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.
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
50
nexiguran ziclumeran 55 mg by single IV infusion
Normal saline (0.9% NaCl) by single IV infusion
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
...and 6 more locations
Modified Neuropathy Impairment Score +7 (mNIS+7)
Time frame: 18 Months
Serum TTR
Time frame: 29 Days
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
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