Clinical trials: Congenital Myopathy
Facioscapulohumeral Muscular Dystrophy
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy.
- Trial name
- ReSolve (NCT03458832)
- Testing
- FSHD-specific functional rating scale, Electrical Impedance Myography
- Where
- 14 sites in United States, France and 4 more
- Volunteers
- 324 people
- Sponsor
- University of Kansas Medical Center
Refractive Errors
Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens
The objective of this clinical investigation is to collect valid scientific safety and effectiveness data on the Acuity 200 (fluoroxyfocon A) Rigid Gas Permeable Contact Lens, manufactured from a newly developed rigid gas…
- Trial name
- Acuity 200 (NCT03971227)
- Testing
- Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
- Where
- 3 sites in United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Acuity Polymers, Inc.
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
Laminopathies, Emery Dreifuss Muscular Dystrophy 2
Modifying Factors in Striated Muscle Laminopathies
Mutations in the LMNA gene, which codes for lamins A and C, proteins of the nuclear lamina, are responsible for a wide spectrum of pathologies, including a group specifically affecting striated skeletal and cardiac muscles, with…
- Conditions
- Laminopathies, Emery Dreifuss Muscular Dystrophy 2, LMNA-Related Congenital Muscular Dystrophy, Dilated Cardiomyopathy-1A
- Trial name
- LMNAModifier (NCT05394506)
- Testing
- Skin Biopsy, Muscle biopsy
- Where
- 8 sites in France
- Volunteers
- 40 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Facioscapulohumeral Muscular Dystrophy
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies.
- Trial name
- NCT02531217
- Testing
- ATYR1940
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 9 people
- Sponsor
- aTyr Pharma, Inc.
Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI)
Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
The primary aim of this observational study is to investigate specific aspects of social cognition in dystrophinopathies. Body awareness, interpersonal distance and emotional processing will be measured in a sample of patients…
- Conditions
- Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI), Social Cognition
- Trial name
- NCT06874166
- Testing
- Electroencephalography (EEG), Social Cognition Tasks
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Cerebrovascular Accident, Post-polio Syndrome
Nomad P-KAFO Study
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that has affected their ability to walk.
- Conditions
- Cerebrovascular Accident, Post-polio Syndrome, Spinal Cord Injuries, Multiple Sclerosis
- Trial name
- NCT05644522
- Testing
- Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Shirley Ryan AbilityLab
Muscle Spasticity, Hypertonia, Muscle
Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals
To assess the safety and tolerability of single and multiple doses of MTR-601 in normal healthy volunteers under fed and fasted conditions. To evaluate the plasma and urine pharmacokinetics (PK) of MTR-601.
- Conditions
- Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis
- Trial name
- NCT06117020
- Testing
- MTR-601
- Where
- United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Motric Bio
Duchenne Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.
- Trial name
- NCT02851797
- Testing
- givinostat
- Where
- 42 sites in United States, Belgium and 9 more
- Volunteers
- 179 people
- Sponsor
- Italfarmaco
Muscular Dystrophy, Duchenne Muscular Dystrophy,
Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
- Trial name
- mdp (NCT01834066)
- Testing
- Stem Cell
- Where
- India
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1)
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and…
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- NCT06809049
- Testing
- Music and movement education
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- Hanns Lochmuller
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders
Relation Between Muscle Architecture and Functional Ability in Children With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive X-linked neuromuscular disorder characterized by muscle degeneration, pseudohypertrophy, and declining functional mobility.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders, Muscle Architecture
- Trial name
- NCT07437378
- Where
- Egypt
- Volunteers
- 26 people
- Sponsor
- Deraya University
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Nemaline Myopathy, Myopathies
Natural History Study for Patients With Nemaline Myopathy in Spain
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of nemaline myopathies.
- Conditions
- Nemaline Myopathy, Myopathies, Myopathic Conditions, NEB
- Trial name
- NCT07488806
- Testing
- Muscle Ultrasound, Motor function scales, Complete physical examination
- Where
- Spain
- Volunteers
- 100 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)
This is a long-term extension trial of RAY121 in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing…
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM)
- Trial name
- NCT06723106
- Testing
- RAY121
- Where
- 62 sites in Australia, Austria and 17 more
- Volunteers
- 144 people
- Sponsor
- Chugai Pharmaceutical
Arthrogryposis
Continuous Passive Motion Device for Children With Arthrogryposis
The Investigators are investigating a continuous passive motion device to exercise the knee of young children with arthrogryposis after they have had knee surgery.
- Trial name
- NCT06192134
- Testing
- CPM device
- Volunteers
- 5 people
- Sponsor
- Nemours Children's Clinic
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
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