Rennes University Hospital
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
Transthyretin Amyloidosis
Amyloidosis, Hereditary
Six minutes walk test
Kansas City questionnaire
COMPASS31 self questionnaire
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.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 20 participants |
| Official Title : | MyocardON-TTR - Myocardial Effects in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy Treated with Patisiran or Vutrisiran |
| Actual Study Start Date : | 2024-04-12 |
| Estimated Primary Completion Date : | 2026-02 |
| Estimated Study Completion Date : | 2026-07 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | 18 Years |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
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