Myocardial Effects in Patients with ATTRv with Polyneuropathy Treated with Patisiran or Vutrisiran
NCT ID: NCT05873868
Last Updated: 2024-12-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
20 participants
OBSERVATIONAL
2024-04-12
2026-07-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
CASE_ONLY
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Patients
Patients hATTR with neurological and cardiac damages treated with Patisiran or Vutrisiran
Six minutes walk test
Six minutes walk test just before first intake of treatment and after 1 and 2 years
Kansas City questionnaire
Kansas City questionnaire just before first intake of treatment and after 1 and 2 years
COMPASS31 self questionnaire
Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Six minutes walk test
Six minutes walk test just before first intake of treatment and after 1 and 2 years
Kansas City questionnaire
Kansas City questionnaire just before first intake of treatment and after 1 and 2 years
COMPASS31 self questionnaire
Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Rennes University Hospital
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Erwan Donal
Role: PRINCIPAL_INVESTIGATOR
Rennes University Hospital
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
CHU Bordeaux Haut-Levêque
Bordeaux, , France
APHP Henri Mondor
Créteil, , France
CHU Grenoble Alpes
Grenoble, , France
APHM Timone
Marseille, , France
CHU Nancy Institut Louis Mathieu
Nancy, , France
APHP Bichat
Paris, , France
CHU Rennes
Rennes, , France
CHU Rangueil Toulouse
Toulouse, , France
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Marie-Laure Gervais
Role: CONTACT
Phone: 299282555
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Patricia Reant, Pr
Role: primary
Thibaud Damy, Pr
Role: primary
Gilles Barone-Rochette, Pr
Role: primary
Gilbert Habib, Pr
Role: primary
Olivier Huttin, Pr
Role: primary
Vincent ALGALARRONDO, Md
Role: primary
Erwan Donal, Pr
Role: primary
Eve Cariou, Md
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
35RC22_9817_MyocardON-TTR
Identifier Type: -
Identifier Source: org_study_id