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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

NCT03588299

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Arkansas Children's Hospital - Hematology / Oncology Little Rock, Arkansas, United States
  • C.S. Mott Children's Hospital - Hematology / Oncology Ann Arbor, Michigan, United States
  • UW Health Carbone Cancer Center Madison, Wisconsin, United States
  • SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD Sofia, Sofia City Province, Bulgaria
View all 13 locations
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Active, not recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
  • University of California Irvine Irvine, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
View all 42 locations
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Active, not recruiting

CINC424A2X01B Rollover Protocol

NCT02386800

Conditions
Acute Myeloid Leukemia, Anemia, Anemia, Hemolytic, +31 more
Locations
  • Novartis Investigative Site Darlinghurst, New South Wales, Australia
  • Novartis Investigative Site Herston, Queensland, Australia
  • Novartis Investigative Site Wooloongabba, Queensland, Australia
  • Novartis Investigative Site Bedford Park, South Australia, Australia
View all 97 locations
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Active, not recruiting

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

NCT05035030

Conditions
Abnormalities, Multiple, Alagille Syndrome, Bile Duct Diseases, +11 more
Locations
  • Rady Children's Hospital San Diego, California, United States
  • UCSF San Francisco, California, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Riley Hospital for Children at IU Health Indianapolis, Indiana, United States
View all 35 locations
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Active, not recruiting

Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A

NCT03932201

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • South Alabama Medical Science Foundation Mobile, Alabama, United States
  • Banner MD Anderson Cancer Center Phoenix, Arizona, United States
  • University California Davis Davis, California, United States
  • Orthopaedic Hospital DBA Orthopaedic Institute for Children Los Angeles, California, United States
View all 29 locations
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Active, not recruiting

Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding

NCT04461639

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Many Locations Multiple Locations, Austria
  • Many Locations Multiple Locations, Germany
  • Many Locations Multiple Locations, Greece
  • Many Locations Multiple Locations, Italy
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Active, not recruiting

Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)

NCT04195555

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ependymoma, Female Urogenital Diseases, +80 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Providence Alaska Medical Center Anchorage, Alaska, United States
  • Banner Children's at Desert Mesa, Arizona, United States
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
View all 174 locations
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