Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
NCT ID NCT06672237
First seen Nov 01, 2025 · Last updated May 06, 2026 · Updated 23 times
Summary
This study tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a genetic disease that damages nerves. The goal is to see if the treatment can stop or slow the disease by lowering a harmful protein. Participants are randomly assigned to receive the drug or a placebo, and neither they nor their doctors will know which they received.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Chang Gung Medical Foundation - Linkou Branch
ACTIVE_NOT_RECRUITINGTaoyuan, Taiwan
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HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
RECRUITINGRibeirão Preto, Brazil
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Hospital Británico de Buenos Aires
RECRUITINGBuenos Aires, Argentina
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Hospital El Cruce
RECRUITINGSan Juan Bautista, Argentina
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Hospital Italiano de Buenos Aires (HIBA)
RECRUITINGBuenos Aires, Argentina
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Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
RECRUITINGCampinas, Brazil
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Instituto de Educacao, Pesquisa e Gestao em Saude
RECRUITINGRio de Janeiro, Brazil
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National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
RECRUITINGTlalpan, Mexico
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National Taiwan University Hospital
ACTIVE_NOT_RECRUITINGTaipei, Taiwan
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National University Hospital
RECRUITINGSingapore, Singapore
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PSEG Centro de Pesquisa Clinica S.A.
RECRUITINGSão Paulo, Brazil
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Singapore General Hospital
RECRUITINGSingapore, Singapore
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Siriraj Hospital
RECRUITINGBangkok, Thailand
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Taipei Veterans General Hospital
ACTIVE_NOT_RECRUITINGTaipei, Taiwan
Conditions
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