NCT04601051View on ClinicalTrials.gov

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)

PHASE1Active, not recruitingINTERVENTIONAL
Enrollment

72

Participants

Timeline

Start Date

November 5, 2020

Primary Completion Date

August 31, 2025

Study Completion Date

August 31, 2026

Conditions
Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid CardiomyopathyWild-Type Transthyretin Cardiac Amyloidosis
Interventions
BIOLOGICAL

NTLA-2001

A clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene editing system delivered by lipid nanoparticles (LNPs) for intravenous (IV) administration

Trial Locations (4)

Unknown

Clinical Trial Site, Paris

Clinical Trial Site, Auckland

Clinical Trial Site, Umeå

Clinical Trial Site, London

Sponsors
All Listed Sponsors
lead

Intellia Therapeutics

INDUSTRY

NCT04601051 - 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) | Biotech Hunter | Biotech Hunter