Clinical trials: Congenital Myopathy
Duchenne Muscular Dystrophy, Gamification
Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program.
- Conditions
- Duchenne Muscular Dystrophy, Gamification, Occupational Therapy
- Trial name
- DMD (NCT06402942)
- Testing
- Gamified occupational therapy programme, Classic occupational therapy programme
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Başak Çağla Arslan
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Facioscapulohumeral
Neurological and Psychiatric Comorbidities Patients With FSHD 1 and 2
The investigators propose to conduct a comparative pilot cognitive and psychiatric profiles of 10 patients Facio-Scapulo-Humeral Dystrophy (= FHSD) type 1 and 10 patients with type 2 FSHD study.
- Trial name
- NCT02032979
- Testing
- Psychiatric test
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Urinary Titin Biomarker in DMD
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful…
- Trial name
- NCT07332013
- Testing
- Descending stair walk
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Duchenne Muscular Dystrophy
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have…
- Trial name
- NCT03985878
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT00428935
- Testing
- rAAV2.5-CMV-minidystrophin (d3990)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nationwide Children's Hospital
MELAS Syndrome, Mitochondrial Diseases
Ketones & Mitochondrial Heteroplasmy
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known mutation in their mtDNA at position 3243 (A>G).
- Trial name
- NCT01252979
- Testing
- Medium-Chain Triglycerides
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- The University of Texas Health Science Center, Houston
COVID-19, Disabilities Multiple
COVID-19 Pandemic and Parents of Disabled Children
Pandemic period could affect the disabled children's rehabilitation and follow-up negatively because of preventive measures and this could create adverse results on their parents.
- Conditions
- COVID-19, Disabilities Multiple, Cerebral Palsy, Muscular Dystrophies
- Trial name
- NCT04525742
- Testing
- Difficulties lived by disabled children's parents in the period of COVID-19 pandemic
- Where
- Turkey (Türkiye)
- Volunteers
- 309 people, healthy volunteers welcome
- Sponsor
- Gaziosmanpasa Research and Education Hospital
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
The primary objective of this study is to evaluate the safety of SRP-9004.
- Trial name
- DISCOVERY (NCT06747273)
- Testing
- SRP-9004
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to muscle weakness, breathing difficulties, heart disease, and early death.
- Trial name
- NCT07642635
- Testing
- Semaglutide (Rybelsus®)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University Medical Center
Muscular Dystrophies, Limb-Girdle Muscular Dystrophy Type 2B
Dysferlinopathy Protein in Peripheral Blood Monocytes.
The objective of the study is to answer the following important questions. Deficiency of the dysferlin protein is the cause of a very rare limb-girdle muscular dystrophy (LGMD-2B) that leads to significant disability.
- Conditions
- Muscular Dystrophies, Limb-Girdle Muscular Dystrophy Type 2B, Miyoshi Myopathy, Distal Myopathy With Anterior Tibial Onset
- Trial name
- NCT06507215
- Testing
- Protein analysis
- Where
- Spain
- Volunteers
- 149 people, healthy volunteers welcome
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Post-Acute COVID-19 Syndrome, ME/CFS
Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research
The unhide® Project is a non-interventional, longitudinal research study designed to establish a secure data repository of demographic, health, and lifestyle information from individuals with brain inflammation and related…
- Conditions
- Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA)
- Trial name
- NCT04806620
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Brain Inflammation Collaborative
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
MELAS Syndrome
Nitric Oxide Production in MELAS Syndrome
Introduction Baylor College of Medicine and Texas Children's Hospital are recruiting individuals with MELAS syndrome for a clinical study.
- Trial name
- NCT01339494
- Testing
- Arginine and citrulline supplementations
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Duchenne Muscular Dystrophy
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is…
- Trial name
- NCT06295718
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Sahra Şirvan
Limb Girdle Muscular Dystrophy
Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts
Limb girdle muscular dystrophies were originally defined as a postnatal progressive muscle disease, which begins and primarily affects the pelvic and scapular muscles.
- Trial name
- CALNATHIS (NCT06390566)
- Testing
- calpaïnopathies
- Where
- France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Cardiomyopathy in DMD: Lisinopril vs. Losartan
This trial is a double-blind randomized clinical trial of lisinopril versus losartan for the treatment of cardiomyopathy in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01982695
- Testing
- Losartan, Lisinopril
- Where
- 6 sites in United States
- Volunteers
- 23 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy (DMD)
Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients?
The intended investigation is a pilot study to evaluate the safety and efficacy of a novel transcutaneous auricular vagus nerve stimulator system, termed TRAVAGUS ONE, to reduce systemic levels of inflammatory mediators in…
- Trial name
- Travagus One (NCT07543302)
- Testing
- Transcutaneous auricular vagus nerve stimulation
- Where
- Sweden
- Volunteers
- 20 people
- Sponsor
- taVNS AB
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose…
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM)
- Trial name
- NCT06371417
- Testing
- RAY121
- Where
- 77 sites in United States, Australia and 18 more
- Volunteers
- 144 people
- Sponsor
- Chugai Pharmaceutical
Arthritis, Rheumatoid, Activities of Daily Living
Adaptation of EDAQ Into Turkish, Examining The Reliability and Validity For People With RA
The aim of the study is to adapt the Evaluation of Daily Activity Questionnaire into Turkish language, and to examine the reliability and validity for people with rheumatoid arthritis.
- Trial name
- NCT05244824
- Testing
- Adaptation into Turkish, Examining The Reliability and Validity for People with RA
- Where
- Turkey (Türkiye)
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.