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Recruiting NCT06672237

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

Phase III Interventional Neuromuscular Disease Neuromuscular Diseases (NMD) Neurodegenerative Disease Neurodegenerative Disease, Hereditary

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: nexiguran ziclumeran, Normal Saline as Placebo.
Who it may be relevant to
Registry conditions: Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary. Basic parameters: 18 years — 85 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Argentina, Brazil, Mexico, Singapore, Taiwan +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Overview

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 60 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.

Interventions

  • Biological nexiguran ziclumeran
    nexiguran ziclumeran 55 mg by single IV infusion
  • Drug Normal Saline as Placebo
    Normal saline (0.9% NaCl) by single IV infusion

Primary outcome measures

  • Modified Neuropathy Impairment Score +7 (mNIS+7) [Time frame: 18 Months]
  • Serum TTR [Time frame: 29 Days]
Secondary outcome measures (3)
  • Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire [Time frame: 18 Months]
  • Modified Body Mass Index (mBMI) [Time frame: 18 Months]
  • Serum TTR [Time frame: 18 Months]

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
  • History of liver disease

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Brazil · 4 centers
  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP) — Campinas
  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto — Ribeirão Preto
  • Instituto de Educacao, Pesquisa e Gestao em Saude — Rio de Janeiro
  • PSEG Centro de Pesquisa Clinica S.A. — São Paulo
Argentina · 3 centers
  • Hospital Británico de Buenos Aires — Buenos Aires
  • Hospital Italiano de Buenos Aires (HIBA) — Buenos Aires
  • Hospital El Cruce — San Juan Bautista
Taiwan · 3 centers
  • National Taiwan University Hospital — Taipei
  • Taipei Veterans General Hospital — Taipei
  • Chang Gung Medical Foundation - Linkou Branch — Taoyuan
Singapore · 2 centers
  • National University Hospital — Singapore
  • Singapore General Hospital — Singapore
Mexico · 1 center
  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ) — Tlalpan
Thailand · 1 center
  • Siriraj Hospital — Bangkok

Identifiers

NCT: NCT06672237 · ITL-2001-CL-311

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗