Completed


Study TitleStudy ChairSponsorPublicationsTotal 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. ClemensReveraGen BioPharma



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 SkippingP. ClemensThe National Institute of Health

Muscle Nerve (2020) Jun 21



83
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular DystrophiesP. ClemensThe 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. ClemensReveraGen BioPharma



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. ClemensNS 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. ClemensReveraGen BioPharma

Pharmacological Research (2018) 136:140-150



48
Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset Facioscapulohumeral Muscular DystrophyJ. MahUnited States FSHD Society

FSHD Global Research Foundation

Muscular Dystrophy Canada

aTyr Pharma



53
Engaging Duchenne CommunityR. Bendixen
L. Morgenroth
Foundation to Eradicate Duchenne (FED)

Clinical Therapeutics (2016) 6:1474-1486



59
Duchenne Muscular Dystrophy Tissue Bank for Exon SkippingA. HarperNational Institute of Health

For access to tissue bank: info@trinds.com



53
CNMC0609 Comparative Study of Clinical Endpoint: HHM vs. CQMST. DuongMuscular Dystrophy Association

Manuscript in draft



33
Evaluation of LGMDS. SparksMuscular Dystrophy Association

Manuscript in draft



52
PITT1109 Cardiac Outcome Measures in Children with Muscular DystrophyP. ClemensNational 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 DMDD. EscolarFoundation to Eradicate Duchenne (FED)

Neurology (2012) 78:904-913



64
CNMC0601 A Randomized Study of Daily vs. High Dose Weekly Prednisone in DMDD. Escolar
P. Clemens
Muscular Dystrophy Association

Neurology (2011) 77:444-452



64
CNMC0302 An Open-Label Pilot Study of Pentoxifylline in Steroid-Naïve DMDD. EscolarMuscular Dystrophy Association

Muscle and Nerve (2011) 44:170-173



17
CINRG Reliability StudiesD. 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 DMDD. EscolarMuscular Dystrophy Association

Annals of Neurology (2005) 58:151–155



50
CNMC0301 An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated DMDD. EscolarMuscular Dystrophy Association

Muscle and Nerve (2011) 44:174-178



16
KUL0401 An Open-Label Pilot Study of Oxatomide in Steroid-Naive DMDG. 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. ClemensNS 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 DystrophyR. BendixenFoundation to Eradicate Duchenne (FED)

Manuscript in draft



18
Community Based Outcome Measures in Boys with DMDR. BendixenFoundation to Eradicate Duchenne (FED)

ReveraGen BioPharma

Manuscript in draft