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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.

12,619 public trials 2,162 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of Florida College of Medicine Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 15 locations
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Completed

A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries

NCT07251933

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Hospital de Ninos Sor Maria Ludovica La Plata, Buenos Aires, Argentina
  • Centro de Alergia e Inmunologia Clinica Mar del Plata Mar del Plata, Buenos Aires, Argentina
  • Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan Ciudad de Buenos Aires, Buenos Aires F.D., Argentina
  • Hospital Italiano de Buenos Aires Buenos Aires, Ciudad Autonoma de BuenosAires, Argentina
View all 24 locations
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Completed

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

NCT05598320

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +5 more
Locations
  • Ascendis Pharma Investigational Site Saint Paul, Minnesota, United States
  • Ascendis Pharma Investigational Site Columbia, Missouri, United States
  • Ascendis Pharma Investigational Site Houston, Texas, United States
  • Ascendis Pharma Investigational Site Madison, Wisconsin, United States
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Completed

Proof of Concept Study of Rilzabrutinib in Adult Patients With Moderate-to-severe Atopic Dermatitis

NCT05018806

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Antelope Valley Clinical Trials Site Number : 8400001 Northridge, California, United States
  • Asthma and Allergy Associates, PC Site Number : 8400008 Colorado Springs, Colorado, United States
  • Florida International Research Center Site Number : 8400002 Miami, Florida, United States
  • Skin Sciences, PLLC Site Number : 8400005 Louisville, Kentucky, United States
View all 31 locations
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Completed

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

NCT05181839

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Centre Hospitalier Universitair de Lille Lille, France
  • Hospices Civils De Lyon Lyon, France
  • APHP Paris - Assistance Publique Hopitaux de Paris Paris, France
  • Charité - Universitätsmedizin Berlin Berlin, State of Berlin, Germany
View all 14 locations
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