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NCT06672237Active, not recruitingPhase 3

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

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)

Source: ClinicalTrials.gov · Last updated 5 Nov 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Phase (registry)
Phase 3
Study type
Interventional
Enrollment
67 (actual)
First submitted
1 Nov 2024
Start date
22 Nov 2024
Primary completion date
Jul 2027
Lead sponsor
Intellia Therapeutics
Diseases
Amyloidosis
NCT number
NCT06672237

What you commit to

  • Study type: Interventional · Phase 3
  • Participants enrolled: 67
  • From 22 Nov 2024 to the primary completion date, Jul 2027
  • 14 sites in 6 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))

Other illness

  • Diabetes mellitus
  • New York Heart Association Class III or IV heart failure
  • Liver failure
  • Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
  • History of liver disease

Other

  • Other causes of amyloidosis (amyloidosis caused by non-TTR protein)
  • Other known causes of sensorimotor or autonomic neuropathy
  • Estimated Glomerular Filtration Rate \< 30 mL/min/1.73 m2
  • Unable or unwilling to take vitamin A supplementation for the duration of the study

Who the study is looking for

  • Diagnosis of ATTRv-PN
  • Karnofsky Performance Status (KPS) ≥ 60

Age in the registry: 18 years to 85 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (14)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Hospital Italiano de Buenos Aires (HIBA)
Coordinator of this site? Confirm its status
Buenos AiresArgentinaUnknown status
Hospital Britanico de Buenos Aires
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Buenos AiresArgentinaUnknown status
Hospital El Cruce
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San Juan BautistaArgentinaUnknown status
Hospital das Clinicas da Universidade Estadual de Campinas UNICAMP
Coordinator of this site? Confirm its status
CampinasBrazilUnknown status
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Coordinator of this site? Confirm its status
Ribeirão PretoBrazilUnknown status
Instituto de Educacao Pesquisa e Gestao em Saude
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Rio de JaneiroBrazilUnknown status
PSEG Centro de Pesquisa Clinica S.A.
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São PauloBrazilUnknown status
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)
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TlalpanMexicoUnknown status
Singapore General Hospital
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SingaporeSingaporeUnknown status
National University Hospital
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SingaporeSingaporeUnknown status
Siriraj Hospital
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BangkokThailandUnknown status
National Taiwan University Hospital
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TaipeiTaiwanUnknown status
Taipei Veterans General Hospital
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TaipeiTaiwanUnknown status
Chang Gung Medical Foundation-Linkou Branch
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TaoyuanTaiwanUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.