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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.

12,633 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

NCT05630066

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Rush Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Columbia University Medical Center New York, United States
  • Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine Carrboro, North Carolina, United States
View all 19 locations
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Completed

CArotenoid in hypoChOlesterolemia

NCT05208879

Conditions
Abetalipoproteinemia, Chylomicron Retention Disease, Chylomicron retention disease, +17 more
Locations
  • Fédération d'endocrinologie, maladies métaboliques, diabète et nutrition Hôpital cardiovasculaire et pneumologique Louis Pradel Bron, France
  • UF nutrition pédiatrique, Service hépatologie, gastroentérologie et nutrition pédiatrique Hôpital Femme Mère Enfant de Lyon (GHE-HFME) Bron, France
  • UF nutrition pédiatrique, Service hépatologie, gastroentérologie et nutrition pédiatrique Hôpital Femme Mère Enfant de Lyon (GHE-HFME) Bron, France
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Completed

Efficacy Study of EVO301 in Moderate to Severe Atopic Dermatitis

NCT06723405

Conditions
Atopic Dermatitis (AD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +11 more
Locations
  • Cornerstone Dermatology Coorparoo, Australia
  • Momentum Clinical Research Darlinghurst Darlinghurst, Australia
  • Pacific Clinical Research Network (PCRN) Auckland Auckland, New Zealand
  • Optimal Clinical Trials North Auckland, New Zealand
View Trial Details
Completed

A Study to Assess Efficay and Safety of LC350189 Different Doses in Gout Patients With Hyperuricemia

NCT03934099

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +12 more
Locations
  • Synexus Clinical Research Birmingham, Alabama, United States
  • Synexus Clinical Research Chandler, Arizona, United States
  • Synexus Clinical Research Mesa, Arizona, United States
  • Synexus Clinical Research Tucson, Arizona, United States
View all 34 locations
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Completed

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

NCT05517421

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Site Number - 1557 Beverly Hills, California, United States
  • Site Number - 1539 Carlsbad, California, United States
  • Site Number - 1502 Los Angeles, California, United States
  • Site Number - 1505 Los Angeles, California, United States
View all 93 locations
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Completed

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

NCT05626634

Conditions
Brain Diseases, CDKL5 deficiency disorder, Central Nervous System Diseases, +23 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Rancho Los Amigos National Rehabilitation Center (RLANRC) Downey, California, United States
  • University of California San Francisco San Francisco, California, United States
  • Northwest Florida Clinical Research Group Gulf Breeze, Florida, United States
View all 28 locations
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