Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial

Publication date: Available online 8 May 2019Source: Molecular Genetics and MetabolismAuthor(s): Harrison N. Jones, Maragatha Kuchibhatla, Kelly D. Crisp, Lisa D. Hobson Webb, Laura Case, Milisa T. Batten, Jill A. Marcus, Richard M. Kravitz, Priya S. KishnaniAbstractIntroductionMorbidity and mortality in adults with late-onset Pompe disease (LOPD) results primarily from persistent progressive respiratory muscle weakness despite treatment with enzyme replacement therapy (ERT). To address this need, we have developed a 12-week respiratory muscle training (RMT) program that provides calibrated, individualized, and progressive pressure-threshold resistance against inspiration and expiration. Our previous results suggest that our RMT regimen is safe, well-tolerated, and results in large increases in respiratory muscle strength. We now conduct an exploratory double-blind, randomized control trial (RCT) to determine: 1) utility and feasibility of sham-RMT as a control condition, 2) the clinically meaningful outcome measures for inclusion in a future efficacy trial. This manuscript provides comprehensive information regarding the design and methods used in our trial and will aid in the reporting and interpretation of our future findings.MethodsTwenty-eight adults with LOPD will be randomized (1,1) in blocks of 4 to RMT (treatment) or sham-RMT (control). Assessments will be conducted at pretest, posttest, 3-months detraining, and 6-months detraining. The primary outcome is maximum ins...
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research