Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Gene-Editing shot aims to halt rare nerve disease in phase 3 trial

NCT ID NCT06672237

Disease control Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2024

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 85 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of ATTRv-PN * Karnofsky Performance Status (KPS) ≥ 60 Exclusion Criteria: * Other causes of amyloidosis (amyloidosis caused by non-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 human immunodeficiency virus (HIV) infection * Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs)) * Estimated Glomerular Filtration Rate \< 30 mL/min/1.73 m2 * Unable or unwilling to take vitamin A supplementation for the duration of the study * History of liver disease

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Amyloid neuropathies are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Amyloid Neuropathies Amyloid Neuropathies, Familial amyloidosis familial amyloid neuropathy hereditary amyloidosis hereditary disease inborn errors of metabolism inherited neurodegenerative disorder metabolic disease nervous system disorder neurodegenerative disease neuromuscular disease peripheral nervous system disorder polyneuropathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyloid neuropathies Amyloid neuropathies, familial Amyloidosis Amyloidosis, familial Amyloidosis, hereditary Genetic disease, inborn Metabolic diseases Metabolism, inborn errors Nerve disorders Nervous system disease Nervous system diseases Neurodegenerative disease Neurodegenerative disease, hereditary Neurodegenerative diseases Neuromuscular disease Neuromuscular diseases Neuromuscular diseases (NMD) Peripheral nervous system disease Peripheral nervous system diseases Polyneuropathies

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    11 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Chang Gung Medical Foundation - Linkou Branch

    ACTIVE_NOT_RECRUITING

    Taoyuan, Taiwan

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    RECRUITING

    Ribeirão Preto, Brazil

  • Hospital Británico de Buenos Aires

    RECRUITING

    Buenos Aires, Argentina

  • Hospital El Cruce

    RECRUITING

    San Juan Bautista, Argentina

  • Hospital Italiano de Buenos Aires (HIBA)

    RECRUITING

    Buenos Aires, Argentina

  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)

    RECRUITING

    Campinas, Brazil

  • Instituto de Educacao, Pesquisa e Gestao em Saude

    RECRUITING

    Rio de Janeiro, Brazil

  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)

    RECRUITING

    Tlalpan, Mexico

  • National Taiwan University Hospital

    ACTIVE_NOT_RECRUITING

    Taipei, Taiwan

  • National University Hospital

    RECRUITING

    Singapore, Singapore

  • PSEG Centro de Pesquisa Clinica S.A.

    RECRUITING

    São Paulo, Brazil

  • Singapore General Hospital

    RECRUITING

    Singapore, Singapore

  • Siriraj Hospital

    RECRUITING

    Bangkok, Thailand

  • Taipei Veterans General Hospital

    ACTIVE_NOT_RECRUITING

    Taipei, Taiwan

More trials for these conditions

Other studies related to the condition(s) this trial covers.