Clinical trials: DMD
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy (DMD)
DMD and Gamified Physiotherapy
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular disease that limits children's physical function, mobility, and participation in daily life.
- Trial name
- Gamified DMD (NCT07368400)
- Testing
- Gamified Mobile Physiotherapy Exercise Application, Standard Home Exercise Instructions
- Volunteers
- 46 people
- Sponsor
- Başak Çağla Arslan
Duchenne Muscular Dystrophy
Aerobic Exercise in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births.
- Trial name
- NCT04173234
- Testing
- Aerobic Training, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Hacettepe University
Muscular Dystrophy, Duchenne
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in…
- Trial name
- CPI (NCT02195999)
- Testing
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
- Trial name
- NCT04060199
- Testing
- Viltolarsen
- Where
- 40 sites in United States, Australia and 17 more
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
The task consists in reach as much bubbles as they can, the bubbles appear on the screen of the computer and should be reached in 10 seconds.
- Trial name
- NCT02891434
- Testing
- Acquisition on TouchScreen, Acquisition on Kinect, Acquisition on LeapMotion
- Where
- Brazil
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy
Tamoxifen in Duchenne Muscular Dystrophy
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of…
- Trial name
- TAMDMD (NCT03354039)
- Testing
- Tamoxifen
- Where
- 11 sites in France, Germany and 4 more
- Volunteers
- 93 people
- Sponsor
- University Hospital, Basel, Switzerland
Becker's Muscular Dystrophy (BMD)
L-citrulline and Metformin in Becker's Muscular Dystrophy
The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02018731
- Testing
- Metformin and Metformin & L-Citrulline, L-Citrulline and Metformin & L-Citrulline
- Where
- Switzerland
- Volunteers
- 20 people
- Sponsor
- University Hospital, Basel, Switzerland
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)
This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in…
- Trial name
- NCT06114056
- Testing
- JWK007 Single intravenous infusion administration
- Where
- China
- Volunteers
- 3 people
- Sponsor
- West China Hospital
Duchenne Muscular Dystrophy (DMD)
E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
Duchenne muscular dystrophy (DMD) is the most common, progressive, irreversible muscular dystrophy. The pulmonary function is crucial for the duration of life in this disease.
- Trial name
- E-PULMoDMD (NCT05516745)
- Testing
- AioCare spirometer, telerehabiliation of the respiratory system
- Where
- Poland
- Volunteers
- 200 people
- Sponsor
- Medical University of Gdansk
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Muscular Dystrophy, Duchenne
Pentoxifylline in Duchenne Muscular Dystrophy
In this study, the primary aim will be to estimate the magnitude and variability of strength change over time that may be expected for subjects on the study treatment.
- Trial name
- NCT00102453
- Testing
- Pentoxifylline
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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.
Duchenne Muscular Dystrophy
Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), the most common muscular dystrophy, leads to skeletal and cardiac muscle damage. Treatment of pulmonary complications has improved survival; however, heart muscle disease or cardiomyopathy has…
- Trial name
- E-SCAR DMD (NCT01521546)
- Testing
- eplerenone
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- Subha Raman
Duchenne Muscular Dystrophy, Muscle Strength
Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
Investigator researched that the effect of trunk and lower extremity muscle strength on hamstring flexibility in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Muscle Strength, Lower Extremity, Hamstring Contractures
- Trial name
- NCT03589573
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe 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
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
This research study wants to learn more about Duchenne Muscular Dystrophy (DMD) and exercise. Today it is unknown how exercising impacts boys with DMD.
- Trial name
- NCT03319030
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock