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.

12,603 public trials 2,158 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Active, not recruiting

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

NCT06211634

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +7 more
Locations
  • University of California, San Diego (UCSD) (Part B/C) La Jolla, California, United States
  • Tulane University Medical Center (Part B/C) New Orleans, Louisiana, United States
  • Mayo Clinic - Rochester (Part B/C) Rochester, Minnesota, United States
  • Hemophilia Center of Western Pennsylvania (HCWP) (Part B/C) Pittsburgh, Pennsylvania, United States
View all 17 locations
View Trial Details
Active, not recruiting

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCT04783181

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +16 more
Locations
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Lucas Research, Inc. Morehead City, North Carolina, United States
View Trial Details
Active, not recruiting

Amyloidosis TTR Flow Reserve Evaluation

NCT05103943

Conditions
Amyloid Cardiomyopathy, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +13 more
Locations
  • CHU d'ANGERS Angers, France
  • CHU Caen Caen, France
  • CHU Orleans Orléans, France
  • CHRU de TOURS Tours, France
View Trial Details
Active, not recruiting

A Study to Evaluate Upadacitinib in Combination With Topical Corticosteroids in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis

NCT03568318

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Total Skin and Beauty Derm Ctr /ID# 200548 Birmingham, Alabama, United States
  • Clinical Research Center AL /ID# 201865 Birmingham, Alabama, United States
  • ACCEL Research Sites /ID# 213364 Birmingham, Alabama, United States
  • Advanced Dermatology and Skin Care Centre /ID# 213550 Mobile, Alabama, United States
View all 193 locations
View Trial Details
Active, not recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • University of Minnesota, Pediatrics Minneapolis, Minnesota, United States
  • Ospedale San Raffaele Milan, Italy
  • Princess Maxima Center Utrecht, Netherlands
  • UMC Utrecht Utrecht, Netherlands
View Trial Details
Active, not recruiting

Study of Aspirin in Patients With Vestibular Schwannoma

NCT03079999

Conditions
Acoustic Neuroma, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cranial Nerve Diseases, +29 more
Locations
  • Stanford Otolaryngology/HNS Stanford, California, United States
  • University of Miami Miami, Florida, United States
  • Massachusetts Eye and Ear Boston, Massachusetts, United States
  • Mayo Clinic Rochester, Minnesota, United States
View Trial Details

Related conditions