Effects of Patisiran or Vutrisiran on Heart Health in ATTRv Patients
MyocardON-TTR - Myocardial Effects in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy Treated with Patisiran or Vutrisiran
This study is testing how two treatments, Patisiran and Vutrisiran, affect heart health in people with hereditary transthyretin amyloidosis who also have nerve damage.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Rennes University Hospital Academic / other |
| Locations | 8 sites (Bordeaux and 7 other locations) |
| Trial ID | NCT05873868 on ClinicalTrials.gov |
What this trial studies
This observational study aims to investigate the cardiac effects of Patisiran and Vutrisiran in patients with hereditary transthyretin amyloidosis (ATTRv) who also have stage 1 or 2 polyneuropathy. By conducting routine clinical and biological examinations, including echocardiography and cardiac MRI, the study will assess the morphological and functional consequences on the heart over a period of two years. Participants will complete quality of life and functional walking tests to provide additional insights into their health status during treatment. The goal is to better understand how these treatments impact cardiac function in this patient population.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with hereditary transthyretin amyloidosis and stage 1 or 2 polyneuropathy who have not previously been treated for ATTRv.
Not a fit: Patients currently receiving Tafamidis or those with contraindications to the required examinations may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the cardiac health of ATTRv patients, potentially leading to improved treatment strategies.
How similar studies have performed: While studies have explored the neurological effects of Patisiran and Vutrisiran, the specific focus on cardiac outcomes in this context is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Bordeaux and 7 other locations
- CHU Bordeaux Haut-Levêque — Bordeaux, France (Recruiting)
- APHP Henri Mondor — Créteil, France (Recruiting)
- CHU Grenoble Alpes — Grenoble, France (Recruiting)
- APHM Timone — Marseille, France (Recruiting)
- CHU Nancy Institut Louis Mathieu — Nancy, France (Recruiting)
- APHP Bichat — Paris, France (Not_yet_recruiting)
- CHU Rennes — Rennes, France (Recruiting)
- CHU Rangueil Toulouse — Toulouse, France (Recruiting)
Study contacts
- Principal investigator: Erwan Donal — Rennes University Hospital
- Study coordinator: Erwan Donal
- Email: erwan.donal@chu-rennes.fr
- Phone: 299284321
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.