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New shot every 6 months could slow rare nerve disease

NCT ID NCT07223203

Disease control Sponsor: Alnylam Pharmaceuticals 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 16, 2026 · Updated 6 times

Summary

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to an existing treatment and a placebo group from a previous study.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Nucresiran (a drug given as an injection under the skin every 6 months)
What this could lead to
If successful, nucresiran could offer a more convenient treatment option for people with hereditary ATTR amyloidosis with polyneuropathy, potentially slowing nerve damage and improving quality of life.
What could go wrong
This is an early-stage Phase 3 trial with only 125 participants, so results may not apply to everyone. The drug may not prove superior to the existing treatment vutrisiran, and side effects are still being studied.

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 125 people

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

Started

Feb 2026

Expected to finish

Jun 2031

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: * Has documented diagnosis of hATTR-PN * Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant * Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive) * Has a Karnofsky Performance Status (KPS) of ≥60% Exclusion Criteria: * Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study * Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis * Has a New York Heart Association (NYHA) heart failure classification \>2 * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2.5 upper limit of normal (ULN) * Has total bilirubin \>1.5 ULN * Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m\^2 * Has other known causes of sensorimotor or autonomic neuropathy

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Conditions

The condition(s) this trial relates to.

amyloidosis familial amyloid neuropathy polyneuropathy

As listed by the trial registrant

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

Hattr-PN Hereditary transthyretin-mediated amyloidosis with polyneuropathy

How to take part

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

  1. The places running it

    56 sites in 21 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Clinical Trial Site

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Clinical Trial Site

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Clinical Trial Site

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • Clinical Trial Site

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    Boston, Massachusetts, 02118, United States

  • Clinical Trial Site

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Clinical Trial Site

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    New York, New York, 10016, United States

  • Clinical Trial Site

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    New York, New York, 10032, United States

  • Clinical Trial Site

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    Chapel Hill, North Carolina, 27514, United States

  • Clinical Trial Site

    RECRUITING

    Nashville, Tennessee, 37232, United States

  • Clinical Trial Site

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    Dallas, Texas, 75246, United States

  • Clinical Trial Site

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    Houston, Texas, 77030, United States

  • Clinical Trial Site

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    Rosario, CP2000, Argentina

  • Clinical Trial Site

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    Darlinghurst, 2010, Australia

  • Clinical Trial Site

    RECRUITING

    Woolloongabba, 4102, Australia

  • Clinical Trial Site

    RECRUITING

    Ribeirão Preto, 14051-140, Brazil

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Rio de Janeiro, 22640-100, Brazil

  • Clinical Trial Site

    RECRUITING

    São Paulo, 04038-002, Brazil

  • Clinical Trial Site

    RECRUITING

    Vancouver, V5Z 1M9, Canada

  • Clinical Trial Site

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    Égkomi, 2371, Cyprus

  • Clinical Trial Site

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    Créteil, 94000, France

  • Clinical Trial Site

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    Le Kremlin-Bicêtre, 94270, France

  • Clinical Trial Site

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    Marseille, 13005, France

  • Clinical Trial Site

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    Berlin, 10117, Germany

  • Clinical Trial Site

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    Heidelberg, 69120, Germany

  • Clinical Trial Site

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    Münster, 48149, Germany

  • Clinical Trial Site

    RECRUITING

    Athens, 115 28, Greece

  • Clinical Trial Site

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    Heraklion, 715 00, Greece

  • Clinical Trial Site

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    Dublin, D24 NR0A, Ireland

  • Clinical Trial Site

    RECRUITING

    Florence, 50134, Italy

  • Clinical Trial Site

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    Milan, 20133, Italy

  • Clinical Trial Site

    RECRUITING

    Padova, 35128, Italy

  • Clinical Trial Site

    RECRUITING

    Pavia, 27100, Italy

  • Clinical Trial Site

    RECRUITING

    Roma, 00168, Italy

  • Clinical Trial Site

    RECRUITING

    Bunkyo C, 113-8519, Japan

  • Clinical Trial Site

    RECRUITING

    Kumamoto, 860-8556, Japan

  • Clinical Trial Site

    RECRUITING

    Matsumoto-shi, 390-8621, Japan

  • Clinical Trial Site

    RECRUITING

    Suita, 565-0871, Japan

  • Clinical Trial Site

    RECRUITING

    Kuala Lumpur, 59100, Malaysia

  • Clinical Trial Site

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    Lisbon, 1649-035, Portugal

  • Clinical Trial Site

    RECRUITING

    Porto, 4099-001, Portugal

  • Clinical Trial Site

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    Seoul, 05030, South Korea

  • Clinical Trial Site

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    Seoul, 06351, South Korea

  • Clinical Trial Site

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    Barcelona, 08035, Spain

  • Clinical Trial Site

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    Huelva, 21005, Spain

  • Clinical Trial Site

    RECRUITING

    L'Hospitalet de Llobregat, 8907, Spain

  • Clinical Trial Site

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    Madrid, 28040, Spain

  • Clinical Trial Site

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    Palma de Mallorca, 07198, Spain

  • Clinical Trial Site

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    Stockholm, 113 61, Sweden

  • Clinical Trial Site

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    Umeå, 907 37, Sweden

  • Clinical Trial Site

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    Bern, 3010, Switzerland

  • Clinical Trial Site

    RECRUITING

    Taipei, 10002, Taiwan

  • Clinical Trial Site

    RECRUITING

    Taipei, 112, Taiwan

  • Clinical Trial Site

    RECRUITING

    Taoyuan City, 33305, Taiwan

  • Clinical Trial Site

    RECRUITING

    Istanbul, 34093, Turkey (Türkiye)

  • Clinical Trial Site

    RECRUITING

    Izmir, 35330, Turkey (Türkiye)

  • Clinical Trial Site

    RECRUITING

    London, NW3 2QG, United Kingdom