3 recruiting, 2 active not recruiting, 2 enrolling by invitation, 1 completed. 5 Phase 1, 4 Phase 2, 3 Phase 3.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) NTLA-2002, Normal Saline IV Administration - Hereditary Angioedema | Phase 3 | Active not recruiting | Intellia Therapeutics | 80 | 2025-01-15 | 2026-10-08 |
| A Phase 3 Study of NTLA-2001 in ATTRv-PN nexiguran ziclumeran, Normal Saline as Placebo - Neuromuscular Disease, Neuromuscular Diseases (NMD) | Phase 3 | Recruiting | Intellia Therapeutics | 60 | 2024-11-22 | 2026-04-16 |
| NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease Biological NTLA-3001 - Lung Disease, Pulmonary Disease | Phase 1 / Phase 2 | Withdrawn | Intellia Therapeutics | 2024-11-20 | 2025-01-17 | |
| 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 B REGV131, LNP1265 - Hemophilia B | Phase 1 / Phase 2 | Recruiting | Regeneron Pharmaceuticals | 130 | 2024-09-11 | 2026-10-21 |
| Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002 Hereditary Angioedema | Enrolling by invitation | Intellia Therapeutics | 100 | 2024-04-12 | 2025-11-28 | |
| MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) NTLA-2001, Placebo - Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Phase 3 | Recruiting | Intellia Therapeutics | 1200 | 2023-12-13 | 2026-03-27 |
| Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001 Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Enrolling by invitation | Intellia Therapeutics | 72 | 2023-07-10 | 2026-08-03 | |
| Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia Arm 1: NTLA-5001, Arm 2: NTLA-5001 - Acute Myeloid Leukemia | Phase 1 / Phase 2 | Terminated | Intellia Therapeutics | 6 | 2021-12-17 | 2023-12-28 |
| NTLA-2002 in Adults With Hereditary Angioedema (HAE) Biological NTLA-2002, Normal Saline IV Administration - Hereditary Angioedema | Phase 1 / Phase 2 | Active not recruiting | Intellia Therapeutics | 37 | 2021-12-10 | 2026-09-25 |
| Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) NTLA-2001 - Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Phase 1 | Completed | Intellia Therapeutics | 72 | 2020-11-05 | 2026-01-30 |
Amyloid Neuropathies, Familial (3) · Amyloidosis (3) · Angioedemas, Hereditary (3) · Amyloidosis, Familial (2) · Amyloidosis, Hereditary, Transthyretin-Related (2) · Cardiomyopathies (2) · Polyneuropathies (2) · Amyloid Neuropathies (1) · Genetic Diseases, Inborn (1) · Hemophilia B (1) · Heredodegenerative Disorders, Nervous System (1) · Immune System Diseases (1) · Immunoproliferative Disorders (1) · Leukemia (1) · Leukemia, Myeloid, Acute (1)
NTLA-2001 (3) · Saline Solution (1)
United Kingdom (8) · France (7) · New Zealand (7) · Australia (5) · Germany (5) · Netherlands (4) · United States (4) · Brazil (3) · Canada (3) · Sweden (3) · Argentina (2) · Italy (2) · Mexico (2) · Singapore (2) · Spain (2)
PMID 34175043, PMID 34215024, PMID 38294975, PMID 39445704, PMID 39555828, PMID 41002250, PMID 42294842
2023 (1)