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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Active, Not Recruiting

SEQUence of Endocrine Therapy in Advanced Luminal Breast Cancer (SEQUEL-Breast)

NCT05392608

Conditions
Breast Diseases, Breast Neoplasms, Congenital, Familial and Genetic Disorders, +7 more
Locations
  • Jeroen Bosch Ziekenhuis 's-Hertogenbosch, Netherlands
  • Noordwest Ziekenhuisgroep Alkmaar, Netherlands
  • Ziekenhuisgroep Twente Almelo, Netherlands
  • Meander Medisch Centrum Amersfoort, Netherlands
View all 25 locations
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Active, Not Recruiting

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

NCT07226622

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • University of California Los Angeles Medical Center Los Angeles, California, United States
  • University of California Davis Health Sacramento, California, United States
  • Yale New Haven Hospital New Haven, Connecticut, United States
  • Northwestern University Chicago, Illinois, United States
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Active, Not Recruiting

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

NCT04624659

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +12 more
Locations
  • Univ of Alabama Birmingham Birmingham, Alabama, United States
  • Phoenix Children's Hsptl Phoenix, Arizona, United States
  • [Legal] Woodland International Research Group, LLC Little Rock, Arkansas, United States
  • [Legal] Collaborative Neuroscience Network, LLC Long Beach, California, United States
View all 142 locations
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Active, Not Recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

NCT06224907

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Cardiovascular Diseases, +9 more
Locations
  • Asahikawa Medical University Hospital Hokkaido, Asahikawa, Japan
  • Saitama Medical University Hospital Saitama, Iruma-gun, Japan
  • Nagoya University Hospital Aichi, Nagoya, Japan
  • Tokyo Medical University Hospital Tokyo, Shinjuku-ku, Japan
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Active, Not Recruiting

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03424018

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +6 more
Locations
  • Children's Hospital & Research Center Oakland Oakland, California, United States
  • Harbor - UCLA Medical Center Torrance, California, United States
  • Alfred I. duPont Hospital for Children Wilmington, Delaware, United States
  • Emory University Atlanta, Georgia, United States
View all 24 locations
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