Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

20 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-04-12

Study Completion Date

2027-12-31

Brief Summary

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ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).

Patisiran and vutrisiran have shown protective effects on the progression of neurological damage.

The effects of Patisiran or vutrisiran on the heart remain incompletely understood. The aim of this study is to better understand the morphological and functional cardiac consequences in ATTRv patients with stage 1 or 2 polyneuropathy with a mixed form treated with Patisiran or vutrisiran

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Detailed Description

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ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).

Patisiran and vutrisiran have shown protective effects on the progression of neurological damage.

The effects of Patisiran or vutrisiran on the heart remain incompletely understood.

During their therapeutic management, including the prescription of Patisiran or vutrisiran, the routine examinations carried out at the inclusion, one and two year later will allow us to observe the consequences on myocardial activity during the routine consultation after 1 and 2 years of treatment.

Examinations are : clinical and biological exams, EKC, echocardiography, cardiac MRI and scintigraphy.

Data at the start of treatment and at 1 and 2 years will be collected, especially cardiac function assesment In addition, during these two consultations, a life quality questionnaire, a dysautonomia questionnaire and a functional walking test will be carried out specifically for the study.

The aim of this study is to better understand the morphological and functional cardiac consequences in ATTRv patients with stage 1 or 2 polyneuropathy with a mixed form treated with Patisiran or vutrisiran.

Conditions

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Transthyretin Amyloidosis Amyloidosis, Hereditary

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients

Patients hATTR with neurological and cardiac damages treated with Patisiran or Vutrisiran

Six minutes walk test

Intervention Type OTHER

Six minutes walk test just before first intake of treatment and after 1 and 2 years

Kansas City questionnaire

Intervention Type OTHER

Kansas City questionnaire just before first intake of treatment and after 1 and 2 years

COMPASS31 self questionnaire

Intervention Type OTHER

Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years

Interventions

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Six minutes walk test

Six minutes walk test just before first intake of treatment and after 1 and 2 years

Intervention Type OTHER

Kansas City questionnaire

Kansas City questionnaire just before first intake of treatment and after 1 and 2 years

Intervention Type OTHER

COMPASS31 self questionnaire

Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years

Intervention Type OTHER

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No