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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.

12,629 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656

Conditions
Atrophy, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations
  • Connecticut Children's Medical Center Farmington, Connecticut, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Clinic for Special Children Strasburg, Pennsylvania, United States
  • St Jude Children's Research Hospital Memphis, Tennessee, United States
View all 42 locations
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Completed

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

NCT04659863

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia - Homozygous, +10 more
Locations
  • Excel Medical Clinical Trials LLC Boca Raton, Florida, United States
  • Novartis Investigative Site Québec, Quebec, Canada
  • Novartis Investigative Site Bron, France
  • University General Hospital of Ioannina Ioannina, GR, Greece
View Trial Details
Completed

Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments

NCT03056755

Conditions
Breast Cancer, Breast Diseases, Breast Neoplasms, +15 more
Locations
  • Banner MD Anderson Cancer Center Gilbert, Arizona, United States
  • Mayo Clinic Arizona Phoenix, Arizona, United States
  • Kaiser Permanente Medical Group Anaheim, California, United States
  • Beverly Hills Cancer Center Beverly Hills, California, United States
View all 94 locations
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Completed

AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa

NCT02556736

Conditions
Advanced Retinitis Pigmentosa, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +6 more
Locations
  • University of California San Francisco - Mission Bay /ID# 235717 San Francisco, California, United States
  • Cincinnati Eye Institute- Edgewood /ID# 236713 Edgewood, Kentucky, United States
  • Duke Eye Center /ID# 235715 Durham, North Carolina, United States
  • Retina Foundation of the Southwest /ID# 235199 Dallas, Texas, United States
View Trial Details
Completed

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

NCT03759379

Conditions
Amyloidosis, Amyloidosis, Familial, Amyloidosis, Hereditary, +8 more
Locations
  • Clinical Trial Site La Mesa, California, United States
  • Clinical Trial Site Aurora, Colorado, United States
  • Clinical Trial Site Jacksonville, Florida, United States
  • Clinical Trial Site Chicago, Illinois, United States
View all 64 locations
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Completed

A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B

NCT05365217

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Beijing Children's Hospital, Capital Medical University Beijing, Beijing Municipality, China
  • Peking Union Medical College Hospital Beijing, Beijing Municipality, China
  • Fujian Medical University Union Hospital-Hematology Fuzhou, Fujian, China
  • Nanfang Hospital, Southern Medical University-Haematology Guangzhou, Guangdong, China
View all 15 locations
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