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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, beta thalassemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease, thalassemiaActive, not recruitingPhase 22026-08-25NCT05353647
Gene Transfer for Sickle Cell DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, beta thalassemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease, thalassemiaActive, not recruitingPhase 12026-01-09NCT03282656
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Primary Immunodeficiency Diseases, X-Linked Combined Immunodeficiency Diseases, X-linked disease, hereditary disease, immune system disorder, inborn error of immunity, severe combined immunodeficiencyRecruitingPhase 1 / Phase 22025-12-11NCT03311503