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Clinical trial condition

Genetic Diseases, Inborn

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

12,627 public trials 2,162 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

NCT02932618

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +8 more
Locations
  • University of Colorado Hemophilia & Thrombosis Center Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University of Florida College of Medicine Jacksonville, Florida, United States
  • Bleeding and Clotting Disorders Institute Peoria, Illinois, United States
View all 46 locations
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Completed

Safety and Efficacy Evaluation of High Intensity Focused Ultrasound Device in Eyebrow Lifting

NCT06724510

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, Cutis Laxa, +7 more
Locations
  • Beijing Anzhen Hospital of Capital Medical University Beijing, China
  • Emergency General Hospital Beijing, China
  • TheThird Hospital of Hebei Medical University Shijiazhuang, China
  • The Second Affiliated Hospital of Xian Jiaotong University Xi'an, China
View Trial Details
Completed

Long-term Safety and Efficacy of Lebrikizumab in Adult and Adolescent Participant With Moderate-to-Severe Atopic Dermatitis

NCT05916365

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Investigator Site 7 Darmstadt, Hesse, Germany
  • Investigator Site 8 Frankfurt am Main, Hesse, Germany
  • Investigator Site 10 Bad Bentheim, Lower Saxony, Germany
  • Investigator Site 9 Osnabrück, Lower Saxony, Germany
View all 32 locations
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Completed

Pilot Study Related to the Effect of Clopidogrel on Plasmatic Soluble CD40 Ligand During Systemic Lupus Erythematous

NCT02320357

Conditions
Blood Protein Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dysgammaglobulinemia, +8 more
Locations
  • Service de Médecine Interne et maladies Infectieuses - Hôpital Saint-André Bordeaux, France
  • Service de Médecine Interne Limoges, France
  • Service de Médecine Interne et Immunopathologie Toulouse, France
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Completed

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT05758922

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
Locations
  • Hospital Pequeno Principe Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
View Trial Details
Completed

Tralokinumab Monotherapy for Children With Moderate-to-severe Atopic Dermatitis - TRAPEDS 1 (TRAlokinumab PEDiatric Trial no. 1)

NCT05388760

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • LEO Pharma Investigational Site Brno, Czechia
  • LEO Pharma Investigational Site Prague, Czechia
  • LEO Pharma Investigational Site Reims, Ardennes, France
  • LEO Pharma Investigational Site Rotterdam, Netherlands
View Trial Details
Completed

Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain

NCT02450604

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations
  • Cs douleur chronique GHSR, CHU Sud Réunion Saint-Pierre, La Réunion, France
  • CeRCa, Unité de Neuromyologie, CHU de Fort de France FORT de France, Martinique, France
  • Centre Douleurs Chroniques, CH de la Côte Basque Bayonne, France
  • Centre Douleurs Chroniques, CHU de Bordeaux Bordeaux, France
View all 21 locations
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