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

NCT05873868 | Active Not Recruiting

• 1 other identifier: 35RC22_9817_MyocardON-TTR
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 5

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

Conditions

Amyloidosis, Hereditary; Transthyretin Amyloidosis

Keywords

Patisiran; Vutrisiran

Outcome Measures

Primary Outcomes (1)

• Difference in longitudinal relaxation time (T1) per mapping between M0 and M24

  Longitudinal relaxation time (T1) is the process by which the net magnetization (M) grows/returns to its initial maximum value (Mo) parallel to Bo in a MRI. Myocardial T1 depends on the pulse sequence, cardiac cycle as well as other factors and increases at higher magnetic field strength. T1-mapping can detect a variety of myocardial pathologies, where it shows increased values. Because of variations between scanners the primary use of a local reference range is recommended and if a local reference range is not available quantitative results should not be clinically reported. An intermediate analysis will be done at 12 months

  24 months

Secondary Outcomes (8)

• Difference in Kansas City Cardiomyopathy Questionnaire score between M0 and M24

  24 months

• Difference in Compass31 questionnaire score between M0 and M24

  24 months

• Difference in distance obtained in the 6-minute walking test between M0 and M24

  24 months

• Difference in Perugini Grading Score between M0 and M24

  24 months

• Difference in the value of the global longitudinal strain between M0 and M24

  24 months

Study Arms (1)

Patients

Patients hATTR with neurological and cardiac damages treated with Patisiran or Vutrisiran

Other: Six minutes walk test; Other: Kansas City questionnaire; Other: COMPASS31 self questionnaire

Interventions

Six minutes walk test OTHER

Six minutes walk test just before first intake of treatment and after 1 and 2 years

Patients

Kansas City questionnaire OTHER

Kansas City questionnaire just before first intake of treatment and after 1 and 2 years

Patients

COMPASS31 self questionnaire OTHER

Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years

Patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy treated with Patisiran or Vutrisiran

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rennes University Hospital: lead

Study Sites (5)

CHU Bordeaux Haut-Levêque

Bordeaux, France

Location

APHP Henri Mondor

Créteil, France

Location

CHU Grenoble Alpes

Grenoble, France

Location

CHU Nancy Institut Louis Mathieu

Nancy, France

Location

CHU Rangueil Toulouse

Toulouse, France

Location

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Amyloidosis, Familial

Condition Hierarchy (Ancestors)

Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Officials

• Erwan Donal

  Rennes University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2023
• First Posted: May 24, 2023
• Study Start: April 12, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: January 5, 2026

Record last verified: 2025-12

Locations

FR (5)