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

NCT ID: NCT06672237

Last Updated: 2025-11-26

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-11-22
• Study Completion Date: 2028-08-31

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.

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; Amyloidosis, Familial; Amyloidosis, Hereditary; Amyloidosis; Polyneuropathies; Amyloid Neuropathies; Amyloid Neuropathies, Familial; Peripheral Nervous System Disease; Peripheral Nervous System Diseases; Metabolism, Inborn Errors; Metabolic Diseases

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

nexiguran ziclumeran

nexiguran ziclumeran 55 mg by single IV infusion

Group Type: EXPERIMENTAL

nexiguran ziclumeran

Intervention Type: BIOLOGICAL

nexiguran ziclumeran 55 mg by single IV infusion

Normal Saline

Placebo; Normal saline (0.9% NaCl) by single IV infusion

Group Type: PLACEBO_COMPARATOR

Normal Saline as Placebo

Intervention Type: DRUG

Normal saline (0.9% NaCl) by single IV infusion

Interventions

nexiguran ziclumeran

nexiguran ziclumeran 55 mg by single IV infusion

Intervention Type: BIOLOGICAL

Normal Saline as Placebo

Normal saline (0.9% NaCl) by single IV infusion

Intervention Type: DRUG

Other Intervention Names

NTLA-2001

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

Intellia Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hospital Británico de Buenos Aires

Buenos Aires, , Argentina

Hospital Italiano de Buenos Aires (HIBA)

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

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

Tlalpan, , Mexico

New Zealand Clinical Research

Auckland, , New Zealand

National University Hospital

Singapore, , Singapore

Singapore General Hospital

Singapore, , Singapore

National Taiwan University Hospital

Taipei, , Taiwan

Taipei Veterans General Hospital

Taipei, , Taiwan

Chang Gung Medical Foundation - Linkou Branch

Taoyuan District, , Taiwan

Siriraj Hospital

Bangkok, , Thailand

Countries

Argentina; Australia; Brazil; Mexico; New Zealand; Singapore; Taiwan; Thailand

Other Identifiers

ITL-2001-CL-311

Identifier Type: -

Identifier Source: org_study_id