NTLA-2001
Sponsors
Intellia Therapeutics Inc., Intellia Therapeutics
Conditions
-Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)
-Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)Transthyretin Amyloidosis (ATTR) With CardiomyopathyTransthyretin Amyloidosis with CardiomyopathyTransthyretin-Related (ATTR) Familial Amyloid CardiomyopathyTransthyretin-Related (ATTR) Familial Amyloid PolyneuropathyWild-Type Transthyretin Cardiac Amyloidosis
Phase 1
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)
CompletedNCT04601051
Start: 2020-11-05End: 2025-09-12Updated: 2026-01-30
Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN).
CompletedCTIS2024-511170-69-00
Start: 2021-08-17End: 2025-04-09Target: 15Updated: 2025-07-11
Phase 2
Phase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
RecruitingNCT06128629
Start: 2023-12-13End: 2028-04-01Target: 1200Updated: 2026-03-27
MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double‑Blind, Placebo‑Controlled Study to Evaluate the Efficacy and Safety of NTLA‑2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR‑CM).
SuspendedCTIS2023-507220-23-00
Start: 2024-07-26Target: 362Updated: 2026-01-27