Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
NCT ID NCT06672237
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 32 times
Summary
This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatment or a placebo infusion. The study measures changes in nerve function and quality of life over time to see if the therapy can slow or stop disease progression.
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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.
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
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nexiguran ziclumeran (NTLA-2001), a gene-editing therapy given as a single IV infusion
What this could lead to
If it works, this could be a one-time treatment that stops or slows nerve damage in people with this rare, inherited disease.
What could go wrong
This is still an early-phase 3 trial with only 60 participants. The placebo group means some won't get the real treatment. Long-term safety and effectiveness are not yet known.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.