Skip to main content
OpenTrials

Clinical trial condition

Genetic Diseases, Inborn

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

12,537 public trials 2,139 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

NCT06128629

Conditions
Amyloidosis, Amyloidosis, Familial, Amyloidosis, Hereditary, Transthyretin-Related, +10 more
Locations
  • University of Arizona Tucson, Arizona, United States
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Western Greater Los Angeles VA Medical Center Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
View all 132 locations
View Trial Details
Recruiting

Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

NCT06273644

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Genetic Diseases, Inborn, +9 more
Locations
  • Nordland Hospital Trust Bodø, Nordland, Norway
  • Studieenheten, Akademiskt Specialistcentrum, Stockholm läns sjukvårdsområde, Region Stockholm Stockholm, Sweden
  • Norrland University Hospital Umeå, Sweden
View Trial Details
Recruiting

Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress

NCT04541654

Conditions
Clonal Hematopoiesis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +9 more
Locations
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Brigham and Women's Hospital Boston, Massachusetts, United States
  • Judy E. Garber Boston, Massachusetts, United States
View Trial Details
Recruiting

Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)

NCT05071222

Conditions
Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +10 more
Locations
  • Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital Paris, France
View Trial Details
Recruiting

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

NCT00920972

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hematologic Diseases, +7 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Children's Hospital of Orange County Orange, California, United States
  • University of California San Diego, California, United States
  • Yale School of Medicine New Haven, Connecticut, United States
View all 28 locations
View Trial Details

Related conditions