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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)

NCT04601051 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 72

Last updated 2026-01-30

No results posted yet for this study

Summary

This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM)

Conditions

  • Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
  • Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
  • Wild-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

Sponsors & Collaborators

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
90 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-05
Primary Completion
2025-09-12
Completion
2025-09-12
FDA Drug
Yes

Countries

  • France
  • New Zealand
  • Sweden
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04601051 on ClinicalTrials.gov