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Rare Diseases

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

466 public trials 83 recruiting studies

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Rare Diseases trial results

Recruiting

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

NCT07362225

Conditions: Accelerated Phase MPN, Accelerated Phase Myeloproliferative Neoplasm, Agnogenic Myeloid Metaplasia, +78 more
Locations: MPN Research Foundation Chicago, Illinois, United States

Memorial Sloan Kettering Cancer Center New York, United States

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Recruiting

Institutional Registry of Rare Diseases

NCT06573723

Conditions: Abnormalities, Multiple, Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, +144 more
Locations: Hospital Italiano de Buenos Aires Buenos Aires, Argentina

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Recruiting

Automatic Phenotyping of Patients on 2D Photography

NCT06219421

Conditions: Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +6 more
Locations: Necker - Hôpital des Enfants Malades Paris, France

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Recruiting

Umbrella Study for Single Patient Treatments in Oncology

NCT06285500

Conditions: Advanced Cancer, Disease Attributes, Pathologic Processes, +2 more
Locations: University Health Network, Princess Margaret Cancer Centre Toronto, Ontario, Canada

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Recruiting

Swiss Rare Disease Registry (SRDR)

NCT05179863

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +1 more
Locations: Kantonsspital Aarau, Pädiatrie Aarau, Switzerland

Kantonsspital Aarau Aarau, Switzerland

Center for Rare Diseases, Basel Basel, Switzerland

Universitäs-Kinderspital beider Basel, UKBB Basel, Switzerland

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Recruiting

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636

Conditions: Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +30 more
Locations: University of California - San Francisco San Francisco, California, United States

Yale Center for XLH/ Yale University School of Medicine New Haven, Connecticut, United States

Indiana University School of Medicine University Hospital Indianapolis, Indiana, United States

Mayo Clinic Rochester, Minnesota, United States

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Recruiting

Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center

NCT07205861

Conditions: Autoimmune Diseases, Blood Coagulation Disorders, Blood Platelet Disorders, +24 more
Locations: Service d'Hématologie Hôpital Saint-Antoine Paris, France

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Recruiting

Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers

NCT06474104

Conditions: Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, +2 more
Locations: Sheba Medical Center - Tel Ashomer Ramat Gan, Israel

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Recruiting

Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport)

NCT05927467

Conditions: Alport Syndrome, Collagen Diseases, Congenital Abnormalities, +16 more
Locations: RaDiCo Eurbio-Alport Paris, Île-de-France Region, France

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Recruiting

Aortic Stiffness in Patients With Genetic Aortopathies

NCT05715203

Conditions: Aneurysm, Aneurysm, Ascending Aorta, Aortic Aneurysm, +10 more
Locations: IRCCS Policlinico San Donato San Donato Milanese, Lombardy, Italy

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