Clinical trials: Muscular Dystrophy
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
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
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage…
- Trial name
- NCT02667483
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Duchenne Muscular Dystrophy
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to <18 years) patients with DMD.
- Trial name
- ULYSSES (NCT05933057)
- Testing
- Givinostat
- Where
- 29 sites in Belgium, Canada and 10 more
- Volunteers
- 138 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who…
- Trial name
- NCT03218995
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment…
- Trial name
- ENDURE (NCT06270719)
- Testing
- Delandistrogene Moxeparvovec, Standard of Care
- Where
- 29 sites in United States
- Volunteers
- 500 people
- Sponsor
- Sarepta Therapeutics, Inc.
Osteoporosis, Muscular Dystrophy
Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy
By supplying an adequate amount of calcium and vitamin D with the addition of weekly bisphosphonate, the investigators will be able to increase bone mass and decrease the incidence of fragility fractures in these children with…
- Conditions
- Osteoporosis, Muscular Dystrophy, Cystic Fibrosis
- Trial name
- NCT01882400
- Testing
- Bisphosphonate treatment
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Gilles Boire
Muscular Dystrophies
LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy
As part of the EMPATIA @ Lecco project, task 2.9 and task 4.5 respectively provide for the refinement and testing of robotic solutions on the market oriented towards patient empowerment.
- Trial name
- NCT04313049
- Testing
- JACO2, VOCAL CONTROL
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS Eugenio Medea
Duchene Muscular Dystrophy
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine…
- Trial name
- PROVIDUS (NCT07127978)
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- ITF Therapeutics LLC
Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabolic derangement during exercise.
- Conditions
- Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors, Carbohydrate Metabolism, Inborn Errors, Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT02635269
- Testing
- Sugar
- Where
- Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
Digestive Events in Duchenne Muscular Dystrophy Patients
Relation between clinical and genetic features and acute digestive events in Duchenne muscular dystrophy patients
- Trial name
- DIGD (NCT04384354)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Limb Girdle Muscular Dystrophy, Type 2I, Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy Type 2I in Norway
Key goals are to establish the natural history of limb-girdle muscular dystrophy type 2I (LGMD 2I) and identify feasible and sensitive tools and biomarkers to measure disease affection and progression, determine the Norwegian…
- Conditions
- Limb Girdle Muscular Dystrophy, Type 2I, Limb Girdle Muscular Dystrophy, Muscular Dystrophies, Limb Girdle Muscular Dystrophy R9 FKRP-related
- Trial name
- NCT03930628
- Where
- Norway
- Volunteers
- 106 people
- Sponsor
- University Hospital of North Norway
Becker Muscular Dystrophy
Sodium Nitrate to Improve Blood Flow
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02847975
- Testing
- Sodium nitrate
- Where
- 2 sites in United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task
HRV is attained using a Polar RS800CX. Then, evaluated through linear, non-linear and chaotic global techniques (CGT). Forty-five male subjects were included in the DMD group and age-matched with forty-five in the healthy Typical…
- Trial name
- NCT04607824
- Testing
- Duchenne Muscular Dystrophy group, Typical Development group
- Where
- Brazil
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Neurological Observations, Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Dystrophy in Children
This study will focus on the symptoms, natural history and clinical impact of facioscapulohumeral muscular dystrophy (FSHD) in children. Symptoms of classical FSHD start in adulthood.
- Conditions
- Neurological Observations, Facioscapulohumeral Muscular Dystrophy, Pediatric Disorder
- Trial name
- iFocus (NCT02625662)
- Where
- Netherlands
- Volunteers
- 32 people
- Sponsor
- University Medical Center Nijmegen
Muscular Dystrophy, Facioscapulohumeral
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
- Trial name
- MOVE Peds (NCT06847282)
- Where
- 7 sites in United States, Australia
- Volunteers
- 80 people
- Sponsor
- University of Kansas Medical Center
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Myotonic Dystrophy 1
Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disorder caused by an expanded CTG trinucleotide repeat in the 3' untranslated region of the DMPK gene.
- Trial name
- C-DMCOS-DM1 (NCT06101940)
- Testing
- MRI scan, Electrocardiography, Pulmonary function test
- Where
- 22 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Huashan Hospital