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Genetic Diseases, Inborn

Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,651 public trials 2,148 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Neurology Measures in FA Children

NCT03418740

Conditions: Ataxia, Brain Diseases, Central Nervous System Diseases, +16 more
Locations: University of California, Los Angeles Los Angeles, California, United States

University of Florida Gainesville, Florida, United States

Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States

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Completed

Thymus Transplantation Safety-Efficacy

NCT01220531

Conditions: 22q11 Deletion Syndrome, Abnormalities, Multiple, Cardiovascular Abnormalities, +23 more
Locations: John W. Sleasman Durham, North Carolina, United States

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Completed

Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A

NCT00323856

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations: Oddzial Chorob Wewnetrznych i Hematologii Poznan, Szkolna, Poland

Katedra i Klinika Hematologii Collegium Medicum UJ Krakow, Poland

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Completed

The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease

NCT06762509

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: the First Affiliated Hospital of Anhui University of Traditional Chinese Medicine, #117 Meishan Street, Shushan District Hefei 230037, China Hefei, China

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Completed

Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC

NCT05956314

Conditions: Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations: Royal London Hospital-Clinical Research Facility-11D (11th Floor) Whitechapel, London, E1 1FR, London, Whitechapel Rd, United Kingdom

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Completed

Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases

NCT05199402

Conditions: Brain Diseases, Central Nervous System Diseases, Congenital Abnormalities, +15 more
Locations: Rima Nabbout Paris, France

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Completed

Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease

NCT03629678

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations: The Ohio State University Medical Center Columbus, Ohio, United States

Vanderbilt University Nashville, Tennessee, United States

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Completed

Fatigue, Depressive Disorders and Insomnia in Adult Patients with Marfan Syndrome and Ehlers-Danlos Syndrome: Survey

NCT05712564

Conditions: Abnormalities, Multiple, Behavior, Behavioral Symptoms, +37 more
Locations: IRCCS Policlinico San Donato San Donato Milanese, Lombardy, Italy

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Completed

EVALUATION of the OVERALL HAEMOSTATIC CAPACITY of MIM8 with GLOBAL HAEMOSTASIS ASSAYS and FIBRIN CLOT ULTRASTRUCTURE

NCT06212505

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations: Centre de Référence Hémophilie, Hôpital Louis Pradel- Hospices Civils de Lyon Bron, Rhone, France

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Completed

A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers

NCT06086613

Conditions: Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +7 more
Locations: Orange County Research Center Lake Forest, California, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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