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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Study of SHP655 (rADAMTS13) in Sickle Cell Disease

NCT03997760

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of Colorado Sickle Cell Treatment and Research Center Aurora, Colorado, United States
  • Sickle Cell Center Denver, Colorado, United States
View all 14 locations
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Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

NCT01960348

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +16 more
Locations
  • Clinical Trial Site La Mesa, California, United States
  • Clinical Trial Site Orange, California, United States
  • Clinical Trial Site Denver, Colorado, United States
  • Clinical Trial Site Chicago, Illinois, United States
View all 52 locations
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Completed

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

NCT03338816

Conditions
ALA Dehydratase Deficient Porphyria (ADP), Acute Hepatic Porphyria, Acute Intermittent Porphyria, +17 more
Locations
  • Clinical Trial Site Little Rock, Arkansas, United States
  • Clinical Trial Site San Francisco, California, United States
  • Clinical Trial Site Boston, Massachusetts, United States
  • Clinical Trial Site Ann Arbor, Michigan, United States
View all 36 locations
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Completed

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

NCT03862807

Conditions
Amyloidosis, Amyloidosis, Familial, Amyloidosis, Hereditary, Transthyretin-Related, +7 more
Locations
  • Clinical Trial Site Créteil, France
  • Clinical Trial Site Le Kremlin-Bicêtre, France
  • Clinical Trial Site Münster, Germany
  • Clinical Trial Site Messina, Italy
View Trial Details
Completed

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

NCT03537651

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Providence Alaska Medical Center Anchorage, Alaska, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
View all 55 locations
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Completed

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

NCT03714373

Conditions
Abnormalities, Multiple, Chromosome Disorders, Congenital Abnormalities, +13 more
Locations
  • University of California, Irvine Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • Rady Children's Hospital San Diego San Diego, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 28 locations
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