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

Phase 3

Recruiting

• 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

• Registration Number: NCT06672237
• Lead Sponsor: Intellia Therapeutics

Brief 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.

Detailed 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.

Study Timeline

• Study First Submitted: 2024-11-01
• Study First Submitted QC: 2024-11-01
• Study First Posted: 2024-11-04
• Study Start: 2024-11-22
• Status Verified: 2025-07
• Last Update Submitted: 2025-08-28
• Last Update Posted: 2025-09-04
• Primary Completion: 2027-07
• Study Completion: 2028-08-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Diagnosis of ATTRv-PN
• Karnofsky Performance Status (KPS) ≥ 60

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 Antisense oligonucleotides (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

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
nexiguran ziclumeran	nexiguran ziclumeran	nexiguran ziclumeran 55 mg by single IV infusion
Normal Saline	Normal Saline as Placebo	Placebo; Normal saline (0.9% NaCl) by single IV infusion

Primary Outcome Measures

Name	Time	Method
Modified Neuropathy Impairment Score +7 (mNIS+7)	18 Months
Serum TTR	29 Days

Secondary Outcome Measures

Name	Time	Method
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire	18 Months
Modified Body Mass Index (mBMI)	18 Months
Serum TTR	18 Months

Trial Locations

Locations (15)

Hospital Italiano de Buenos Aires (HIBA); 🇦🇷 Buenos Aires, Argentina

Hospital Británico de Buenos Aires; 🇦🇷 Buenos Aires, Argentina

Hospital El Cruce; 🇦🇷 San Juan Bautista, Argentina

Westmead Hospital; 🇦🇺 Westmead, Australia

Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP); 🇧🇷 Campinas, Brazil

HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto; 🇧🇷 Ribeirão Preto, Brazil

Instituto de Educacao, Pesquisa e Gestao em Saude; 🇧🇷 Rio de Janeiro, Brazil

PSEG Centro de Pesquisa Clinica S.A.; 🇧🇷 São Paulo, Brazil

New Zealand Clinical Research; 🇳🇿 Auckland, New Zealand

National University Hospital; 🇸🇬 Singapore, Singapore

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