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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.

12,619 public trials 2,162 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Phase 2b Study of Zagociguat in Patients With MELAS

NCT06402123

Conditions
Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +23 more
Locations
  • UC San Diego - Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
View all 20 locations
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Completed

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

NCT03584165

Conditions
Choroid Diseases, Choroideremia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +6 more
Locations
  • UCLA - Jules Stein Eye Institute Los Angeles, California, United States
  • Vitreo Retinal Associates PA - The Millennium Center Gainesville, Florida, United States
  • University of Miami Miami, Florida, United States
  • Johns Hopkins Hospital Baltimore, Maryland, United States
View all 25 locations
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Completed

Evaluation of Biomarkers of Atopic Dermatitis in Pediatric Patients (PEDISTAD BIOMARKER STUDY)

NCT03849716

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis Atopic, +9 more
Locations
  • C Squared Research Center Site Number : 8400068 Birmingham, Alabama, United States
  • Cahaba Dermatology Site Number : 8400046 Birmingham, Alabama, United States
  • C2 Research Center, LLC Site Number : 8400071 Montgomery, Alabama, United States
  • Axis Clinical Trials Site Number : 8400025 Los Angeles, California, United States
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Completed

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

NCT04085523

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +6 more
Locations
  • Ascendis Pharma Investigational Site Little Rock, Arkansas, United States
  • Ascendis Pharma Investigational Site Aurora, Colorado, United States
  • Ascendis Pharma Investigational Site Saint Paul, Minnesota, United States
  • Ascendis Pharma Investigational Site Columbia, Missouri, United States
View all 15 locations
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