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Heart check: do Nerve-Targeting drugs also shield the heart in rare amyloidosis?

NCT ID NCT05873868

Knowledge-focused Sponsor: Rennes University Hospital 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study follows 20 adults with hereditary transthyretin amyloidosis (ATTRv) who have both nerve and heart involvement. They are treated with patisiran or vutrisiran for two years. Researchers use MRI scans and questionnaires to see how the drugs affect heart structure and function.

What this could mean

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

Active substance
patisiran or vutrisiran
What this could lead to
If successful, this could clarify whether these drugs protect the heart in mixed-form ATTRv amyloidosis, guiding future treatment decisions.
What could go wrong
This is a small observational study (20 people) with no control group, so results may not prove cause and effect or apply broadly.

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.

Participants

About 20 people

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

Started

Apr 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Who is studied

Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy treated with Patisiran or Vutrisiran

Ages

18 years and older

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: * Patients aged 18 years or older * Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy * Patient not previously treated for ATTRv * Patients for whom treatment with patisiran or vutrisiran has been initiated by a hospital neurologist in accordance with recommendations for a minimum of 24 months. * Patients with NYHA stage 1 and 2 cardiac disease. * Beneficiary of a social security scheme * Person who does not object to his/her participation in the research Exclusion Criteria: * Patients treated with Tafamidis simultaneously with patisiran or vutrisiran * Adults under legal protection (legal guardianship, curatorship, guardianship), persons deprived of liberty. * Contraindications to the explorations provided for in the protocol: claustrophobia, metallic implant contraindicating MRI, woman of childbearing age

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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

  • APHP Henri Mondor

    Créteil, France

  • CHU Bordeaux Haut-Levêque

    Bordeaux, France

  • CHU Grenoble Alpes

    Grenoble, France

  • CHU Nancy Institut Louis Mathieu

    Nancy, France

  • CHU Rangueil Toulouse

    Toulouse, France

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