Clinical trial NCT05873868: Myocardial Effects in Patients with ATTRv with Polyneuropathy Treated with Patisiran or Vutrisiran
Official title: MyocardON-TTR - Myocardial Effects in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy Treated with Patisiran or Vutrisiran
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transthyretin Amyloidosis
Amyloidosis, Hereditary
Keywords: Patisiran
Vutrisiran
Lead sponsor: Rennes University Hospital
Sponsor class: OTHER
First posted: 2023-05-24
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy treated with Patisiran or Vutrisiran

Brief 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

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Difference in longitudinal relaxation time (T1) per mapping between M0 and M24
timeFrame: 24 months
Secondary outcomes:
measure: Difference in Kansas City Cardiomyopathy Questionnaire score between M0 and M24
timeFrame: 24 months
measure: Difference in Compass31 questionnaire score between M0 and M24
timeFrame: 24 months
measure: Difference in distance obtained in the 6-minute walking test between M0 and M24
timeFrame: 24 months
measure: Difference in Perugini Grading Score between M0 and M24
timeFrame: 24 months
measure: Difference in the value of the global longitudinal strain between M0 and M24
timeFrame: 24 months
measure: Difference in the value of the global left atrial longitudinal strain between M0 and M24
timeFrame: 24 months
measure: Difference in the value of the global strain of the right ventricular free wall between M0 and M124
timeFrame: 24 months
measure: Evolution of myocardial work between M0 and M24
timeFrame: 24 months
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: CHU Bordeaux Haut-Levêque
city: Créteil
country: France
facility: APHP Henri Mondor
city: Grenoble
country: France
facility: CHU Grenoble Alpes
city: Marseille
country: France
facility: APHM Timone
city: Nancy
country: France
facility: CHU Nancy Institut Louis Mathieu
city: Paris
country: France
facility: APHP Bichat
city: Rennes
country: France
facility: CHU Rennes
city: Toulouse
country: France
facility: CHU Rangueil Toulouse
Study officials:
affiliation: Rennes University Hospital
name: Erwan Donal
role: PRINCIPAL_INVESTIGATOR
References: