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

Last updated: December 23, 2024
Sponsor: Rennes University Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Amyloidosis

Treatment

Six minutes walk test

Kansas City questionnaire

COMPASS31 self questionnaire

Clinical Study ID

NCT05873868
35RC22_9817_MyocardON-TTR
  • Ages > 18
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients aged 18 years or older

  • Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2polyneuropathy

  • Patient not previously treated for ATTRv

  • Patients for whom treatment with patisiran or vutrisiran has been initiated by ahospital 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

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

Study Design

Total Participants: 20
Treatment Group(s): 3
Primary Treatment: Six minutes walk test
Phase:
Study Start date:
April 12, 2024
Estimated Completion Date:
July 31, 2026

Study Description

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.

Connect with a study center

  • CHU Bordeaux Haut-Levêque

    Bordeaux,
    France

    Active - Recruiting

  • APHP Henri Mondor

    Créteil,
    France

    Active - Recruiting

  • CHU Grenoble Alpes

    Grenoble,
    France

    Active - Recruiting

  • APHM Timone

    Marseille,
    France

    Active - Recruiting

  • CHU Nancy Institut Louis Mathieu

    Nancy,
    France

    Active - Recruiting

  • APHP Bichat

    Paris,
    France

    Site Not Available

  • CHU Rennes

    Rennes,
    France

    Active - Recruiting

  • CHU Rangueil Toulouse

    Toulouse,
    France

    Active - Recruiting

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