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New shot every 3 months could slow rare nerve disease

NCT ID NCT03759379

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new drug called vutrisiran in 164 people with a rare inherited disease that damages nerves and organs. Participants received either vutrisiran as a shot every 3 months or the standard treatment patisiran as an IV infusion every 3 weeks. The goal was to see if vutrisiran could slow nerve damage and improve quality of life compared to a placebo group from an earlier study.

What this could mean

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

Active substance
vutrisiran (also known as AMVUTTRA)
What this could lead to
If it works, this could provide a more convenient treatment option for hATTR amyloidosis, given as a shot every 3 months instead of an IV infusion every 3 weeks.
What could go wrong
This is a completed Phase 3 trial, but it used an external placebo group from an earlier study, which may introduce bias. The drug may not work for everyone and can cause side effects like infusion reactions or liver issues.

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

164 people

The number who actually took part.

Started

Feb 2019

Finished

Nov 2025

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: * Male or female of 18 to 85 years of age (inclusive); * Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation; * Has adequate neurologic impairment score (NIS); * Has adequate polyneuropathy disability (PND) score; * Has adequate Karnofsky Performance Status (KPS). Exclusion Criteria: * Had a prior liver transplant or is likely to undergo liver transplantation during the study; * Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis; * Has New York Heart Association heart failure classification \>2; * Clinically significant liver function test abnormalities; * Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection; * Received an experimental drug within 30 days of dosing; * Received prior TTR-lowering treatment; * Has other known causes of neuropathy.

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Conditions

The condition(s) this trial relates to.

familial amyloid neuropathy hereditary amyloidosis

As listed by the trial registrant

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

Amyloidosis, hereditary Transthyretin amyloidosis

Contacts and locations

Locations

  • Clinical Trial Site

    La Mesa, California, 91942, United States

  • Clinical Trial Site

    Aurora, Colorado, 80045, United States

  • Clinical Trial Site

    Jacksonville, Florida, 32224, United States

  • Clinical Trial Site

    Chicago, Illinois, 60611, United States

  • Clinical Trial Site

    Fairway, Kansas, 66205, United States

  • Clinical Trial Site

    Baltimore, Maryland, 21224, United States

  • Clinical Trial Site

    Boston, Massachusetts, 02118, United States

  • Clinical Trial Site

    Rochester, Minnesota, 55902, United States

  • Clinical Trial Site

    St Louis, Missouri, 63130, United States

  • Clinical Trial Site

    New York, New York, 10032, United States

  • Clinical Trial Site

    Chapel Hill, North Carolina, 27599, United States

  • Clinical Trial Site

    Columbus, Ohio, 43210, United States

  • Clinical Trial Site

    Portland, Oregon, 97239, United States

  • Clinical Trial Site

    Philadelphia, Pennsylvania, 19104, United States

  • Clinical Trial Site

    Dallas, Texas, 75246, United States

  • Clinical Trial Site

    Buenos Aires, C1428AQK, Argentina

  • Clinical Trial Site

    Northmead, New South Wales, NSW 2152, Australia

  • Clinical Trial Site

    Brisbane, Queensland, 4102, Australia

  • Clinical Trial Site

    Melbourne, Victoria, 3128, Australia

  • Clinical Trial Site

    Brussels, 1070, Belgium

  • Clinical Trial Site

    Leuven, 3000, Belgium

  • Clinical Trial Site

    Rio de Janeiro, CEP21941, Brazil

  • Clinical Trial Site

    Sofia, 1431, Bulgaria

  • Clinical Trial Site

    Montreal, H3A 2B4, Canada

  • Clinical Trial Site

    Vancouver, V5Z 1M9, Canada

  • Clinical Trial Site

    Nicosia, 2371, Cyprus

  • Clinical Trial Site

    Bordeaux, 33076, France

  • Clinical Trial Site

    Créteil, 94000, France

  • Clinical Trial Site

    Le Kremlin-Bicêtre, 94270, France

  • Clinical Trial Site

    Lille, 59037, France

  • Clinical Trial Site

    Marseille, 13005, France

  • Clinical Trial Site

    Nantes, 44093, France

  • Clinical Trial Site

    Heidelberg, 69120, Germany

  • Clinical Trial Site

    Mainz, 55131, Germany

  • Clinical Trial Site

    Münster, 48149, Germany

  • Clinical Trial Site

    Athens, 11528, Greece

  • Clinical Trial Site

    Messina, 98100, Italy

  • Clinical Trial Site

    Milan, 20133, Italy

  • Clinical Trial Site

    Pavia, 27100, Italy

  • Clinical Trial Site

    Rome, 00168, Italy

  • Clinical Trial Site

    Kumamoto, 860-8556, Japan

  • Clinical Trial Site

    Nagano, 390-8621, Japan

  • Clinical Trial Site

    Nagoya, 466-8560, Japan

  • Clinical Trial Site

    Osaka, 565-0871, Japan

  • Clinical Trial Site

    Kuala Lumpur, 59100, Malaysia

  • Clinical Trial Site

    Mexico City, Mexico City, 14080, Mexico

  • Clinical Trial Site

    Groningen, 9713 AP, Netherlands

  • Clinical Trial Site

    Lisbon, 1649-035, Portugal

  • Clinical Trial Site

    Porto, 4099-001, Portugal

  • Clinical Trial Site

    Daegu, 41944, South Korea

  • Clinical Trial Site

    Seoul, 05030, South Korea

  • Clinical Trial Site

    Seoul, 06351, South Korea

  • Clinical Trial Site

    Barcelona, 08035, Spain

  • Clinical Trial Site

    Hospitalet de Llobregat (Barcelona), 08907, Spain

  • Clinical Trial Site

    Huelva, 21005, Spain

  • Clinical Trial Site

    Madrid, 28040, Spain

  • Clinical Trial Site

    Madrid, 28222, Spain

  • Clinical Trial Site

    Valencia, 46026, Spain

  • Clinical Trial Site

    Solna, SE-171 64, Sweden

  • Clinical Trial Site

    Umeå, 907 37, Sweden

  • Clinical Trial Site

    Taipei, 100, Taiwan

  • Clinical Trial Site

    Taipei, 11217, Taiwan

  • Clinical Trial Site

    Taoyuan City, 333, Taiwan

  • Clinical Trial Site

    London, NW3 2QG, United Kingdom

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