Clinical Trials on Muscular Dystrophy, Facioscapulohumeral in New Zealand

Total 5206 results

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
Arrowhead Pharmaceuticals

Recruiting

Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Facio-Scapulo-Humeral Dystrophy

New Zealand
aTyr Pharma, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Netherlands, France, Italy
University of Rochester

Completed

Study of Testosterone and rHGH in FSHD (STARFISH)

Facioscapulohumeral Muscular Dystrophy

United States
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
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
University of Kansas Medical Center
National Institute of Neurological Disorders and Stroke (NINDS)

Active, not recruiting

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Facioscapulohumeral Muscular Dystrophy

United States
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
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
Acceleron Pharma Inc. (a wholly owned subsidiary...

Terminated

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral Muscular Dystrophy

United States, Spain, Canada
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
Acceleron Pharma Inc. (a wholly owned subsidiary...

Terminated

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Charcot-Marie-Tooth Disease | Facioscapulohumeral Muscular Dystrophy

United States, Spain, Canada
Arrowhead Pharmaceuticals

Recruiting

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Myotonic Dystrophy 1

New Zealand, Australia
University of Kansas Medical Center
Hoffmann-La Roche; Seattle Children's Hospital; University of Rochester; University... and other collaborators

Recruiting

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)

FSHD

United States, Canada, United Kingdom
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, New Zealand, United Kingdom
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (ACHIEVE)

Myotonic Dystrophy Type 1 (DM1)

United Kingdom, France, Netherlands, Italy, Germany, New Zealand
Virginia Commonwealth University
Radboud University Medical Center; University of California, Los Angeles; University... and other collaborators

Recruiting

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Myotonic Dystrophy 1

United States, Netherlands, United Kingdom, New Zealand, Germany, Italy, Canada, France
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Active, not recruiting

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

Duchenne Muscular Dystrophy

Korea, Republic of, Chile, Japan, China, United Kingdom, Netherlands, Spain, Norway, Russian Federation, Australia, Greece, Italy, New Zealand, Turkey, Mexico, Czechia, Canada
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
AMO Pharma Limited

Completed

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Congenital Myotonic Dystrophy

United States, Canada, New Zealand, Australia, United Kingdom
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
Benitec Biopharma, Inc.

Recruiting

A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

Oculopharyngeal Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.

Active, not recruiting

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Muscular Dystrophy, Duchenne

United States, Korea, Republic of, India, Slovenia, Turkey, United Kingdom, Germany, Switzerland, Spain, Czechia, France, Serbia, Taiwan, Netherlands, Mexico, Colombia, Denmark, New Zealand, Norway, Greece, Hungary, Ireland, Italy, Jo... and more
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Completed

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

Duchenne Muscular Dystrophy

Korea, Republic of, Chile, Taiwan, China, United States, United Kingdom, Netherlands, Spain, Australia, Norway, Russian Federation, Greece, Italy, Turkey, Mexico, Canada, Hong Kong, New Zealand, Ukraine
PepGen Inc

Recruiting

A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

Duchenne Muscular Dystrophy

Canada
Sarepta Therapeutics, Inc.

Completed

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD)

United States
Santhera Pharmaceuticals

No longer available

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United States
PTC Therapeutics

Completed

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Duchenne Muscular Dystrophy

United States
PTC Therapeutics

Completed

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.

Completed

Study of Eteplirsen in DMD Patients (PROMOVI)

Duchenne Muscular Dystrophy (DMD)

United States
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Enrolling by invitation

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION)

Duchenne Muscular Dystrophy

United States
Pfizer

Active, not recruiting

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Australia
PTC Therapeutics

Completed

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.

Completed

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)

Duchenne Muscular Dystrophy (DMD)

United States, Belgium, Canada, United Kingdom, Australia, Spain, Italy
Avidity Biosciences, Inc.

Active, not recruiting

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)

Nervous System Diseases | Genetic Diseases, Inborn | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Neurodegenerative Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Heredodegenerative Disorders, Nervous System | Myotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy... and other conditions

United States
Santhera Pharmaceuticals

Terminated

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)

Duchenne Muscular Dystrophy (DMD)

United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS); Newcastle... and other collaborators

Completed

Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)

Duchenne Muscular Dystrophy

United States, Canada, United Kingdom, Germany, Italy
Columbia University
National Eye Institute (NEI); Centre Hospitalier National d'Ophtalmologie des... and other collaborators

Recruiting

Study of BEST1 Vitelliform Macular Dystrophy

Retinitis Pigmentosa | Best Vitelliform Macular Dystrophy

United States, Germany, France
GlaxoSmithKline

Completed

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) (DMD114876)

Muscular Dystrophies

United States
GlaxoSmithKline

Completed

Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy

Muscular Dystrophies

Germany, Belgium, Turkey, Netherlands, Spain, Israel, Australia, France, United Kingdom
Columbia University
Muscular Dystrophy Association; Stevens Institute of Technology

Completed

Wearable Technology to Assess Gait Function in SMA and DMD

Duchenne Muscular Dystrophy | Spinal Muscular Atrophy Type 3

United States
PTC Therapeutics

Completed

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

United States, Germany, Spain, Sweden, Canada, Italy, Australia, Belgium, France, Israel, United Kingdom
Wave Life Sciences Ltd.

Terminated

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

Duchenne Muscular Dystrophy

United States, Belgium, United Kingdom, Czechia, Canada, France, Italy, Sweden
Hoffmann-La Roche

Completed

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Belgium, Canada, Australia, Japan, France, Netherlands, Spain, Argentina, Germany, Italy, United Kingdom, Sweden
PepGen Inc

Recruiting

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)

Myotonic Dystrophy 1

United States, Canada
PTC Therapeutics
Genzyme, a Sanofi Company

Terminated

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

United States, Belgium, Australia, Canada, France, Germany, Israel, Italy, Spain, Sweden, United Kingdom
Capricor Inc.

Recruiting

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)

Nervous System Diseases | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Muscular Diseases | Neuromuscular Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Muscular Dystrophy, Duchenne

United States
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)

Duchenne Muscular Dystrophy

Belgium, United States, Japan, Spain, Italy, United Kingdom

Clinical Trials on Muscular Dystrophy, Facioscapulohumeral in New Zealand

Sponsors and Collaborators

Medical Conditions

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CROs in Cameroon

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