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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

A Safety and Tolerability Study of VRDN-003 in Participants With Thyroid Eye Disease (TED)

NCT06812325

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Catalina Eye Care Tuscon, Arizona, United States
  • United Medical Research Institute Inglewood, California, United States
  • Orbital Plastics Newport Beach, California, United States
  • California Eye Specialists Medical Group INC Pasadena, California, United States
View all 64 locations
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Active, not recruiting

A Study to See How Well Lebrikizumab Works in Adults and Adolescents With Moderate Atopic Dermatitis (Eczema) and High Itch Burden

NCT07006792

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Total Skin & Beauty Dermatology Center, PC DBA Total Dermatology Birmingham, Alabama, United States
  • Research Solutions of Arizona Litchfield Park, Arizona, United States
  • Alliance Dermatology and Mohs Center Phoenix, Arizona, United States
  • California Dermatology & Clinical Research Institute Encinitas, California, United States
View all 66 locations
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Active, not recruiting

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

NCT05668741

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • University of Alabama at Birmingham - Child Health Research Unit Birmingham, Alabama, United States
  • Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach Long Beach, California, United States
  • Stanford University - Palo Alto - Pulmonology Palo Alto, California, United States
  • National Jewish Health Denver, Colorado, United States
View all 26 locations
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Active, not recruiting

X-linked Hypophosphatemia Disease Monitoring Program

NCT03651505

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +27 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Rady Children's Hospital San Diego San Diego, California, United States
  • University of California San Francisco San Francisco, California, United States
View all 38 locations
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Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Lucile Packard Children's Hospital Stanford Palo Alto, California, United States
  • University of California at Davis Medical Center Sacramento, California, United States
  • Rady Children's Hospital-San Diego San Diego, California, United States
View all 46 locations
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Active, not recruiting

Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT05101148

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +14 more
Locations
  • Research Site Rochester, Minnesota, United States
  • Research Site Akron, Ohio, United States
  • Research Site Bydgoszcz, Poland
  • Research Site Gdansk, Poland
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Active, not recruiting

UPLC-MS/MS Monitoring of Emicizumab Therapy

NCT04472169

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • CHU de Caen Caen, France
  • Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU Lille, France
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Active, not recruiting

Phase 2b Study of EVO756 in Adults With Atopic Dermatitis

NCT07150845

Conditions
Atoptic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +14 more
Locations
  • Saguaro Dermatology Phoenix, Arizona, United States
  • Center for Dermatology Clinical Research Fremont, California, United States
  • Metropolis Dermatology - Downtown Los Angeles Los Angeles, California, United States
  • Skin and Beauty Center Pasadena, California, United States
View all 29 locations
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Active, not recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

NCT06052800

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number : 1580004 Taichung, Taiwan
  • Investigational Site Number : 1580005 Tainan, Taiwan
  • Investigational Site Number : 1580001 Taipei, Taiwan
  • Investigational Site Number : 1580003 Taipei, Taiwan
View Trial Details

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