Clinical trials: Congenital Myopathy
Duchenne Muscular Dystrophy
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy
- Trial name
- NCT04587908
- Testing
- TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort]
- Where
- 5 sites in Japan
- Volunteers
- 104 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Quality of Life, Muscle Weakness
Effects of Progressive Resistance Training Compared With Aerobic Training in Patients With Hemophilia
This study aims to assess the effects of progressive resistance training compared with aerobic training, among those with hemophilia. Participants will be divided into two groups.
- Trial name
- NCT04892628
- Testing
- Progressive resistance training, Aerobic training
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- Dow University of Health Sciences
Arthrogryposis Multiplex Congenita (AMC)
Technical Feasibility Study of Ultrasound Muscle Imaging in Antenatal Ultrasound
The objective of the study is to evaluate the performance of muscle ultrasound sections on antenatal ultrasound between 21-24 amenorrhea weeks for the screening of muscle atrophy, in a sample of low-risk and high-risk pregnancies…
- Trial name
- FetUS (NCT06130592)
- Testing
- ultrasound muscle imaging technique
- Where
- 2 sites in France
- Volunteers
- 230 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Duchenne Muscular Dystrophy
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed…
- Trial name
- GalaxyDMD (NCT03917719)
- Testing
- Edasalonexent
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 130 people
- Sponsor
- Catabasis Pharmaceuticals
Cancer, Immune Defect
Adverse Myocardial and Vascular Side Effects of Immune Checkpoint Inhibitors
Our knowledge on cardiovascular side effects of immune checkpoint inhibitors (ICIs) is restricted to this date to observational retrospective data (mainly case series and pharamcovigilance analysis).
- Conditions
- Cancer, Immune Defect, Cardiovascular Abnormalities
- Trial name
- AMICI (NCT04586894)
- Testing
- Cardiac MRI, Smart cloth, Biobanking
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Arthrogryposis, Craniofacial Abnormalities
Study of Resting and Exercising Body Functioning in Freeman-Sheldon Syndrome and Related Conditions
The hypotheses of the present study of Freeman-Sheldon syndrome (FSS) and related conditions are: (1) that exercise capacity is lower in FSS patients versus normal controls, and the lower exercise capacity is due to changes in…
- Trial name
- FSS-EDICT I (NCT01306994)
- Testing
- Lactate, Glucose, and Adenosine Triphosphate Blood Levels, Physiological Stress Test, Functional Enquiry Form
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Freeman-Sheldon Research Group, Inc.
Gut Microbiome, Gastrointestinal Microbiome
A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
- Conditions
- Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder
- Trial name
- NCT04031469
- Testing
- There is no intervention in this study
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- ProgenaBiome
Rheumatic Diseases, Rheumatoid Arthritis (RA)
Clinical Assessment for Rheumatologic Disease - Research and Advancement in Safety and Efficacy
The CARe RAiSE project represents a pioneering translational initiative aimed at advancing precision medicine in the treatment of autoimmune rheumatic diseases.
- Conditions
- Rheumatic Diseases, Rheumatoid Arthritis (RA), Giant Cell Arteritis (GCA), Psoriatic Arthritis (PsA)
- Trial name
- CARe RAiSE (NCT07150000)
- Testing
- Ex Vivo Assay
- Where
- Germany
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Bonn
Autoimmune Diseases, Autoimmune Diseases of the Nervous System
Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
Antibody-mediated inflammatory diseases of the nervous system (also known as autoimmune diseases of the nervous system) are autoimmune diseases in which autoimmune cells and immune molecules attack the nervous system as the main…
- Conditions
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder, Myasthenia Gravis
- Trial name
- CARTinNS (NCT04561557)
- Testing
- CT103A cells, Cyclophosphamide and fludarabine
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Tongji Hospital
Nemaline Myopathy
Natural History Study for Patients With Nemaline Myopathy in the UK
The goal of this study is to to learn more about what assessments would be useful to measure for NM and what normally happens during the lives of people with NM to support future clinical trial development.
- Trial name
- NatHis-NM-MDUK (NCT06670378)
- Where
- 4 sites in United Kingdom
- Volunteers
- 45 people
- Sponsor
- University of Oxford
Cerebral Palsy, Arthrogryposis
Effects of Power Mobility on Young Children With Severe Motor Impairments
The purpose this study is to determine the effects of power mobility on the development and function of young children of young children whose severe physical disabilities limit their exploratory behaviors and may unnecessarily…
- Conditions
- Cerebral Palsy, Arthrogryposis, Spinal Muscular Atrophy, Other Central Nervous System or Musculoskeletal Disorders
- Trial name
- NCT01028833
- Testing
- Power mobility
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Oklahoma
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Muscular Dystrophy, Duchenne
Rimeporide in Patients With Duchenne Muscular Dystrophy
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime.
- Trial name
- RIM4DMD (NCT02710591)
- Testing
- Rimeporide
- Where
- 4 sites in France, Italy and 2 more
- Volunteers
- 20 people
- Sponsor
- EspeRare Foundation
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
Duchenne Muscular Dystrophy
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Duchenne Muscular Dystrophy
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD.
- Trial name
- NCT00758225
- Testing
- Idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy, Gait Disorders in Children
Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
The aim of this study was to develop a reliable and valid gait classification scale for Duchenne Muscular Dystrophy (QIGS-DMD). The items of the QIGS-DMD were designed based on the literature review considering existing…
- Trial name
- NCT05564962
- Testing
- Quality and Independence of Gait Classification Scale for Duchenne Muscular Dystrophy (QIGS-DMD)
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for…
- Trial name
- NCT01099761
- Testing
- ACE-031 0.5 mg/kg q4wk, ACE-031 1.0 mg/kg q2wk
- Where
- 5 sites in Canada
- Volunteers
- 24 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Muscular Dystrophy, Arrhythmia
Arrhythmias in Myotonic Muscular Dystrophy
Adult myotonic muscular dystrophy (Steinert's disease) is the most common inherited neuromuscular disorder. Cardiac rhythm disturbances occur frequently in this disease state and may be responsible for up to one-third of deaths.
- Conditions
- Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
- Trial name
- DM1 (NCT00622453)
- Testing
- Screening
- Where
- United States
- Volunteers
- 448 people
- Sponsor
- Indiana University
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.