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Rare Diseases

Explore clinical trials studying Rare Diseases. Study availability and eligibility vary by location and protocol.

473 public trials 86 recruiting studies

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Rare Diseases trial results

Recruiting

Intact Cord Resuscitation in CDH

NCT04429750

Conditions: Congenital Abnormalities, Congenital Diaphragmatic Hernia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations: Chu Amiens Picardie Amiens, France

Hop Jeanne de Flandre Chu Lille Lille, France

Chu de Nantes - Nantes, France

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Recruiting

Transcriptomic Analysis of Fibroblasts and Blood in Patients With Rare Diseases

NCT07075107

Conditions: Disease Attributes, Intellectual Disability, Mental Disorders, +9 more
Locations: Assistance publique - hôpitaux de Marseille Marseille, Provence-Alpes-Côt-d'Azue, France

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Recruiting

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

NCT06826612

Conditions: Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +19 more
Locations: Beth Israel Deaconess Medical Center Boston, Massachusetts, United States

University of Cincinnati/Cincinnati Children's Hospital Cincinnati, Ohio, United States

Ohio State University Columbus, Ohio, United States

University of Pennsylvania Philadelphia, Pennsylvania, United States

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Recruiting

Direct to Patient Minimal Risk Biospecimen and Data Collection Research

NCT07128966

Conditions: Bronchial Diseases, Bronchiolitis, Bronchiolitis Obliterans, +31 more
Locations: Joined Bio Lexington, Massachusetts, United States

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Recruiting

Diagnostic Value of Exome/ Genome Sequencing, Conventional Methods in Rare Diseases and Familial Tumor Syndromes

NCT04731857

Conditions: Disease Attributes, Disease Susceptibility, Genetic Predisposition, +5 more
Locations: University Hospital Tübingen Tübingen, Germany

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Recruiting

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: UCSF Benioff Children's Hospital Oakland Oakland, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) Boston, Massachusetts, United States

Weill Cornell Medicine; Division of Pediatric Neurology New York, United States

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Recruiting

Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability

NCT07527624

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +1 more
Locations: Imagine Clinical Research Paris, Île-de-France Region, France

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Recruiting

BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe

NCT06856902

Conditions: Behavior, Cardiovascular Diseases, Disease Attributes, +10 more
Locations: UDUS - Heinrich-Heine-University Duesseldorf Düsseldorf, Germany

FISM - Italian Multiple Sclerosis Foundation Genova, Italy

WDO - World Duchenne Organization Veenendaal, Netherlands

AHUS - Akershus University Hospital Lørenskog, Akershus, Norway

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Recruiting

Gene Discovery Core, The Manton Center

NCT02743845

Conditions: Disease Attributes, Orphan Diseases, Pathologic Processes, +4 more
Locations: Boston Children's Hospital Boston, Massachusetts, United States

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Recruiting

Genetic of Intellectual Deficiency and Autism Spectrum Disorders (RaDiCo-GenIDA)

NCT06871696

Conditions: Autism Spectrum Disorder, Child Development Disorders, Pervasive, Disease Attributes, +11 more
Locations: RaDiCo-GenIDA Paris, Île-de-France Region, France

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