Actively Recruiting
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)
Led by Intellia Therapeutics · Updated on 2026-04-16
60
Participants Needed
14
Research Sites
56 weeks
Total Duration
On this page
Sponsors
I
Intellia Therapeutics
Lead Sponsor
R
Regeneron Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the safety and effectiveness of a single dose of nexiguran ziclumeran (NTLA-2001) compared to a placebo in people with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This phase 3, multinational, randomized, double-blind, placebo-controlled trial involves about 60 participants with this nerve disease and genetic condition affecting the peripheral nervous system. Participants will be randomly assigned to receive a single intravenous infusion of either nexiguran ziclumeran 55 mg or a placebo of normal saline. To give everyone a chance to receive the study drug, participants may switch to the other treatment group at either 12 or 18 months, depending on specific study criteria. The study is designed to compare the effects of the drug and placebo over time. During the study, participants will be monitored for nerve function using the Modified Neuropathy Impairment Score +7 (mNIS+7) over 18 months and blood levels of serum transthyretin at 29 days and 18 months. Quality of life, body mass index, and other health measures will also be evaluated. The study includes careful safety monitoring and will last up to 18 months with ongoing assessments to track participants' nerve health and overall well-being.
CONDITIONS
Brief 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) 60
You will not qualify if you...
- Other causes of amyloidosis not caused by 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 treatment with TTR silencers (siRNA or antisense oligonucleotides)
- 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 18 months
Participants receive a single infusion of either nexiguran ziclumeran or placebo. Participants may have the option to cross over to the opposite study arm at Month 12 or Month 18 depending on study criteria.
Periodic visits for assessments during treatment
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
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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