Transthyretin AmyloidosisMyocardON-TTRNCT05873868
Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran
An observational study of Transthyretin Amyloidosis and Amyloidosis, Hereditary, run by Rennes University Hospital at 5 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis, Amyloidosis, Hereditary
- What is being tested?
- Six minutes walk test, Kansas City questionnaire and COMPASS31 self questionnaire
- How many people
- 20 participants
- Where
- 5 sitesFrance
- Study dates
- 12 Apr 2024to Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy treated with Patisiran or Vutrisiran
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
France5 sites
CHU Bordeaux Haut-Levêque
Bordeaux
APHP Henri Mondor
Créteil
CHU Grenoble Alpes
Grenoble
CHU Nancy Institut Louis Mathieu
Nancy
CHU Rangueil Toulouse
Toulouse
About this study
Official title: MyocardON-TTR - Myocardial Effects in Patients With Hereditary Transthyretin-mediated Amyloidosis With Polyneuropathy Treated With Patisiran or Vutrisiran
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
Read the 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
- Being tested
- Six minutes walk test, Kansas City questionnaire, COMPASS31 self questionnaire
- Keywords
- Patisiran, Vutrisiran
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Amyloidosis, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Six minutes walk testOther
Six minutes walk test just before first intake of treatment and after 1 and 2 years
Kansas City questionnaireOther
Kansas City questionnaire just before first intake of treatment and after 1 and 2 years
COMPASS31 self questionnaireOther
Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years
| Study group | Role | What they receive |
|---|---|---|
| Patients | Patients hATTR with neurological and cardiac damages treated with Patisiran or Vutrisiran Receives: Other: Six minutes walk test; Other: Kansas City questionnaire; Other: COMPASS31 self questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 24 months
Secondary outcomes (8)
Difference in Kansas City Cardiomyopathy Questionnaire score between M0 and M24
Kansas City Cardiomyopathy Questionnaire is a self-administered questionnaire developed to independently measure the patient's perception of their health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life. Five scores are generated, all scores are scaled 0-100, where 0 denotes the lowest reportable health status and 100 the highest. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in Compass31 questionnaire score between M0 and M24
The COMPASS-31 (Composite Autonomic Symptom Score-31) scale measures neurodegenerative system symptoms through 31 patient-reported questions. Assessment is through six weighted domains: orthostatic intolerance [10 points]; vasomotor [6 points]; secretomotor [7 points]; gastrointestinal [28 points]; bladder [9 points] and pupillomotor [15 points]. A higher score indicates worse autonomic dysfunction. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in distance obtained in the 6-minute walking test between M0 and M24
The 6 Minute Walk Test is a sub-maximal exercise test used to assess aerobic capacity and endurance. The distance in meter covered over a time of 6 minutes is used as the outcome by which to compare changes in performance capacity. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in Perugini Grading Score between M0 and M24
The Perugini grading scale is a semi-quantitative method of scoring cardiac uptake following injection of 99mTc-DPD, 99mTc-Pyrophosphate or 99mTc-HMDP scintigraphy in the investigation of cardiac amyloidosis (particularly ATTR amyloidosis). The grading scale visually compares tracer uptake in the myocardium and ribs.
grade 0 is no cardiac and normal rib uptake; grade 1 is cardiac less than rib uptake; grade 2 is cardiac equal to rib uptake; and grade 3 is cardiac greater than rib uptake with mild/absent rib uptake. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in the value of the global longitudinal strain between M0 and M24
global longitudinal strain is a simple parameter in echocardiography to analyse atrial function abnormalities expressed in percentage that expresses longitudinal shortening as a percentage (change in length as a proportion to baseline length) for left ventricular (LV) systolic dysfunction. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in the value of the global left atrial longitudinal strain between M0 and M24
Global left atrial longitudinal strain is a parameter of echocardiography to analyse atrial function abnormalities expressed in percentage. An intermediate analysis will be done at 12 months
Measured: 24 months
Difference in the value of the global strain of the right ventricular free wall between M0 and M124
Global strain of the right ventricular free wall of echocardiography to analyse right ventricule function expressed in percentage. An intermediate analysis will be done at 12 months
Measured: 24 months
Evolution of myocardial work between M0 and M24
Myocardial work is composed of 4 parameters to assess myocardial function using echocardiography. An intermediate analysis will be done at 12 months
Global Constructive Work (expressed in mmHg%) : Positive work performed in systole (shortening) + Negative work performed in isovolumetric relaxation (lengthening). Normal range : 1582-2881
Global Wasted Work (expressed in mmHg%) : Negative work performed in systole (lengthening) + Positive work performed in isovolumetric relaxation (shortening). Normal range : 226 ± 28
Global Work Efficiency (expressed in %) Percentage (0-100%) of constructive work over total work => Constructive work/(constructive work + wasted work). Normal range : 91 ± 0.8
Global Work Index (expressed in mmHg%) : Amount of myocardial work performed by the left ventricle during systole => area of PSL from mitral valve closure to mitral valve opening. Normal range : 1292-2505
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2023
- Study start12 Apr 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by Rennes University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rennes University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Erwan Donal, Principal investigator, Rennes University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05873868
- Sponsor's protocol ID
- 35RC22_9817_MyocardON-TTR
- Acronym
- MyocardON-TTR
- Status last verified
- Dec 2025
- First submitted
- 15 May 2023
- Last updated
- 5 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MyocardON-TTR - Myocardial Effects in Patients With Hereditary Transthyretin-mediated Amyloidosis With Polyneuropathy Treated With Patisiran or Vutrisiran
- ClinicalTrials.gov ID
- NCT05873868
- Acronym / sponsor ID
- MyocardON-TTR / 35RC22_9817_MyocardON-TTR
- Conditions
- Transthyretin Amyloidosis, Amyloidosis, Hereditary
- Keywords
- Patisiran, Vutrisiran
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Rennes University HospitalUniversity, hospital or other
- Study start
- 12 Apr 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 24 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients | – | Other: Six minutes walk test, Other: Kansas City questionnaire, Other: COMPASS31 self questionnairePatients hATTR with neurological and cardiac damages treated with Patisiran or Vutrisiran |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy treated with Patisiran or Vutrisiran
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Six minutes walk testSix minutes walk test just before first intake of treatment and after 1 and 2 years | Other | Patients |
| Kansas City questionnaireKansas City questionnaire just before first intake of treatment and after 1 and 2 years | Other | Patients |
| COMPASS31 self questionnaireCompass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years | Other | Patients |
Primary outcome
Difference in longitudinal relaxation time (T1) per mapping between M0 and M24
Time frame: 24 months
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
8 secondary outcomes
Difference in Kansas City Cardiomyopathy Questionnaire score between M0 and M24
Time frame: 24 months
Difference in Compass31 questionnaire score between M0 and M24
Time frame: 24 months
Difference in distance obtained in the 6-minute walking test between M0 and M24
Time frame: 24 months
Difference in Perugini Grading Score between M0 and M24
Time frame: 24 months
Difference in the value of the global longitudinal strain between M0 and M24
Time frame: 24 months
Difference in the value of the global left atrial longitudinal strain between M0 and M24
Time frame: 24 months
Difference in the value of the global strain of the right ventricular free wall between M0 and M124
Time frame: 24 months
Evolution of myocardial work between M0 and M24
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Transthyretin Amyloidosis
Active, not recruitingObservational study
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ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
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A Phase 3 Study of NTLA-2001 in ATTRv-PN
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RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erwan Donal principal investigator
Rennes University Hospital
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloidosis and Amyloidosis, Hereditary, run by Rennes University Hospital at 5 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Difference in longitudinal relaxation time (T1) per mapping between M0 and M24 (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2026.