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

Last updated: March 25, 2025
Sponsor: Intellia Therapeutics
Overall Status: Active - Recruiting

Phase

3

Condition

Multiple Sclerosis

Amyotrophic Lateral Sclerosis (Als)

Metabolic Disorders

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

  • Westmead Hospital

    Westmead,
    Australia

    Active - Recruiting

  • PSEG Centro de Pesquisa Clinica S.A.

    Sao Paulo,
    Brazil

    Active - Recruiting

  • New Zealand Clinical Research

    Auckland,
    New Zealand

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei,
    Taiwan

    Active - Recruiting

  • Taipei Veterans General Hospital

    Taipei,
    Taiwan

    Active - Recruiting

  • Chang Gung Medical Foundation - Linkou Branch

    Taoyuan,
    Taiwan

    Active - Recruiting

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