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.

12,619 public trials 2,162 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Completed

ATHN 9: Severe VWD Natural History Study

NCT03853486

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +8 more
Locations
  • Center for Inherited Blood Disorders Orange, California, United States
  • University of Colorado Denver Hemophilia and Thrombosis Center Aurora, Colorado, United States
  • Connecticut Bleeding and Clotting Disorders Center Farmington, Connecticut, United States
  • University of Florida Hemophilia Treatment Center Gainesville, Florida, United States
View all 22 locations
View Trial Details
Completed

Therapies for Down Syndrome Regression Disorder

NCT05662228

Conditions
Abnormalities, Multiple, Autoimmune Diseases, Autoimmune Diseases of the Nervous System, +16 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View Trial Details
Completed

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Los Angeles Biomedical Research Institute at Harbor-UCLA Torrance, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 30 locations
View Trial Details
Completed

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT05358886

Conditions
Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Amnova Clinical Research Irvine, California, United States
  • Thompson Autism & Neurodevelopment Center - CHOC Orange, California, United States
  • UC Davis Health System Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 17 locations
View Trial Details
Completed

Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR-positive, HER2-negative, Advanced Breast Cancer (aBC) With PIK3CA Mutation, Whose Disease Progressed on or After Endocrine Treatment

NCT05631795

Conditions
Advanced Breast Cancer, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine Gland Neoplasms, +8 more
Locations
  • Novartis Investigative Site Guwahati, Assam, India
  • Novartis Investigative Site Surat, Gujarat, India
  • Novartis Investigative Site Thalassery, Kerala, India
  • Novartis Investigative Site Trivandrum, Kerala, India
View Trial Details
Completed

Phase II Study to Evaluate Efficacy and Safety of D-0120 in Combination With Allopurinol in Subjects With Gout

NCT05665699

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • Study Center Birmingham, Alabama, United States
  • Site Center Plantation, Florida, United States
  • Study Center Evergreen Park, Illinois, United States
  • Study Center Brownsburg, Indiana, United States
View all 19 locations
View Trial Details
Completed

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

NCT03263559

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Colorado - Denver/Children's Hospital of Colorado Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
View all 32 locations
View Trial Details

Related conditions