Clinical trials: Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT02516085
- Testing
- Metformin, L-Arginine
- Volunteers
- 5 people
- Sponsor
- University Hospital, Basel, Switzerland
Muscular Dystrophy, Duchenne
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Improved standards of care and the regular early use of glucocorticoid treatment have changed the natural history of Duchenne muscular dystrophy (DMD), affecting both survival and time of loss of functional milestones.
- Trial name
- NCT05833633
- Testing
- MUSCLE MAGNETIC RESONANCE IMAGING (MRI)
- Where
- Italy
- Volunteers
- 17 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Duchenne Muscular Dystrophy (DMD), Virtual Reality
The Effect of Virtual Reality Applications on Upper Extremity Functions in Patients With Duchenne Muscular Dystrophy
This study aims to evaluate the effects of fully immersive virtual reality (VR) applications on upper extremity (UE) functions in individuals diagnosed with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT07039799
- Testing
- Virtual Reality + Conventional Physiotherapy, Conventional Physiotherapy, Formative Usability Evaluation of the VR Rehabilitation Platform
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people
- Sponsor
- Istanbul University
Duchenne Muscular Dystrophy
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with…
- Trial name
- NCT03508947
- Testing
- WVE-210201
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy
The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy
Children with Duchenne Muscular Dystrophy (DMD) have difficulties towards the end of the ambulatory period, especially in activities that require lower extremity proximal muscle strength such as walking, climbing stairs, standing…
- Trial name
- NCT04287582
- Testing
- Electromyographic device
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Osteoporosis, Duchenne Muscular Dystrophy
Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle…
- Trial name
- NCT05281120
- Testing
- Low-level mechanical vibrations WITH vertical sinusoidal acceleration, Low-level mechanical vibrations WITHOUT vertical sinusoidal acceleration
- Volunteers
- 20 people
- Sponsor
- Istituto Auxologico Italiano
Duchenne Muscular Dystrophy
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01207908
- Testing
- IGF-1
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Duchenne Muscular Dystrophy
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported…
- Trial name
- DMD-001 SUMMIT (NCT06564974)
- Testing
- Vamorolone
- Where
- 33 sites in United States, Puerto Rico
- Volunteers
- 250 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Duchenne Muscular Dystrophy
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
- Trial name
- NCT04768062
- Testing
- Viltolarsen
- Where
- 31 sites in Australia, Canada and 15 more
- Volunteers
- 74 people
- Sponsor
- NS Pharma, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies
Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Trial name
- GRASP-01-002 (NCT05257473)
- Where
- 11 sites in United States, New Zealand and 1 more
- Volunteers
- 59 people
- Sponsor
- Virginia Commonwealth University
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy
Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy
Although there are studies showing that the effect on motor performance over time in children with DMD is associated with a decrease in the level of physical activity, no publication has been found that directly examines the…
- Trial name
- NCT05641805
- Testing
- PAQ-C (Physical activitiy questionare for children), MoCA (Montreal Cognitive Assessment), PBS (Pediatric Balance Scale), Modified Mini Mental State Examination, BMI (Body Mass Index)
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Muş Alparslan University
Duchenne Muscular Dystrophy
DMD- Interactive Virtual Reality Study
This study will help determine if an Interactive Virtual Reality system can improve the physiotherapy of young patients with Duchenne muscular dystrophy (DMD).
- Trial name
- DMD-IVR (NCT06274983)
- Testing
- IVR platform trial
- Where
- United Kingdom
- Volunteers
- 14 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Muscular Dystrophy, Duchenne, DMD
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
- Trial name
- NCT04337112
- Testing
- viltolarsen
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Ramipril Versus Carvedilol in Duchenne and Becker Patients
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and…
- Trial name
- NCT00819845
- Testing
- carvedilol, ramipril
- Where
- Italy
- Volunteers
- 194 people, healthy volunteers welcome
- Sponsor
- Catholic University, Italy
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
- Trial name
- NCT06839469
- Where
- 3 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Columbia University
Duchenne Muscular Dystrophy, Exon 44
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Trial name
- NCT05996003
- Testing
- NS-089/NCNP-02
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.
- Trial name
- NCT05249361
- Testing
- no intervention
- Where
- 2 sites in South Korea
- Volunteers
- 48 people
- Sponsor
- Samsung Medical Center
Duchenne Muscular Dystrophy
Evaluation of Home Based Assessments on Participants With DMD
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
- Trial name
- NCT05657938
- Testing
- Wearable Device, Duchenne Video Assessment (DVA)
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Solid Biosciences Inc.