NCT05873868

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

MyocardON-TTR - Myocardial Effects in Patients With Hereditary Transthyretin-mediated Amyloidosis With Polyneuropathy Treated With Patisiran or Vutrisiran

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 20 (estimated)
• Sponsor: Rennes University Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Transthyretin Amyloidosis
• Amyloidosis, Hereditary

Interventions

Type	Name	Description
OTHER	Six minutes walk test	Six minutes walk test just before first intake of treatment and after 1 and 2 years
OTHER	Kansas City questionnaire	Kansas City questionnaire just before first intake of treatment and after 1 and 2 years
OTHER	COMPASS31 self questionnaire	Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years

Timeline

• Start date: 2024-04-12
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2023-05-24
• Last updated: 2026-01-05

Locations

5 sites across 1 country: France