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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.

12,674 public trials 2,143 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

α1-antitrypsin (AAT) Levels and Functions in Allogeneic Bone Marrow Transplantation and Throughout Progression Into GVHD

NCT03188601

Conditions
Alpha 1-Antitrypsin, Bone Marrow Transplant Failure, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations
  • Hadassah Jerusalem, Center, Israel
  • Tel Aviv Sourasky Medical Center - Ichilov Hospital Tel Aviv, Center, Israel
  • Rambam MC Haifa, North, Israel
  • Soroka Medical Center Beersheba, South, Israel
View Trial Details
Completed

Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)

NCT04285346

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +12 more
Locations
  • Marinus Research Site Los Angeles, California, United States
  • Marinus Research Site Palo Alto, California, United States
  • Marinus Research Site Boston, Massachusetts, United States
  • Marinus Research Site Livingston, New Jersey, United States
View Trial Details
Completed

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

NCT04030598

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Medical Research of Arizona Scottsdale, Arizona, United States
  • University of California San Diego (UCSD) San Diego, California, United States
  • AIRE Medical of Los Angeles Santa Monica, California, United States
  • Midwest Immunology Clinical Plymouth, Minnesota, United States
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Completed

Treatment With the Evoke System for Facial and Submental Laxity

NCT04719013

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, Cutis Laxa, +5 more
Locations
  • Laser & Skin Surgery Medical Group Sacramento, California, United States
  • AboutSkin dermatology Greenwood Village, Colorado, United States
  • Lupo Center for Aesthetic and General Dermatology New Orleans, Louisiana, United States
  • Dallas Plastic Surgery Institute Dallas, Texas, United States
View Trial Details
Completed

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America

NCT03845400

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Clinical Research Center of Alabama Birmingham, Alabama, United States
  • Medical Research of Arizona Scottsdale, Arizona, United States
  • University of California San Diego San Diego, California, United States
  • AIRE Medical of Los Angeles Santa Monica, California, United States
View all 29 locations
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