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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Functional Capacity in Anderson-Fabry Disease Patients

NCT06884358

Conditions
Anderson-Fabry Disease, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Spedali Civili Hospital Brescia, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Italy
  • IRCCS Policlinico San Donato San Donato Milanese, Milan, Italy
  • Fondazione IRCCS San Gerardo dei Tintori Monza, Italy
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Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

NCT06349473

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +10 more
Locations
  • California Clinical Trials Medical Group (CCTMG) Glendale, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • LA Center for Bleeding and Clotting Disorders - Metairie Metairie, Louisiana, United States
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Recruiting

Targeted Investigation of Microbiome 2 Treat Atopic Dermatitis (TIME-2)

NCT06504160

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • University of California, San Diego: Dermatology Clinical Trials Unit San Diego, California, United States
  • National Jewish Health: Division of Pediatric Allergy and Clinical Immunology Denver, Colorado, United States
  • Northwestern University Feinberg School of Medicine: Department of Dermatology Chicago, Illinois, United States
  • New York University Langone Health: Department of Pediatric Allergy and Immunology New York, United States
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Recruiting

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

NCT06850038

Conditions
Abnormalities, Multiple, Alagille Syndrome, Bile Duct Diseases, +11 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • UCSF Benioff Children's Hospital San Francisco, California, United States
  • The Children's Mercy Hospital Kansas City, Missouri, United States
  • SSM Health Cardinal Glennon Children's Hospital St Louis, Missouri, United States
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Recruiting

An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome

NCT05066217

Conditions
Brain Diseases, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • UC Irvine Medical Center Orange, California, United States
  • UCI Center for Innovative Health Therapies Orange, California, United States
  • Nemours Children's Health Wilmington, Delaware, United States
View all 26 locations
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Recruiting

Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

NCT05022459

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Stanford University Palo Alto, California, United States
  • Rady Children's Hospital San Diego, California, United States
  • Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States
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Recruiting

Sapanisertib and Serabelisib (PIKTOR) in Various Combinations in Patients With HR+/HER2- Advanced/Metastatic Breast Cancer

NCT07558733

Conditions
Breast Diseases, Breast Neoplasms, Congenital Abnormalities, +15 more
Locations
  • START Los Angeles Los Angeles, California, United States
  • Rocky Mountain Cancer Centers Lone Tree, Colorado, United States
  • Oncology Associates of Oregon Springfield, Oregon, United States
  • SCRI Oncology Partners Nashville, Tennessee, United States
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Recruiting

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Duchene Muscular Dystrophy, +12 more
Locations
  • Arkansas Children's Research Institute Little Rock, Arkansas, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
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Recruiting

Study to Enable New Diagnostics for Pulmonary Microbes in People With CF

NCT07312734

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Cystic Fibrosis (CF), +7 more
Locations
  • University of California San Diego La Jolla, California, United States
  • The Minnesota Cystic Fibrosis Center Minneapolis, Minnesota, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • University of Nebraska Medical Center Omaha, Nebraska, United States
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