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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.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

NCT05891249

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • Local Institution - 0002 Ahmedabad, Gujarat, India
  • Local Institution - 0001 New Delhi, National Capital Territory of Delhi, India
  • Local Institution - 0007 Kolkata, West Bengal, India
  • Local Institution - 0005 Assam, India
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Active, not recruiting

A Study of Lebrikizumab (LY3650150) With/Without Topical Corticosteroid Treatment in Participants With Moderate-to-Severe Atopic Dermatitis

NCT06280716

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Wannan Medical College Yijishan Hospital Wuhu, Anhui, China
  • China-Japan Friendship Hospital Beijing, Beijing Municipality, China
  • Peking University People's Hospital Beijing, Beijing Municipality, China
  • Beijing Children's hospital, Capital Medical University Beijing, Beijing Municipality, China
View all 46 locations
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Active, not recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +9 more
Locations
  • Rush University Medical Center & Children's Hospital Chicago, Illinois, United States
  • Gillette Children's Specialty Healthcare Saint Paul, Minnesota, United States
  • Washington University, St. Louis St Louis, Missouri, United States
  • CHU Ste-Justine Montreal, Quebec, Canada
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Active, not recruiting

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

NCT05899673

Conditions
Alpha1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • UCSD Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Stanford Medicine Outpatient Center Redwood City, California, United States
  • UF Clinical and Translational Science Institute Gainesville, Florida, United States
  • University Of Iowa Hospitals And Clinics Iowa City, Iowa, United States
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Active, not recruiting

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386

Conditions
Amyloid Cardiomyopathy, Transthyretin-Related, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +13 more
Locations
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • University of Colorado Hospital - Anschutz Medical Campus Aurora, Colorado, United States
  • Northwestern University Chicago, Illinois, United States
  • University of Chicago Medical Center Chicago, Illinois, United States
View all 72 locations
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Active, not recruiting

CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

NCT03350828

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • The Children's Hospital Alabama, University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California San Diego La Jolla, California, United States
  • Childrens Hospital Los Angeles Los Angeles, California, United States
View all 52 locations
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Active, not recruiting

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT05367960

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
  • MIND Institute UC Davis Medical Center Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 17 locations
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