2 recruiting, 1 completed. 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Recruiting 2 |
| Phase 1 | 1 | Completed 1 |
Follow NTLA-2001: new trials by email Literature Review of NTLA-2001 trials Research Report on NTLA-2001
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
Amyloidosis (3) · Amyloid Neuropathies, Familial (2) · Amyloidosis, Familial (2) · Amyloidosis, Hereditary, Transthyretin-Related (2) · Cardiomyopathies (2) · Polyneuropathies (2) · Amyloid Neuropathies (1) · Genetic Diseases, Inborn (1) · Heredodegenerative Disorders, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1) · Neurodegenerative Diseases (1) · Neuromuscular Diseases (1) · Peripheral Nervous System Diseases (1)
Argentina (2) · Brazil (2) · France (2) · Mexico (2) · New Zealand (2) · Singapore (2) · Sweden (2) · Taiwan (2) · United Kingdom (2) · Australia (1) · Austria (1) · Belgium (1) · Canada (1) · Czechia (1) · Denmark (1)