Completed

Study Title	Study Chair	Sponsor	Publications	Total Enrollment
A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)	P. Clemens	ReveraGen BioPharma	Neurology Sep (2019) 93(13): e1312-e1323	46
A Phase IIb Randomized, Double-blind, Parallel Placebo- and Active-controlled Study to Assess the Efficacy and Safety of Vamorolone in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD)	P. Clemens M. Guglieri	ReveraGen BioPharma	JAMA Neurol. 2022 Oct 1;79(10):1005-1014	133
Becker Muscular Dystrophy- A Natural History Study to Predict Efficacy of Exon Skipping	P. Clemens	The National Institute of Health	Muscle Nerve (2020) Jun 21	83
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies	P. Clemens	The Department of Defense	Manuscript in draft	33
A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long‑Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)	P. Clemens	ReveraGen BioPharma	Neurology Sep (2019) 93(13): e1312-e1323	48
A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)	P. Clemens	NS Pharma, Inc.	JAMA Neurol. (2020) May 26	16
A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)	P. Clemens	ReveraGen BioPharma	Pharmacological Research (2018) 136:140-150	48
Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset Facioscapulohumeral Muscular Dystrophy	J. Mah	United States FSHD Society FSHD Global Research Foundation Muscular Dystrophy Canada aTyr Pharma	Neurology Apr (2018) 90(15): e1333-e1338	53
Engaging Duchenne Community	R. Bendixen L. Morgenroth	Foundation to Eradicate Duchenne (FED)	Clinical Therapeutics (2016) 6:1474-1486	59
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping	A. Harper	National Institute of Health	For access to tissue bank: info@trinds.com	53
CNMC0609 Comparative Study of Clinical Endpoint: HHM vs. CQMS	T. Duong	Muscular Dystrophy Association	Manuscript in draft	33
Evaluation of LGMD	S. Sparks	Muscular Dystrophy Association	Manuscript in draft	52
PITT1109 Cardiac Outcome Measures in Children with Muscular Dystrophy	P. Clemens	National Institute of Health	JASE (2015) 28(8): 999-1008	48
CNMC0705 A Double-Blind Randomized Placebo-Controlled Study of Daily Pentoxifylline as Rescue Treatment in DMD	D. Escolar	Foundation to Eradicate Duchenne (FED)	Neurology (2012) 78:904-913	64
CNMC0601 A Randomized Study of Daily vs. High Dose Weekly Prednisone in DMD	D. Escolar P. Clemens	Muscular Dystrophy Association	Neurology (2011) 77:444-452	64
CNMC0302 An Open-Label Pilot Study of Pentoxifylline in Steroid-Naïve DMD	D. Escolar	Muscular Dystrophy Association	Muscle and Nerve (2011) 44:170-173	17
CINRG Reliability Studies	D. Escolar J. Mayhew		Muscle and Nerve (2001) 24:787-93 and Muscle and Nerve (2007) 35:36-42	31
CNMC0599 A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess the Efficacy and Safety of Glutamine and Creatine in DMD	D. Escolar	Muscular Dystrophy Association	Annals of Neurology (2005) 58:151–155	50
CNMC0301 An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated DMD	D. Escolar	Muscular Dystrophy Association	Muscle and Nerve (2011) 44:174-178	16
KUL0401 An Open-Label Pilot Study of Oxatomide in Steroid-Naive DMD	G. Buyse D. Escolar	Association Française Contre les Myopathie Muscular Dystrophy Association	European Journal of Pediatric Neurology (2007) 11:337 – 340	14
A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)	P. Clemens	NS Pharma, Inc.	J Neuromuscul Dis. 2022;9(4):493-501	16
Use of a Powered Arm Support Device for Upper Limb Function in Non-Ambulatory Men with Duchenne Muscular Dystrophy	R. Bendixen	Foundation to Eradicate Duchenne (FED)	Manuscript in draft	18
Community Based Outcome Measures in Boys with DMD	R. Bendixen	Foundation to Eradicate Duchenne (FED) ReveraGen BioPharma	Manuscript in draft