Actively Recruiting
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Led by Intellia Therapeutics · Updated on 2026-04-16
60
Participants Needed
14
Research Sites
192 weeks
Total Duration
On this page
Sponsors
I
Intellia Therapeutics
Lead Sponsor
R
Regeneron Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) 260
You will not qualify if you...
- 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 HIV infection
- Prior use of TTR silencer (siRNA or ASOs)
- Estimated Glomerular Filtration Rate less than 30 mL/min/1.73 m2
- Unable or unwilling to take vitamin A supplements during the study
- History of liver disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
Hospital Británico de Buenos Aires
Buenos Aires, Argentina
Actively Recruiting
2
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires, Argentina
Actively Recruiting
3
Hospital El Cruce
San Juan Bautista, Argentina
Actively Recruiting
4
Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
Campinas, Brazil
Actively Recruiting
5
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, Brazil
Actively Recruiting
6
Instituto de Educacao, Pesquisa e Gestao em Saude
Rio de Janeiro, Brazil
Actively Recruiting
7
PSEG Centro de Pesquisa Clinica S.A.
São Paulo, Brazil
Actively Recruiting
8
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
Tlalpan, Mexico
Actively Recruiting
9
National University Hospital
Singapore, Singapore
Actively Recruiting
10
Singapore General Hospital
Singapore, Singapore
Actively Recruiting
11
National Taiwan University Hospital
Taipei, Taiwan
Active, Not Recruiting
12
Taipei Veterans General Hospital
Taipei, Taiwan
Active, Not Recruiting
13
Chang Gung Medical Foundation - Linkou Branch
Taoyuan, Taiwan
Active, Not Recruiting
14
Siriraj Hospital
Bangkok, Thailand
Actively Recruiting
Research Team
T
Trial Manager at Intellia
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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