Clinical Trials on Congenital Muscular Dystrophy

Total 57532 results

Myotonic Dystrophy Foundation

Recruiting

Myotonic Dystrophy Family Registry (MDFR)

Myotonic Dystrophy | Myotonic Dystrophy 1 | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Steinert Disease | Dystrophia Myotonica 1 | Myotonic Dystrophy 2 | Dystrophia Myotonica | Dystrophia Myotonica 2 | Myotonia Dystrophica | Myotonic Dystrophy, Congenital | Myotonic... and other conditions

United States
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Global Registry for COL6-related Dystrophies

Bethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1

United Kingdom
Cure CMD
Congenital Muscle Disease International Registr

Withdrawn

LAMA2-related Muscular Dystrophy Brain Study

LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)

United States
Institut de Myologie, France
Association Française contre les Myopathies (AFM), Paris

Not yet recruiting

Natural History Study of Children With LAMA2-related Dystrophies (LAMA2)

Merosin Deficient Congenital Muscular Dystrophy

France
Prothelia, Inc.
Cure CMD; The Bönnemann Laboratory, NINDS, National Institutes of Health; Oscar... and other collaborators

Completed

Retrospective Natural History Study of Infants and Toddlers With LAMA2-CMD (LAMA2 rNHS)

Merosin Deficient Congenital Muscular Dystrophy

United States
Institut National de la Santé Et de la Recherche...

Recruiting

Modifying Factors in Striated Muscle Laminopathies (LMNAModifier)

Laminopathies | Emery Dreifuss Muscular Dystrophy 2 | LMNA-Related Congenital Muscular Dystrophy | Dilated Cardiomyopathy-1A

France
Istituto Ortopedico Rizzoli

Completed

Low Protein Diet in Patients With Collagen VI Related Myopathies (LPD)

Bethlem Myopathy | Ullrich Congenital Muscular Dystrophy

Italy
Hope Biosciences Stem Cell Research Foundation
Hope Biosciences

No longer available

HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

Congenital Muscular Dystrophy Due to Lamin A/C Mutation

United States
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS)

Recruiting

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Muscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular Dystrophy

United States
University Hospital, Montpellier

Unknown

Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy

Duchenne Muscular Distrophy (DMD)

France
Bionano Genomics
Columbia University; University of Iowa; Medical College of Wisconsin; Augusta... and other collaborators

Recruiting

Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort

Intellectual Disability | Autism Spectrum Disorder | Fragile X Syndrome | Developmental Disability | Congenital Anomaly | Facioscapulohumeral Muscular Dystrophy 1

United States
Children's Hospital Medical Center, Cincinnati

Active, not recruiting

Pediatric Radio Frequency Coils Generic

Musculoskeletal Abnormalities | Duchenne Muscular Dystrophy

United States
Santhera Pharmaceuticals

Completed

Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO) (CALLISTO)

Congenital Muscular Dystrophy

United States
Boston Children's Hospital
Muscular Dystrophy Association

Recruiting

Molecular and Genetic Studies of Congenital Myopathies

Centronuclear Myopathy | Myotubular Myopathy | Nemaline Myopathy | Central Core Disease | Congenital Fiber Type Disproportion | Multiminicore Disease | Rigid Spine Muscular Dystrophy | Undefined Congenital Myopathy

United States
Avidity Biosciences, Inc.

Recruiting

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)

Muscular Dystrophies | Muscular Dystrophy, Facioscapulohumeral | FSHD | Facio-Scapulo-Humeral Dystrophy | FMD | Facioscapulohumeral Muscular Dystrophy 1 | FSHD2 | FSHD1 | FMD2 | Fascioscapulohumeral Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy Type 1 | Fascioscapulohumeral Muscular Dystrophy... and other conditions

United States, United Kingdom, Canada
Rigshospitalet, Denmark

Completed

Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass

Spinal Muscular Atrophy | Merosin Deficient Congenital Muscular Dystrophy

Denmark
Ohio State University
Gilead Sciences

Completed

Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1

Myotonic Dystrophy 1 | Myotonia Congenita | Paramyotonia Congenita

United States
University of Bern
University Hospital Inselspital, Berne; University of Lausanne Hospitals; University... and other collaborators

Recruiting

Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD)

Congenital Muscular Dystrophy | SMA | DMD | BMD | IMD

Switzerland
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating MYO-029 in Adult Muscular Dystrophy

Becker Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy | Limb-Girdle Muscular Dystrophy

United States
Children's Hospital Medical Center, Cincinnati
National Heart, Lung, and Blood Institute (NHLBI); Cure CMD

Completed

A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy

Congenital Muscular Dystrophy

United States
Massachusetts General Hospital
University of Pittsburgh; Boston Children's Hospital; Brigham and Women's Hospital and other collaborators

Recruiting

Biomarker Development for Muscular Dystrophies

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy

United States
Virginia Commonwealth University

Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

Congenital Myotonic Dystrophy | Childhood Myotonic Dystrophy | CDM | CHDM

United States, Italy
University of North Carolina, Chapel Hill
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

Muscular Dystrophies | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy

United States
Cure CMD

Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)

Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions

United States
Radboud University Medical Center

Completed

The Natural History of Patients With Mutations in SEPN1 (SELENON) or LAMA2

MDC1A | SELENON-related Myopathy

Netherlands
Boston Children's Hospital
National Institute of Neurological Disorders and Stroke (NINDS)

Recruiting

Molecular Analysis of Patients With Neuromuscular Disease

Limb-girdle Muscular Dystrophy | Neuromuscular; Disorder, Hereditary | Duchenne/Becker Muscular Dystrophy

United States
Rigshospitalet, Denmark

Completed

Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I

Becker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy Type 2I

Denmark
Laurent Servais
SYSNAV

Recruiting

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Duchenne Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy

Belgium
Virginia Commonwealth University
Edgewise Therapeutics, Inc.

Recruiting

Defining Endpoints in Becker Muscular Dystrophy (GRASP-01-002)

Muscular Dystrophies | Becker Muscular Dystrophy | Muscular Dystrophy in Children | Muscular Dystrophy, Becker

United States, United Kingdom
Massachusetts General Hospital

Completed

Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids

Duchenne Muscular Dystrophy | Dystrophinopathy | Becker's Muscular Dystrophy

United States
Newcastle University
Ludwig-Maximilians - University of Munich; LGMD2i Research Fund; CureLGMD2i

Recruiting

Global FKRP Registry

Limb Girdle Muscular Dystrophy | Congenital Muscular Dystrophy | LGMDR9 | LGMD2I | Walker-Warburg Syndrome | Muscle-Eye-Brain Disease | FKRP Gene Mutation

United Kingdom
Children's Hospital Medical Center, Cincinnati

Completed

Flu Vaccine Study in Neuromuscular Patients 2011

Duchenne Muscular Dystrophy | Spinal Muscular Atrophy | Congenital Muscular Dystrophy
University Hospital of North Norway
University of Tromso; Norwegian Muscle Disease Association (FFM); Norwegian National... and other collaborators

Active, not recruiting

Limb-Girdle Muscular Dystrophy Type 2I in Norway

Muscular Dystrophies | Limb Girdle Muscular Dystrophy | Limb Girdle Muscular Dystrophy, Type 2I | Limb Girdle Muscular Dystrophy R9 FKRP-related

Norway
Cooperative International Neuromuscular Research...
United States Department of Defense

Completed

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy

United States, Japan, Canada
Cooperative International Neuromuscular Research...
National Institutes of Health (NIH)

Completed

Cardiac Outcome Measures in Children With Muscular Dystrophy

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy

United States
AMO Pharma Limited

Recruiting

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)

Congenital Myotonic Dystrophy

United States, Canada, Australia, New Zealand
Virginia Commonwealth University
University of Western Ontario, Canada, Children's Health Research Institute; Fondazione Serena Onlus - Centro Clinico NeMO Milano

Recruiting

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy (TREAT-CDM)

Congenital Myotonic Dystrophy

United States, Italy, Canada
AMO Pharma Limited

Completed

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Congenital Myotonic Dystrophy

United States, Canada, New Zealand, Australia, United Kingdom
Cooperative International Neuromuscular Research...
National Institutes of Health (NIH)

Completed

Cardiac Magnetic Resonance in Children With Muscular Dystrophy

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy

United States
Centre Hospitalier Universitaire de Nice

Recruiting

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 (REINFORCE)

Facioscapulohumeral Muscular Dystrophy 1

France, Canada
Hoffmann-La Roche

Recruiting

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy (MANOEUVRE)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Denmark, United Kingdom, Italy
Fulcrum Therapeutics

Active, not recruiting

Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Spain, Italy, Denmark, Canada, Germany, France, Netherlands, United Kingdom
Fulcrum Therapeutics

Active, not recruiting

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Canada, France, Spain
Fulcrum Therapeutics

Completed

Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) (FSHD)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Canada, France, Spain
Northwestern University

Completed

Weekly Steroids in Muscular Dystrophy (WSiMD)

Becker Muscular Dystrophy | Limb-girdle Muscular Dystrophy

United States
Avidity Biosciences, Inc.

Completed

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA)

Myotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease

United States
Politecnico di Milano
IRCCS Eugenio Medea; Villa Beretta Rehabilitation Center

Completed

User-centred Assistive System for Arm Functions in Neuromuscular Subjects (USEFUL)

Muscular Dystrophies | Muscular Dystrophy, Duchenne | Muscular Dystrophy, Becker | Muscular Dystrophy, Limb-Girdle Type 2

Italy
Cooperative International Neuromuscular Research...
Carolinas Medical Center lead study site

Completed

Evaluation of Limb-Girdle Muscular Dystrophy

Becker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)

United States
Grete Andersen, MD

Completed

Lamotrigine as Treatment of Myotonia

Hyperkalemic Periodic Paralysis | Myotonia Congenita | Paramyotonia Congenita | Dystrophia Myotonica Type 1 | Potassium-Aggravated Myotonia

Denmark
aTyr Pharma, Inc.

Completed

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, France, Italy

Clinical Trials on Congenital Muscular Dystrophy

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