Clinical Trials on Infantile Neuroaxonal Dystrophy

Total 1456 results

Medical University of Vienna

Completed

Assessment of Corneal Graft Attachment in Patients With Fuchs Endothelial Corneal Dystrophy Following DMEK Using Ultra-high Resolution OCT

Fuchs' Endothelial Dystrophy

Austria
Centre Hospitalier Régional Metz-Thionville

Completed

Predictive Factors of Good Results After Primary Descemet's Membrane Endothelial Keratoplasty (DMEK) (GOODRMEK)

Endothelial Corneal Dystrophy
Lancashire Teaching Hospitals NHS Foundation Trust
Duchenne UK

Recruiting

Hydrotherapy in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United Kingdom
BioMarin Pharmaceutical

Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Spain, Turkey, United Kingdom, Italy
Solid Biosciences Inc.

Recruiting

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Duchenne Muscular Dystrophy

United States
Hoffmann-La Roche
Sarepta Therapeutics, Inc.

Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Duchenne Muscular Dystrophy

Spain, Germany
West China Hospital

Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

China
Hacettepe University

Not yet recruiting

Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey
Edgewise Therapeutics, Inc.

Recruiting

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)

Duchenne Muscular Dystrophy

United States
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
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Recruiting

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United States
Beni-Suef University

Not yet recruiting

Bicycle Training on Ventilatory Functions in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations

Muscular Dystrophy, Duchenne

Italy
REGENXBIO Inc.

Recruiting

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD

Duchenne Muscular Dystrophy

United States
Muş Alparlan University
Hacettepe University

Recruiting

Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey
Edgewise Therapeutics, Inc.

Recruiting

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) (LYNX)

Duchenne Muscular Dystrophy

United States
Edgewise Therapeutics, Inc.
Medpace, Inc.; SYSNAV; ImagingNMD

Recruiting

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Becker Muscular Dystrophy

United States, Spain, Netherlands, United Kingdom
ReveraGen BioPharma, Inc.
Santhera Pharmaceuticals

Recruiting

A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)

Becker Muscular Dystrophy

United States, Italy
Taiho Pharmaceutical Co., Ltd.

Recruiting

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

Duchenne Muscular Dystrophy

Japan
Genethon

Recruiting

Natural History of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

France, United Kingdom
Edgewise Therapeutics, Inc.
Medpace, Inc.

Completed

A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy (ARCH)

Becker Muscular Dystrophy

United States
FibroGen

Terminated

Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD (LELANTOS-2)

Duchenne Muscular Dystrophy

United States, Spain, Canada, Australia, China, Italy, Belgium, France, Netherlands, Austria, United Kingdom
Halic University
Istanbul University; Biruni University

Completed

Upper Extremity Muscle Strength, Balance and Functional Skills in DMD

Duchenne Muscular Dystrophy

Turkey
Pfizer

Active, not recruiting

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Australia
Sarepta Therapeutics, Inc.

Active, not recruiting

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)

Duchenne Muscular Dystrophy

United States, Korea, Republic of, Italy, United Kingdom, Canada, Poland, Spain, France, Germany, Belgium, Australia, Czechia, Serbia, Greece, Argentina, Bulgaria, Denmark, Hungary, India, Ireland, Israel, Mexico, Russian Federation, ...
Sarepta Therapeutics, Inc.
Hansa Biopharma AB

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Duchenne Muscular Dystrophy

Spain
Wave Life Sciences Ltd.

Active, not recruiting

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Duchenne Muscular Dystrophy

Jordan, United Kingdom
Hacettepe University

Completed

Dual Task in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey
Çağtay Maden

Completed

Validity and Reliability of the 6 Minute Pegboard Ring Test

Duchenne Muscular Dystrophy

Turkey
Hacettepe University

Completed

Motor Imagery in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Active, not recruiting

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

Duchenne Muscular Dystrophy

United States, Japan, Taiwan, Belgium, Spain, Germany, Hong Kong, Italy, United Kingdom
Italfarmaco
Cromsource

Enrolling by invitation

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Duchenne Muscular Dystrophy

Spain, United States, Canada, Belgium, France, Israel, Italy, Netherlands, Serbia, Germany, United Kingdom
Kevin Flanigan
Sarepta Therapeutics, Inc.

Completed

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

Duchenne Muscular Dystrophy

United States
Solid Biosciences Inc.

Active, not recruiting

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

Duchenne Muscular Dystrophy

United States
Kevin Flanigan

Active, not recruiting

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.

Terminated

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

Duchenne Muscular Dystrophy

Belgium, France, Italy, United Kingdom
Solid Biosciences Inc.

Completed

Evaluation of Home Based Assessments on Participants With DMD

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Enrolling by invitation

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)

Muscular Dystrophy, Duchenne

United States
Megan Waldrop
Audentes Therapeutics

Active, not recruiting

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

Duchenne Muscular Dystrophy

United States
University Hospital, Basel, Switzerland

Completed

Tamoxifen in Duchenne Muscular Dystrophy (TAMDMD)

Duchenne Muscular Dystrophy

France, Spain, Germany, Netherlands, Switzerland, United Kingdom
Pfizer

Active, not recruiting

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States
PTC Therapeutics

Completed

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

Duchenne Muscular Dystrophy

United States
Epirium Bio Inc.

Completed

Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

Becker Muscular Dystrophy

United States
Roxanna Marie Bendixen

Terminated

Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD

Duchenne Muscular Dystrophy

United States
Craig McDonald, MD
Cardero Therapeutics, Inc.

Completed

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

Becker Muscular Dystrophy

United States
Hacettepe University

Completed

Aerobic Exercise in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey
Mallinckrodt ARD LLC

Terminated

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE)

Muscular Dystrophy, Duchenne

Spain, Bulgaria, Italy, United States, Mexico, Israel, Serbia, Turkey
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
Pfizer

Terminated

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Canada, United Kingdom, Japan, Italy
Sarepta Therapeutics, Inc.
University College, London; Catholic University of the Sacred Heart; University... and other collaborators

Completed

Phase I/II Study of SRP-4053 in DMD Patients

Duchenne Muscular Dystrophy

United Kingdom, United States, France, Italy

Clinical Trials on Infantile Neuroaxonal Dystrophy

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in France

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Rare Diseases

Drug Interventions

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