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)
Status: Active_not_recruiting
Location: See all (16) locations...
Intervention Type: Biological, Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 85
Healthy Volunteers: f
View:
• Diagnosis of ATTRv-PN
• Karnofsky Performance Status (KPS) ≥ 60
Locations
Other Locations
Argentina
Hospital Británico de Buenos Aires
Buenos Aires
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires
Hospital El Cruce
San Juan Bautista
Australia
Westmead Hospital
Westmead
Brazil
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
Mexico
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
Tlalpan
New Zealand
New Zealand Clinical Research
Auckland
Singapore
National University Hospital
Singapore
Singapore General Hospital
Singapore
Taiwan
National Taiwan University Hospital
Taipei
Taipei Veterans General Hospital
Taipei
Chang Gung Medical Foundation - Linkou Branch
Taoyuan District
Thailand
Siriraj Hospital
Bangkok
Time Frame
Start Date: 2024-11-22
Completion Date: 2028-08
Participants
Target number of participants: 50
Treatments
Experimental: nexiguran ziclumeran
nexiguran ziclumeran 55 mg by single IV infusion
Placebo_comparator: Normal Saline
Placebo; Normal saline (0.9% NaCl) by single IV infusion
Related Therapeutic Areas
Sponsors
Collaborators: Regeneron Pharmaceuticals
Leads: Intellia Therapeutics