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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

NCT06924970

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • UCHealth at University of Colorado Anschutz Medical Campus Aurora, Colorado, United States
  • UConn Health Farmington, Connecticut, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • MedStar Washington Hospital Center Washington D.C., District of Columbia, United States
View all 30 locations
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Active, not recruiting

Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)

NCT04944979

Conditions
Agammaglobulinemia, Blood Protein Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations
  • Benioff Children's Hospital - Mission Bay San Francisco, California, United States
  • IMMUNOe Health and Research Centers Centennial, Colorado, United States
  • Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States
  • Velocity Clinical Research - MedPharmics - Lafayette Lafayette, Louisiana, United States
View all 21 locations
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Active, not recruiting

Chronic Pain and Hemophilia

NCT05202951

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Chronic Pain, +12 more
Locations
  • CHU de Bordeaux Bordeaux, France
  • Centre Hospitalier Universitaire de Caen Normandie Caen, France
  • CH Métropole Savoie Chambéry, France
  • CHU de Clermont-Ferrand Clermont-Ferrand, France
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Active, not recruiting

Study to Assess the Long-term Safety of Dupilumab Administered in Participants ≥6 Months to <18 Years of Age With Atopic Dermatitis (AD)

NCT02612454

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Regeneron Investigational Site Birmingham, Alabama, United States
  • Regeneron Investigational Site Gilbert, Arizona, United States
  • Regeneron Investigational Site Bakersfield, California, United States
  • Regeneron Investigational Site Long Beach, California, United States
View all 84 locations
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Active, not recruiting

A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001

NCT06852963

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases Hereditary, +7 more
Locations
  • University of Florida College of Medicine Jacksonville, Florida, United States
  • Bascom Palmer Eye Institute - University of Miami Miami, Florida, United States
  • Kellogg Eye Center - University of Michigan Ann Arbor, Michigan, United States
  • Casey Eye Institute - OHSU Portland, Oregon, United States
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Active, not recruiting

Safety, Tolerability and PK of ATTO-3712 in Healthy Volunteers and Patients With Atopic Dermatitis

NCT07205081

Conditions
Atopic Dermatitis (AD), Atopic Eczema, Atopic Eczema/Dermatitis (Non-Specific), +16 more
Locations
  • Attovia Clinical Site 203 Encinitas, California, United States
  • Attovia Clinical Site 202 Saint Joseph, Missouri, United States
  • Attovia Clinical Site 102 Peterborough, Ontario, Canada
  • Altasciences Montreal, Quebec, Canada
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Active, not recruiting

Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)

NCT05882734

Conditions
Bronchial Neoplasms, Carcinoma, Bronchogenic, Carcinoma, Non-Small-Cell Lung, +19 more
Locations
  • UCLA Hematology and Oncology - Santa Monica Santa Monica, California, United States
  • UPMC Cancer Center Pittsburgh, Pennsylvania, United States
  • Tennessee Cancer Specialists - Biomedical Research Knoxville, Tennessee, United States
  • The University of Texas MD Anderson Cancer Center Houston, Texas, United States
View all 54 locations
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Active, not recruiting

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Baltimore, Maryland, United States
  • Research Site Kansas City, Missouri, United States
  • Research Site Durham, North Carolina, United States
  • Research Site Nedlands, Australia
View all 32 locations
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Active, not recruiting

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

NCT05422222

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University Clinical and Translational Research Unit Palo Alto, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston Atlanta, Georgia, United States
View all 38 locations
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