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.

12,656 public trials 2,147 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Completed

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

NCT03073733

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations
  • Gavin Herbert Eye Inst, Univ Cal Irvine Irvine, California, United States
  • Retina-Vitreous Associates Medical Group Los Angeles, California, United States
  • Ophthalmic Consultants of Boston Boston, Massachusetts, United States
View Trial Details
Completed

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

NCT02526160

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • University of California-San Francisco Medical Center San Francisco, California, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Indiana University Department of Medicine Indianapolis, Indiana, United States
View all 25 locations
View Trial Details
Completed

Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

NCT02537431

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • UCSF Medical Center at Mission San Francisco, California, United States
  • Yale University School of Medicine - Yale New-Haven Hospital/Yale Center for Clinical Investigation New Haven, Connecticut, United States
  • Indiana University Department of Medicine University Hospital Indianapolis, Indiana, United States
  • Duke University Medical Center Durham, North Carolina, United States
View all 14 locations
View Trial Details
Completed

A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

NCT01340482

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • University of California, San Francisco Medical Center San Francisco, California, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Clinical Research Center, Indiana University School of Medicine Indianapolis, Indiana, United States
  • Duke Clinical Research Unit Durham, North Carolina, United States
View Trial Details
Completed

A Study of KRN23 in X-linked Hypophosphatemia

NCT00830674

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Yale University School of Medicine New Haven, Connecticut, United States
  • General Clinical Research Center, Indiana University School of Medicine Indianapolis, Indiana, United States
  • Duke Clinical Research Unit Durham, North Carolina, United States
  • University of Texas Health Science Center at Houston Houston, Texas, United States
View Trial Details
Completed

Study to Assess the Safety and PK of Oral and IV Xenleta in Adults With Cystic Fibrosis

NCT05225805

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • University of Kansas School of Medicine Kansas City, Kansas, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • New York Medical College Valhalla, New York, United States
  • University Hospitals Cleveland Medical Center Cleveland, Ohio, United States
View Trial Details
Completed

ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)

NCT02078427

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Royal Prince Alfred Hospital Camperdown, New South Wales, Australia
  • South Metropolitan Health Service trading as Fiona Stanley Hospital Murdoch, Western Australia, Australia
  • Landes Frauen und Kinderklinik Linz (LFKK Linz) Linz, Austria
  • Kepler Universitätsklinikum Klinik für Kinder-und Jugendheilkunde Linz, Austria
View all 110 locations
View Trial Details
Completed

Tradipitant in Treatment-resistant Pruritus Associated With Atopic Dermatitis

NCT02651714

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Vanda Investigational Site Encinitas, California, United States
  • Vanda Investigational Site Encino, California, United States
  • Vanda Investigational Site Lomita, California, United States
  • Vanda Investigational Site San Diego, California, United States
View all 28 locations
View Trial Details
Completed

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

NCT05076149

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Banner University of Arizona Medical Center Tucson, Arizona, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
View all 170 locations
View Trial Details

Related conditions