# 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** · PHASE1 · COMPLETED · sponsor: **Intellia Therapeutics** · enrollment: 72 (actual)

## Conditions studied

- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
- Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Wild-Type Transthyretin Cardiac Amyloidosis

## Interventions

- **BIOLOGICAL:** NTLA-2001

## Key facts

- **NCT ID:** NCT04601051
- **Lead sponsor:** Intellia Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2020-11-05
- **Primary completion:** 2025-09-12
- **Final completion:** 2025-09-12
- **Target enrollment:** 72 (ACTUAL)
- **Last updated:** 2026-01-30


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04601051

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration 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)". Retrieved via AI Analytics 2026-07-18 from https://api.ai-analytics.org/clinical/NCT04601051. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*