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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorderActive, not recruitingPhase 32026-10-08NCT06634420
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia BCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 1 / Phase 22026-10-08NCT06379789
NTLA-2002 in Adults With Hereditary Angioedema (HAE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorderActive, not recruitingPhase 1 / Phase 22026-09-25NCT05120830
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, familial amyloid neuropathy, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficienciesEnrolling by invitationNot stated2026-08-03NCT05697861
A Phase 3 Study of NTLA-2001 in ATTRv-PNAmyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, familial amyloid neuropathy, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, proteostasis deficienciesRecruitingPhase 32026-04-16NCT06672237
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, cardiomyopathy, cardiovascular disorder, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, metabolic disease, proteostasis deficienciesRecruitingPhase 32026-03-27NCT06128629
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorderEnrolling by invitationNot stated2025-11-28NCT06262399