# A Phase 3 Study of NTLA-2001 in ATTRv-PN

Clinical trial · Intellia Therapeutics

- Archive id: nct:NCT06672237
- Published: 2024-11-04
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT06672237
- Source URL: https://clinicaltrials.gov/study/NCT06672237
- Provenance: clinicaltrials

## Abstract

This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.

_Abstract reproduced under ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters._

## Trial details

- status: RECRUITING
- phase: PHASE3
- study type: INTERVENTIONAL
- enrollment: 60
- sponsor: Intellia Therapeutics
- conditions: Neuromuscular Disease; Neuromuscular Diseases (NMD); Neurodegenerative Disease; Neurodegenerative Disease, Hereditary; Neurodegenerative Diseases; Neuromuscular Diseases; Nerve Disorders; Nervous System Disease; Nervous System Diseases; Genetic Disease, Inborn
- interventions: BIOLOGICAL: nexiguran ziclumeran; DRUG: Normal Saline as Placebo
- start date: 2024-11-22
- start date type: ACTUAL
- primary completion date: 2027-07-01
- has results: false
- data source: ClinicalTrials.gov
- data processed: 2026-10-29T09:00:05
- modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

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