Collaborating authors are listed in Appendix 1. Other members of the PTC124-GD-007-DMD Study Group are listed in Appendix 2 in the Supporting Information.
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
Version of Record online: 17 JUL 2013
Copyright © 2013 Wiley Periodicals, Inc.
Muscle & Nerve
Volume 48, Issue 3, pages 357–368, September 2013
How to Cite
McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J., Eagle, M., Gappmaier, E., Glanzman, A. M., PTC124-GD-007-DMD Study Group, Spiegel, R., Barth, J., Elfring, G., Reha, A. and Peltz, S. W. (2013), The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve, 48: 357–368. doi: 10.1002/mus.23905
The study was sponsored by PTC Therapeutics, Inc. (South Plainfield, New Jersey, USA), which was responsible for all aspects of the study, and by a grant from the FDA Office of Orphan Products.
Disclosures: C.M.M is a member of the Cooperative Neuromuscular Research Group (CINRG) Executive Committee and has served on advisory committees for PTC Therapeutics, Inc., Sarepta Therapeutics, Inc., GlaxoSmithKline, Prosensa, Halo Therapeutics, Shire HGT, Cardero Therapeutics, and Novartis AG. E.K.H. is a member of the CINRG Executive Committee and has served as a consultant for Genzyme Corporation and PTC Therapeutics, Inc. R.T.A. has served as a consultant for PTC Therapeutics, Inc., and Sarepta Therapeutics. J.M.F. serves on a scientific advisory board for Prosensa, on the editorial board of Neuromuscular Disorders, and serves/has served as a member of the CINRG Executive Committee and as a consultant for Prosensa, GlaxoSmithKline, Genzyme Corporation, PTC Therapeutics, Inc., and Acceleron Pharma. M.E. has served as a clinical evaluator trainer for PTC Therapeutics, and as a consultant for Prosensa and GlaxoSmithKline. E.G. has served as a clinical evaluator trainer for PTC Therapeutics. A.M.G. has served as a clinical evaluator trainer for PTC Therapeutics. R.S. is the Chief Medical Officer for PTC Therapeutics. J.B. is the Vice President for Clinical Development at PTC Therapeutics. G.E. is the Executive Director of Biostatistics at PTC Therapeutics. A.R. is the Director of Clinical Development at PTC Therapeutics. S.W.P. is the Chief Executive Officer and founding scientist, PTC Therapeutics, Inc.
- Issue online: 27 AUG 2013
- Version of Record online: 17 JUL 2013
- Accepted manuscript online: 14 MAY 2013 10:04AM EST
- Manuscript Accepted: 7 MAY 2013
- 6-minute walk test;
- Duchenne muscular dystrophy;
- energy expenditure index;
- muscular dystrophy;
- natural history;
- timed function test
Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) and other endpoints. Methods: Screening and baseline evaluations included the 6-minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate–determined energy expenditure index, and other exploratory endpoints. Results: The 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods. Conclusions: The ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression. Muscle Nerve 48: 357–368, 2013