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

Last updated: November 24, 2025
Sponsor: Intellia Therapeutics
Overall Status: Active - Not Recruiting

Phase

3

Condition

Myasthenia Gravis (Chronic Weakness)

Multiple Sclerosis

Amyotrophic Lateral Sclerosis (Als)

Treatment

Normal Saline as Placebo

nexiguran ziclumeran

Clinical Study ID

NCT06672237
ITL-2001-CL-311
  • Ages 18-85
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Diagnosis of ATTRv-PN

  • Karnofsky Performance Status (KPS) ≥ 60

Exclusion

Exclusion Criteria:

  • Other causes of amyloidosis (amyloidosis caused by non-TTR protein)

  • Other known causes of sensorimotor or autonomic neuropathy

  • Diabetes mellitus

  • New York Heart Association Class III or IV heart failure

  • Liver failure

  • Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection

  • Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisenseoligonucleotides (ASOs))

  • Estimated Glomerular Filtration Rate < 30 mL/min/1.73 m2

  • Unable or unwilling to take vitamin A supplementation for the duration of the study

Study Design

Total Participants: 50
Treatment Group(s): 2
Primary Treatment: Normal Saline as Placebo
Phase: 3
Study Start date:
November 22, 2024
Estimated Completion Date:
August 31, 2028

Study Description

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 50 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.

Connect with a study center

  • Hospital Británico de Buenos Aires

    Buenos Aires,
    Argentina

    Site Not Available

  • Hospital Británico de Buenos Aires

    Buenos Aires 3435910,
    Argentina

    Site Not Available

  • Hospital Italiano de Buenos Aires (HIBA)

    Buenos Aires 3435910,
    Argentina

    Site Not Available

  • Hospital El Cruce

    Florencio Varela,
    Argentina

    Site Not Available

  • Hospital El Cruce

    San Juan Bautista 3433923,
    Argentina

    Site Not Available

  • Westmead Hospital

    Westmead,
    Australia

    Site Not Available

  • Westmead Hospital

    Westmead 2143973,
    Australia

    Site Not Available

  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)

    Campinas 3467865,
    Brazil

    Site Not Available

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    Ribeirao Preto,
    Brazil

    Site Not Available

  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)

    Ribeirão Preto,
    Brazil

    Site Not Available

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    Ribeirão Preto 3451328,
    Brazil

    Site Not Available

  • Instituto de Educacao, Pesquisa e Gestao em Saude

    Rio de Janeiro,
    Brazil

    Site Not Available

  • Instituto de Educacao, Pesquisa e Gestao em Saude

    Rio de Janeiro 3451190,
    Brazil

    Site Not Available

  • PSEG Centro de Pesquisa Clinica S.A.

    Sao Paulo,
    Brazil

    Site Not Available

  • PSEG Centro de Pesquisa Clinica S.A.

    São Paulo 3448439,
    Brazil

    Site Not Available

  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)

    Tlalpan 3515428,
    Mexico

    Site Not Available

  • New Zealand Clinical Research

    Auckland,
    New Zealand

    Site Not Available

  • New Zealand Clinical Research

    Auckland 2193733,
    New Zealand

    Site Not Available

  • National University Hospital

    Singapore,
    Singapore

    Active - Recruiting

  • Singapore General Hospital

    Singapore,
    Singapore

    Site Not Available

  • National University Hospital

    Singapore 1880252,
    Singapore

    Site Not Available

  • Singapore General Hospital

    Singapore 1880252,
    Singapore

    Site Not Available

  • National Taiwan University Hospital

    Taipei,
    Taiwan

    Active - Recruiting

  • Taipei Veterans General Hospital

    Taipei,
    Taiwan

    Site Not Available

  • National Taiwan University Hospital

    Taipei 1668341,
    Taiwan

    Site Not Available

  • Taipei Veterans General Hospital

    Taipei 1668341,
    Taiwan

    Site Not Available

  • Chang Gung Medical Foundation - Linkou Branch

    Taoyuan,
    Taiwan

    Site Not Available

  • Chang Gung Medical Foundation - Linkou Branch

    Taoyuan District 1667905,
    Taiwan

    Site Not Available

  • Siriraj Hospital

    Bangkok,
    Thailand

    Site Not Available

  • Siriraj Hospital

    Bangkok 1609350,
    Thailand

    Site Not Available

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