Home
Conditions
Genetic Diseases, Inborn

Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,613 public trials 2,162 recruiting studies

Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Active, not recruiting

PDE6A Gene Therapy for Retinitis Pigmentosa

NCT04611503

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations: Universitätsklinikum Tübingen, Department für Augenheilkunde Tübingen, Germany

View Trial Details

Active, not recruiting

Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)

NCT06300476

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +6 more
Locations: West China Hospital Chengdu, Sichuan, China

View Trial Details

Active, not recruiting

Physical Abilities of Teenagers With Familial Mediterranean Fever

NCT06257342

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Familial Mediterranean Fever, Genetic Diseases, Inborn, +1 more
Locations: CH de Versailles Le Chesnay, France

View Trial Details

Active, not recruiting

Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

NCT03963154

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations: •Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts Paris, France

View Trial Details

Active, not recruiting

Stockholm hyperTRIglyceridemia REGister (STRIREG) Study

NCT06104943

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypertriglyceridemia, +10 more
Locations: Not specified

View Trial Details

Active, not recruiting

Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

NCT00555555

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +12 more
Locations: Not specified

View Trial Details

Active, not recruiting

Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate

NCT00319228

Conditions: Antithrombin III Deficiency, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +10 more
Locations: Not specified

View Trial Details

Active, not recruiting

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

NCT04798235

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Queen's University/Kingston Health Sciences Centre Kingston, Ontario, Canada

View Trial Details

Active, not recruiting

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

NCT04687020

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: UC Davis Sacramento, California, United States

Lurie Children's Hospital Chicago, Illinois, United States

Duke University Medical Center Durham, North Carolina, United States

Children's Hospital of Richmond at VCU Richmond, Virginia, United States

View Trial Details

Active, not recruiting

NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia

NCT04870866

Conditions: Ataxia, Ataxia Telangiectasia, Brain Diseases, +19 more
Locations: Hilde Loge Nilsen Lørenskog, Norway

Oslo University Hospital Oslo, Norway

View Trial Details

Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

Related conditions