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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.

12,633 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

An Asian Study to Evaluate Efficacy and Safety of Oral Enzalutamide in Progressive Metastatic Prostate Cancer Participants

NCT02294461

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +20 more
Locations
  • CN00103 Beijing, China
  • CN00104 Beijing, China
  • CN00106 Beijing, China
  • CN00111 Beijing, China
View all 47 locations
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Completed

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
  • Montreal Children's Hospital Research Institute - McGill University Montreal, Quebec, Canada
View Trial Details
Completed

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Centre Phoenix, Arizona, United States
  • University of California Irvine Orange, California, United States
  • University of Florida Gainesville, Florida, United States
  • Emory University Division of Medical Genetics Decatur, Georgia, United States
View all 19 locations
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Completed

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

NCT03257462

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • Spruce Biosciences Clinical Site Orange, California, United States
  • Spruce Biosciences Clinical Site San Diego, California, United States
  • Spruce Biosciences Clinical Site Melbourne, Florida, United States
  • Spruce Biosciences Clinical Site Atlanta, Georgia, United States
View Trial Details
Completed

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • UCLA Medical Center Site Number : 1003 Santa Monica, California, United States
  • Emory University School of Medicine Site Number : 1005 Atlanta, Georgia, United States
  • Infusion Associates Site Number : 1001 Grand Rapids, Michigan, United States
  • Metropolitan Hospital Center Site Number : 1004 New York, United States
View Trial Details
Completed

A Study in Patients With Atopic Eczema to Test How Effective BI 655130 is and How Well it is Tolerated

NCT03822832

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis, Atopic, +8 more
Locations
  • Center for Dermatology and Plastic Surgery Scottsdale, Arizona, United States
  • Clinical Physiology Associates Fort Myers, Florida, United States
  • Finlay Medical Research Corp Miami, Florida, United States
  • University of South Florida Tampa, Florida, United States
View all 23 locations
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Completed

Infusion Duration Study To Assess Tolerability of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate

NCT04511702

Conditions
Arthritis, Chronic Uncontrolled Gout, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Orthopedic Physicians Alaska Anchorage, Alaska, United States
  • Arizona Arthritis & Rheumatology Research Gilbert, Arizona, United States
  • Arthritis & Rheumatology Research Mesa, Arizona, United States
View all 39 locations
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Completed

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

NCT03474965

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +19 more
Locations
  • University Of Alabama Birmingham, Alabama, United States
  • Childrens National Hospital Washington D.C., District of Columbia, United States
  • University of Florida Gainesville, Florida, United States
  • Joe DiMaggio Childrens Hospital Hollywood, Florida, United States
View all 35 locations
View Trial Details

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