Skip to main content
OpenTrials

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
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Active, not recruiting

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

NCT03992430

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of Florida Gainesville, Florida, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Hospital Universitario San Ignacio Bogotá, Colombia
View all 59 locations
View Trial Details
Active, not recruiting

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

NCT05768854

Conditions
Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +7 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Childrens Hospital LA Los Angeles, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Yale New Haven Hospital New Haven, Connecticut, United States
View all 20 locations
View Trial Details
Active, not recruiting

Study in Paediatric Patients With Atopic Dermatitis Treated With Dupilumab in Spain

NCT06415175

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Investigational Site Number : 7240004 Santiago de Compostela, A Coruña [La Coruña], Spain
  • Investigational Site Number : 7240006 Esplugues de Llobregat, Barcelona [Barcelona], Spain
  • Investigational Site Number : 7240005 Oviedo, Principality of Asturias, Spain
  • Investigational Site Number : 7240008 Seville, Sevilla, Spain
View all 15 locations
View Trial Details
Active, not recruiting

Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

NCT03924401

Conditions
Anemia, Anemia, Aplastic, Anemia, Hemolytic, +19 more
Locations
  • Children's of Alabama Birmingham, Alabama, United States
  • Nemours/Alfred I. DuPont Hospital for Children Wilmington, Delaware, United States
  • Childrens Healthcare of Atlanta Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View Trial Details
Active, not recruiting

Setrusumab vs Placebo for Osteogenesis Imperfecta

NCT05125809

Conditions
Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +7 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Shriners Hospital for Children - Northern California Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 44 locations
View Trial Details
Active, not recruiting

Safety and Efficacy of Upadacitinib in Combination With Topical Corticosteroids in Children From 2 to Less Than 12 Years of Age in Japan With Moderate to Severe Atopic Dermatitis

NCT06701331

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Central Clinic - Nagoya /ID# 269205 Nagoya, Aichi-ken, Japan
  • Fujita Health University Hospital /ID# 269201 Toyoake, Aichi-ken, Japan
  • Miyata Dermatology Clinic /ID# 269200 Matsudo-Shi, Chiba, Japan
  • Hoshikuma Dermatology ・ Allergy Clinic /ID# 270192 Fukuoka, Japan
View all 25 locations
View Trial Details
Active, not recruiting

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

NCT06832371

Conditions
Arterial Occlusive Diseases, Arteriosclerosis, Atherosclerosis, +18 more
Locations
  • CHRU Lille - Centre Hospitalier Universitaire de Lille Lille, France
  • Centre Hospitalier Universitaire de Lyon (CHU Lyon) Lyon, France
  • Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM) Marseille, France
  • Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix" Paris, France
View all 30 locations
View Trial Details

Related conditions