy. FSH-DY Group. Neurology 1997;48:46-49.
5. Walter MC, Lochmuller H, Reilich P, et al. Creatine monohydrate in muscular dystrophies: a double-blind placebo-controlled clinical study. Neurology 2000;54:1848-1850.
6. Walter MC, Reilich P, Lochmuller H, et al. Creatine monohydrate in myotonic dystrophy: a double-blind, placebo-controlled clinical study. J Neurol 2002;249:1717-1722.
7. McPherron AC, Lawler AM, Lee SJ. Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member. Nature 1997;387:83-90.
8. McPherron AC, Lee SJ. Double muscling in cattle due to mutations in the myostatin gene. Proc Natl Acad Sci U S A 1997;94:12457-12461.
9. Grobet L, Martin LJ, Poncelet D, et al. A deletion in the bovine myostatin gene causes the double-muscled phenotype in cattle. Nat Genet 1997;17:71-74.
10. Mosher DS, Quignon P, Bustamante CD, et al. A mutation in the myostatin gene increases muscle mass and enhances racing performance in heterozygote dogs. PLoS Genet 2007;3:779-786.
11. Clop A, Marcq F, Takeda H, et al. A mutation creating a potential illegitimate microRNA target site in the myostatin gene affects muscularity in sheep. Nat Genet 2006;38:813-818.
12. Schuelke M, Wagner KR, Stolz LE, et al. Myostatin mutation associated with gross muscle hypertrophy in a child. N Engl J Med 2004;350:2682-2688.
13. Wagner KR, McPherron AC, Winik N, Lee SJ. Loss of myostatin attenuates severity of muscular dystrophy in mdx mice. Ann Neurol 2002;52:832-836.
14. Wagner KR, Liu X, Chang X, Allen RE. Muscle regeneration in the prolonged absence of myostatin. Proc Natl Acad Sci U S A 2005;102:2519-2524.
15. Bogdanovich S, Krag TO, Barton ER, et al. Functional improvement of dystrophic muscle by myostatin blockade. Nature 2002;420:418-421.
16. Girgenrath S, Song K, Whittemore LA. Loss of myostatin expression alters fiber-type distribution and expression of myosin heavy chain isoforms in slow- and fast-type skeletal muscle. Muscle Nerve 2005;31:34-40.
17. Whittemore LA, Song K, Li X, et al. Inhibition of myostatin in adult mice increases skeletal muscle mass and strength. Biochem Biophys Res Commun 2003;300:965-971.
18. Moore SA, Shilling CJ, Westra S, et al. Limb-girdle muscular dystrophy in the United States. J Neuropathol Exp Neurol 2006;65:995-1003.
19. Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983;6:91-103.
20. Korones DN, Brown MR, Palis J. "Liver function tests" are not always tests of liver function. Am J Hematol 2001;66:46-48.
21. The FSH-DY Group. A prospective, quantitative study of the natural history of facioscapulohumeral muscular dystrophy (FSHD): implications for therapeutic trials. Neurology 1997;48:38-46.
22. Tawil R, McDermott MP, Mendell J, et al. Facioscapulohumeral muscular dystrophy (FSHD): design of natural history study and results of baseline testing. FSH-DY Group. Neurology 1994;44:442-446.
23. Tamez-Pena J, Parker KJ, Totterman S. Unsupervised statistical segmentation of multispectral volumetric MR images. Proceedings of SPIE - The International Society for Optical Engineering Medical Imaging '99: Image Processing 1999;3661:300-311.
24. Bezdak JC, Hall LO, Clarke LP. Review of MR image segmentation techniques using pattern recognition. Med Phys 1993;20:1033-1048.
25. Ware JE, Kosinsk IM, Dewey JE. How to score version two of the SF-36 Health Survey. Lincoln, RI: QualityMetric, 2000.
26. Ware JE, Sherbourne CD. The MOS 36-Item Short-Form Health Survey (SF-36). Med Care 1992;30:473-483.
27. Ware JE, Kosinski M, Keller SK. SF-36 Physical and Mental Health Summary Scales: a user's manual. Boston: The Health Institute, 1994.
28. Holzbaur EL, Howland DS, Weber N, et al. Myostatin inhibition slows muscle atrophy in rodent models of amyotrophic lateral sclerosis. Neurobiol Dis 2006;23:697-707.
29. Kissel JT, McDermott MP, Natarajan R, et al. Pilot trial of albuterol in facioscapulohumeral muscular dystrophy. Neurology 1998;50:1402-1406.
30. Romero N, Braun S, Benveniste O, et al. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther 2004;15:1065-1076.
31. Angelini C, Fanin M, Menegazzo, et al. Homozygous alpha-sarcoglycan mutation in two siblings: one asymptomatic and one steroid-responsive mild limb-girdle muscular dystrophy patient. Muscle Nerve 1998;21:769-775.
32. Connolly AM, Pestronk A, Mehta S, Al-Lozi M. Primary alpha-sarcoglycan deficiency responsive to immunosuppression over three years. Muscle Nerve 1998;21:1549-1553.
33. Darin N, Krksmark A-K, Ahlander AC, et al. Inflammation and response to steroid treatment in limb-girdle muscular dystrophy 2I. Eur J Paediatr Neurol 2007;11:353-357.
34. Hoigne R, Schlumberger HP, Vervloet D, Zoppi M. Epidemiology of allergic drug reactions. Monogr Allergy 1993;31:147-170.