Clinical trials: Congenital Myopathy
Dysferlinopathy, LGMD2B Now Classified as LGMDR2
Clinical Outcome Study for Dysferlinopathy
The "Clinical Outcome Study for Dysferlinopathy" is being performed in centres in Europe (UK- Newcastle; Spain- Barcelona, Sevilla; San Sebastian;Denmark, Copenhagen, Italy- Padova; France- Paris,), USA (Charlotte, NC; Columbus…
- Conditions
- Dysferlinopathy, LGMD2B Now Classified as LGMDR2, Miyoshi Myopathy
- Trial name
- Jain COS (NCT01676077)
- Where
- 16 sites in United States, Chile and 7 more
- Volunteers
- 200 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Duchenne Muscular Dystrophy
Dual Task in Duchenne Muscular Dystrophy
This study was planned to determine the effects of the dual-task performance of children with DMD with motor dysfunction and varying degrees of cognitive impairment compared to their healthy peers, to compare the dual-task…
- Trial name
- NCT05575648
- Testing
- 10 meter walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy (DMD)
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
- Trial name
- SIDEROS (NCT02814019)
- Testing
- Idebenone 150 mg film-coated tablets
- Where
- 63 sites in United States, Austria and 13 more
- Volunteers
- 255 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne and Becker Muscular Dystrophy, Polycytemia Vera
Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval
The study will evaluate the effect of a therapeutic dose and a supratherapeutic dose of ITF2357 on the QT/QTc interval.
- Trial name
- NCT04821063
- Testing
- ITF2357 10 mg/mL, Moxifloxacin Hydrochloride
- Where
- Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Italfarmaco
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
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
Myotonic Dystrophy Type 1 (DM1)
Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is one of the most common neuromuscular diseases in adults. As respiratory dysfunction is the most common cause of death in patients with DM1, a respiratory disease progression must be monitored…
- Trial name
- NCT04835298
- Testing
- Dyspnea
- Where
- France
- Volunteers
- 34 people
- Sponsor
- CHU de Reims
Psychoeducation, Parents
Psychoeducation Program for Parents of Children with Duchenne Muscular Dystrophy
Having and caring for a child with disabilities brings emotional, social and economic difficulties for many families. Families may experience many physiological and psychological problems due to the stress and anxiety they…
- Conditions
- Psychoeducation, Parents, Psychological Wellness, Duchenne Muscular Dystrophy
- Trial name
- NCT06412328
- Testing
- Psychoeducation
- Where
- Turkey (Türkiye)
- Volunteers
- 26 people
- Sponsor
- Lokman Hekim University
Mitochondrial Diseases, Mitochondrial Myopathies
Nicotinamide Riboside and Mitochondrial Biogenesis
Mitochondria are important parts of the cell that are responsible for producing energy. The amount of energy they produce depends on how much energy the body needs to function and this energy production can be severely impaired…
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Progressive External Ophthalmoplegia, Progressive Ophthalmoplegia
- Trial name
- NCT03432871
- Testing
- Nicotinamide Riboside
- Where
- United Kingdom
- Volunteers
- 13 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Myopathy
Maintenance of Muscle Mass in Older People: the Negative Impact of Statin Therapy
A major contributor to frailty and immobility in the elderly is the age related loss of muscle mass (sarcopenia). Cardiovascular disease (CVD) is the leading cause of mortality in the elderly, with high blood cholesterol and…
- Trial name
- NCT01047163
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Duchenne Muscular Dystrophy (DMD)
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD) using computer vision technology.
- Trial name
- NCT06755138
- Testing
- Observational Assessment
- Where
- South Korea
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Seoul National University Hospital
Mitochondrial Disease, Metabolic Myopathy
GDF-15 as a Biomarker for Mitochondrial Disease
Mitochondrial disorders are a group of inherited disorders causing malfunctional mitochondria. Mitochondria are found in every cell of the body, and the disorders therefore give symptoms from every tissue, especially those with…
- Conditions
- Mitochondrial Disease, Metabolic Myopathy, Muscular Dystrophy
- Trial name
- GDF-15 (NCT02745938)
- Where
- Denmark
- Volunteers
- 97 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Muscular Dystrophies
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114876 (NCT01462292)
- Testing
- GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week
- Where
- 14 sites in United States
- Volunteers
- 51 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy, Duchenne Disease
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children
- Trial name
- NCT06290713
- Testing
- Tadalafil, Exercise Training
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
Myotonic Dystrophy 1
Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
The goal of this observational study is to assess movement in individuals with Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2) using digital biomarker tools.
- Trial name
- NCT06089018
- Where
- United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Stanford University
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