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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.

12,597 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

NCT01659606

Conditions
Anemia, Anemia, Aplastic, Aplastic Anemia, +18 more
Locations
  • University of Chicago Chicago, Illinois, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • Boston Children's Hospital (pediatric patients) Boston, Massachusetts, United States
  • Dana-Farber Cancer Institute (adult patients) Boston, Massachusetts, United States
View all 13 locations
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Active, not recruiting

ATI-045 Versus Placebo in Patients With Moderate-to-Severe Atopic Dermatitis

NCT07011706

Conditions
AD, Atopic, Atopic Dermatitis, +12 more
Locations
  • Aclaris Clinical Site Phoenix, Arizona, United States
  • Aclaris Clinical Site Tucson, Arizona, United States
  • Aclaris Study Site Bryant, Arkansas, United States
  • Aclaris Clinical Site Beverly Hills, California, United States
View all 60 locations
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Active, not recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Clinical Trial Site Campbelltown, Australia
  • Clinical Trial Site Grenoble, France
  • Clinical Trial Site Lille, France
  • Clinical Trial Site Paris, France
View Trial Details
Active, not recruiting

Colorectal Cancer Screening in Cystic Fibrosis

NCT05362344

Conditions
Adenoma, Colonic Diseases, Colorectal Cancer, +19 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • Stanford University Medical Center Palo Alto, California, United States
  • National Jewish Health, University of Colorado Denver, Colorado, United States
  • University of Miami Hospital Miami, Florida, United States
View all 26 locations
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Active, not recruiting

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

NCT04924608

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +17 more
Locations
  • Research Site Gainesville, Florida, United States
  • Research Site Rockville, Maryland, United States
  • Research Site St Louis, Missouri, United States
  • Research Site Commack, New York, United States
View all 34 locations
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Active, not recruiting

A Longitudinal Observational Study of Patients Undergoing Therapy for IMISC

NCT03661866

Conditions
Alopecia, Alopecia Areata, Atopic Dermatitis, +35 more
Locations
  • Clear Dermatology & Aesthetics Center/Investigative MD Scottsdale, Arizona, United States
  • Johnson Dermatology Fort Smith, Arkansas, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • Arkansas Dermatology Little Rock, Arkansas, United States
View all 62 locations
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Active, not recruiting

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

NCT01687608

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Orthopaedic Hemophilia Treatment Center Los Angeles, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California Davis Medical Center Sacramento, California, United States
  • University of California at San Diego Medical Center San Diego, California, United States
View all 15 locations
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