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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.

12,589 public trials 2,156 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Baltimore, Maryland, United States
  • Research Site Kansas City, Missouri, United States
  • Research Site Durham, North Carolina, United States
  • Research Site Nedlands, Australia
View all 32 locations
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Active, not recruiting

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

NCT05422222

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University Clinical and Translational Research Unit Palo Alto, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston Atlanta, Georgia, United States
View all 38 locations
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Active, not recruiting

Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta

NCT05972551

Conditions
Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +7 more
Locations
  • Childrens Hospital of Alabama Birmingham, Alabama, United States
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • University of California, Los Angeles Interventional Clinical Trials Los Angeles, California, United States
  • Nemours Hospital for Children Wilmington, Delaware, United States
View all 65 locations
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Active, not recruiting

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799

Conditions
Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Southwest Autism Research and Resource Center Phoenix, Arizona, United States
  • Science 37 Culver City, California, United States
  • Amnova Clinical Research, LLC Irvine, California, United States
  • UC Davis Health System, MIND Institute Sacramento, California, United States
View all 26 locations
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Active, not recruiting

Systems Biology of Early Atopy

NCT04798079

Conditions
Allergic Diseases, Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Sean N. Parker Center for Allergy & Asthma Research at Stanford University Stanford, California, United States
  • National Jewish Health Denver, Colorado, United States
  • Northwestern University Feinberg School of Medicine: Dept of Dermatology Chicago, Illinois, United States
View all 13 locations
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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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