One-Shot gene therapy aims to halt rare, devastating nerve disease
NCT ID NCT06672237
Summary
This Phase 3 trial is testing whether a single dose of an experimental gene therapy called NTLA-2001 can safely slow or stop the progression of hereditary amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. About 50 participants worldwide will be randomly assigned to receive either the therapy or a placebo infusion. The main goal is to see if the treatment reduces nerve damage and lowers levels of the harmful protein that causes the disease.
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Contacts and locations
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Locations
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Chang Gung Medical Foundation - Linkou Branch
Taoyuan District, Taiwan
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HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, Brazil
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Hospital Británico de Buenos Aires
Buenos Aires, Argentina
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Hospital El Cruce
San Juan Bautista, Argentina
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Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires, Argentina
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Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
Campinas, Brazil
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Instituto de Educacao, Pesquisa e Gestao em Saude
Rio de Janeiro, Brazil
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National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
Tlalpan, Mexico
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National Taiwan University Hospital
Taipei, Taiwan
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National University Hospital
Singapore, Singapore
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New Zealand Clinical Research
Auckland, New Zealand
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PSEG Centro de Pesquisa Clinica S.A.
São Paulo, Brazil
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Singapore General Hospital
Singapore, Singapore
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Siriraj Hospital
Bangkok, Thailand
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Taipei Veterans General Hospital
Taipei, Taiwan
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Westmead Hospital
Westmead, Australia
Conditions
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