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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.

12,593 public trials 2,156 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Evaluation of Efficacy and Safety of AR882 and XOI Co-administration in Uricase Treatment Failed Patients

NCT07116746

Conditions
Arthritis, Arthritis, Gouty, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations
  • Arthrosi Investigative Site (105) Margate, Florida, United States
  • Arthrosi Investigative Site (102) Miami, Florida, United States
  • Arthrosi Investigative Site (109) Tampa, Florida, United States
  • Arthrosi Investigative Site (106) Jackson, Mississippi, United States
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Active, not recruiting

Promising ROd-cone DYstrophy Gene therapY

NCT05748873

Conditions
Cone-Rod Dystrophies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +6 more
Locations
  • Bascom Palmer Eye Institute/University of Miami Miami, Florida, United States
  • Mass Eye and Ear Boston, Massachusetts, United States
  • Casey Eye Institute Portland, Oregon, United States
  • UPMC Eye Center Pittsburgh, Pennsylvania, United States
View Trial Details
Active, not recruiting

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

NCT06634420

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Medical Research of Arizona Scottsdale, Arizona, United States
  • University of California, San Diego (UCSD) San Diego, California, United States
  • Raffi Tachdjian MD, Inc Santa Monica, California, United States
  • Allergy & Asthma Clinical Research Walnut Creek, California, United States
View all 29 locations
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Active, not recruiting

CF Bronchodilation

NCT03522831

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +10 more
Locations
  • UBC Cardiopulmonary Exercise Physiology Laboratory Vancouver, British Columbia, Canada
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Active, not recruiting

Phase 2a Study to Assess the Efficacy,Safety and Tolerability of GIA632 in Adult Participants With Moderate to Severe Atopic Dermatitis

NCT07220577

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Ctr for Dermatology Clinical Res Fremont, California, United States
  • Aesthetics Skin Care Dermatologic Surgery Rockville, Maryland, United States
  • Care Access Hoboken Hoboken, New Jersey, United States
  • Novartis Investigative Site Pleven, Bulgaria
View all 28 locations
View Trial Details
Active, not recruiting

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

NCT01089101

Conditions
Astrocytoma, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +25 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Lucile Packard Children's Hospital Stanford University Palo Alto, California, United States
  • UCSF Medical Center-Mount Zion San Francisco, California, United States
  • UCSF Medical Center-Parnassus San Francisco, California, United States
View all 17 locations
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Active, not recruiting

A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome

NCT05690386

Conditions
Cardiovascular Abnormalities, Cardiovascular Diseases, Chromosome Disorders, +17 more
Locations
  • Ascendis Pharma Investigational Site Palo Alto, California, United States
  • Ascendis Pharma Investigational Site San Diego, California, United States
  • Ascendis Pharma Investigational Site Aurora, Colorado, United States
  • Ascendis Pharma Investigational Site Orlando, Florida, United States
View all 19 locations
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