Wild-type Amyloid CardiopathyN-SACNCT04828928
Neuropathy in Patients Monitored for Wild-type TTR Cardiac Amyloidosis (Non-mutated)
A study testing electromyogram in people with Wild-type Amyloid Cardiopathy, run by University Hospital, Bordeaux at 3 sites in France.
Trial summary
Transthyretin (TTR) amyloidosis is a rare disabling disorder that can be hereditary or sporadic. Depending on the form, various tissues are affected. While in hereditary cases, neuropathy is predominant, cardiac impairment is the main manifestation in the sporadic form. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Wild-type Amyloid Cardiopathy
- What is being tested?
- electromyogram
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 3 sitesFrance
- Study dates
- 23 Mar 2021to 31 Mar 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients of both gender, over 18 years old, with transthyretin amyloid cardiopathy according to one of the two American Heart Association definitions of 2016
- No mutation in the TTR gene
- Patients giving their free and informed consent to participate after information about the research
- Patients affiliated to or benefiting from a social security scheme
Who cannot join
- Patients with chronic neuropathy related to a known aetiology
- Patients under guardianship or curatorship
Trial locations and contacts
3 sites in 1 country.
France3 sites
CHU de Bordeaux
Bordeaux, 33076
CHU de Nantes
Nantes, 44093
CHU de Toulouse
Toulouse, 31059
About this study
Official title: Prospective Study to Investigate Neuropathy in Patients Monitored for Wild-type TTR Cardiac Amyloidosis (Non-mutated)
Transthyretin (TTR) amyloidosis is a rare disabling disorder that can be hereditary or sporadic. Depending on the form, various tissues are affected. While in hereditary cases, neuropathy is predominant, cardiac impairment is the main manifestation in the sporadic form.
The main objective of this project is to evaluate the proportion of patients with neuropathy in a population of patients with a non-mutated TTR amyloid cardiopathy condition.
Read the detailed description
Transthyretin (TTR) amyloidosis belongs to a group of severe and multi-systemic diseases caused by an extracellular accumulation of fibrillar proteins arranged in beta-pleated sheets. This pathology can be hereditary (mutations in the TTR gene) or sporadic. Depending on the form, various tissues are affected: peripheral nervous system (leading to neuropathy, especially vegetative), heart, kidney... While in forms linked to TTR mutations neuropathy is the main manifestation, in the sporadic form (also called wild-type), the cardiac impairment is predominant. Other organ damages are rarely reported in this second form. In the THAOS registry (Coelho T. et al, 2013), a clinical peripheral neuropathy is reported in 28.4% out of 67 patients with the sporadic form of the disease, although the authors do not provide a precise description of the neuropathy type. We propose to prospectively study patients with a wild-type amyloid cardiopathy condition to identify and describe the associated neuropathy. A pilot study conducted at the Bordeaux University Hospital demonstrated the feasibility and interest of this research: it showed the presence of an undetermined aetiology polyneuropathy in 35.7% out of 14 patients followed for senile cardiac amyloidosis.
Tafamidis is used on familial amyloid neuropathy and a recent study shows an effect on senile amyloid cardiopathy (Maurer MS et al., 2018) which strengthens the need to determine the frequency of neuropathies associated with wild-type amyloid cardiopathy and to type them more accurately.
- Conditions
- Wild-type Amyloid Cardiopathy
- Being tested
- electromyogram
- Keywords
- wild-type amyloid cardiopathy, neuropathy, descriptive study
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
electromyogramProcedure or surgery
Patients meeting the criteria will be seen in consultation with standardized interview and clinical examination. An electromyogram will be then carried out to check for the presence of neuropathy. Finally, for patients diagnosed with neuropathy, a biological check-up to look for another cause of neuropathy will be performed. In patients who already had an EMG as part of their medical follow-up, the examination will not be repeated if it strictly meets the conditions of the minimum protocol and if it was done within the year prior to inclusion. In patients who already had an identical biological assessment in the year prior to inclusion, the sample will not be taken.
| Study group | Role | What they receive |
|---|---|---|
| proportion of patients with neuropathy | Experimental | to prospectively study patients with a wild-type amyloid cardiopathy condition to identify and describe the associated neuropathy Receives: Procedure: electromyogram |
What is being measured
The primary outcome is the main question the study is designed to answer.
Presence of a clinical and/or electrophysiological neuropathy
The diagnosis of peripheral neuropathy should meet P.J. Dyck's definition of peripheral neuropathy (New England Journal of Medicine, 1982), based on:
- the clinical judgment resulting from the standardized clinical examination (carried out by an expert neurologist) and according to the HAS recommendations on the diagnosis of peripheral neuropathies;
- electrophysiological abnormalities, interpreted in the clinical context, after an examination carried out by an expert neurophysiologist.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Secondary outcomes (11)
Clinical data of patients with polyneuropathy with no identified etiology: Epidemiological characteristics
age, sex, age at diagnosis, age at assessment
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: history characteristics
carpal tunnel, diabetes, narrow cervical canal, alcohol consumption, radiculalgia in the lower limbs
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Heart attack
Heart failure ( yes/no), pacemaker (Yes/no) and Functional classification of heart disease according to the New York Heart Association (NYHA) score.
The NYHA score is a classification in 4 stages of increasing severity is proposed by the New York Heart Association, it is based on the intensity of the symptoms:
I: Asymptomatic, discomfort during exceptional efforts. II: Moderate discomfort for significant efforts. III: Discomfort felt during moderate efforts. IV: Discomfort during the slightest effort or at rest. Référence: Hurst Hurst JW, Morris DC, Alexander RW. The use of the New York Heart Association's classification of cardiovascular disease as part of the patient's complete Problem List. Clin Cardiol . JW, Morris DC, Alexander RW. L'utilisation de la New York Heart Association de classification du des maladies cardiovasculaires dans le cadre du patient compléter la liste des problèmes. Clin Cardiol. 1999 Jun ;22 (6):385-90.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - KARNOFSKI index
The KARNOFSKY index is used to assess the general condition of the patient: it is a scale from 0 to 100, the maximum representing the absence of complaints related to the disease.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - NIS-LL-
Neuropathy Impairment Score-Lower Limb (NIS-LL): this is a score out of 88, the minimum (0) representing an asymptomatic patient
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - ONLS-
The Overall Neuropathy Limitation Scale (ONLS) is used to assess the functional impact of neuropathy: score out of 12, the minimum (0) representing a patient without any functional impairment related to his neuropathy.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - RODS-
The Rasch Score-built Overall Disability Scale (RODS) is used to assess the impact of neuropathy on activities of daily living: it is a scale from 0 to 48 with the maximum representing a patient asymptomatic.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - CADT-
The Compound Autonomic Dysfunction Test (CADT) is used to assess vegetative impairment: it is rated out of 16 maximum if normal (erectile dysfunction will not be taken into account in men).
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores -MoCA-
The Montreal Cognitive Assessment (MoCA) is used for a cognitive assessment: out of 30, the maximum being a subject without impairment.
Measured: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Electrophysiological data
This data includes:
- Motor and / or sensory impairment
- Axonal and / or demyelinating character
- Length-dependent or non-length-dependent character
- Topography of the disease
Measured: within 6 months of inclusion (at the time of the electromyogram)
Estimation of the frequency of the presence of neuropathy in our study population in order to compare it to a reference population
Estimation of the frequency of the presence of neuropathy in our study population in order to compare it to a reference population
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 posted2 Apr 2021
- Study start23 Mar 2021
- Primary completion31 Mar 2023
- Study completion31 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wild-type Amyloid CardiopathyStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Guilhem SOLE, MD, Principal investigator, Université Hospital, Bordeaux
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04828928
- Sponsor's protocol ID
- CHUBX 2020/27
- Other IDs
- 2020-A02660-39
- Acronym
- N-SAC
- Status last verified
- Oct 2023
- First submitted
- 26 Mar 2021
- Last updated
- 2 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Study to Investigate Neuropathy in Patients Monitored for Wild-type TTR Cardiac Amyloidosis (Non-mutated)
- ClinicalTrials.gov ID
- NCT04828928
- Acronym / sponsor ID
- N-SAC / CHUBX 2020/27
- Conditions
- Wild-type Amyloid Cardiopathy
- Keywords
- wild-type amyloid cardiopathy, neuropathy, descriptive study
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 23 Mar 2021 (actual)
- Primary completion
- 31 Mar 2023 (actual)
- Study completion
- 31 Mar 2023 (actual)
- First posted
- 2 Apr 2021 (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.
Transthyretin (TTR) amyloidosis is a rare disabling disorder that can be hereditary or sporadic. Depending on the form, various tissues are affected. While in hereditary cases, neuropathy is predominant, cardiac impairment is the main manifestation in the sporadic form.
The main objective of this project is to evaluate the proportion of patients with neuropathy in a population of patients with a non-mutated TTR amyloid cardiopathy condition.
Detailed description
Transthyretin (TTR) amyloidosis belongs to a group of severe and multi-systemic diseases caused by an extracellular accumulation of fibrillar proteins arranged in beta-pleated sheets. This pathology can be hereditary (mutations in the TTR gene) or sporadic. Depending on the form, various tissues are affected: peripheral nervous system (leading to neuropathy, especially vegetative), heart, kidney... While in forms linked to TTR mutations neuropathy is the main manifestation, in the sporadic form (also called wild-type), the cardiac impairment is predominant. Other organ damages are rarely reported in this second form. In the THAOS registry (Coelho T. et al, 2013), a clinical peripheral neuropathy is reported in 28.4% out of 67 patients with the sporadic form of the disease, although the authors do not provide a precise description of the neuropathy type. We propose to prospectively study patients with a wild-type amyloid cardiopathy condition to identify and describe the associated neuropathy. A pilot study conducted at the Bordeaux University Hospital demonstrated the feasibility and interest of this research: it showed the presence of an undetermined aetiology polyneuropathy in 35.7% out of 14 patients followed for senile cardiac amyloidosis.
Tafamidis is used on familial amyloid neuropathy and a recent study shows an effect on senile amyloid cardiopathy (Maurer MS et al., 2018) which strengthens the need to determine the frequency of neuropathies associated with wild-type amyloid cardiopathy and to type them more accurately.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| proportion of patients with neuropathy | Experimental | Procedure: electromyogramto prospectively study patients with a wild-type amyloid cardiopathy condition to identify and describe the associated neuropathy |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients of both gender, over 18 years old, with transthyretin amyloid cardiopathy according to one of the two American Heart Association definitions of 2016
- No mutation in the TTR gene
- Patients giving their free and informed consent to participate after information about the research
- Patients affiliated to or benefiting from a social security scheme
Key exclusion criteria (2 listed)
- Patients with chronic neuropathy related to a known aetiology
- Patients under guardianship or curatorship
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 |
|---|---|---|
| electromyogramPatients meeting the criteria will be seen in consultation with standardized interview and clinical examination. An electromyogram will be then carried out to check for the presence of neuropathy. Finally, for patients diagnosed with neuropathy, a biological check-up to look for another cause of neuropathy will be performed. In patients who already had an EMG as part of their medical follow-up, the examination will not be repeated if it strictly meets the conditions of the minimum protocol and if it was done within the year prior to inclusion. In patients who already had an identical biological assessment in the year prior to inclusion, the sample will not be taken. | Procedure or surgery | proportion of patients with neuropathy |
Primary outcome
Presence of a clinical and/or electrophysiological neuropathy
Time frame: within 6 months of inclusion (at the time of the electromyogram)
The diagnosis of peripheral neuropathy should meet P.J. Dyck's definition of peripheral neuropathy (New England Journal of Medicine, 1982), based on: * the clinical judgment resulting from the standardized clinical examination (carried out by an expert neurologist) and according to the HAS recommendations on the diagnosis of peripheral neuropathies; * electrophysiological abnormalities, interpreted in the clinical context, after an examination carried out by an expert neurophysiologist.
11 secondary outcomes
Clinical data of patients with polyneuropathy with no identified etiology: Epidemiological characteristics
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: history characteristics
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Heart attack
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - KARNOFSKI index
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - NIS-LL-
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - ONLS-
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - RODS-
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores - CADT-
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Clinical scores -MoCA-
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Clinical data of patients with polyneuropathy with no identified etiology: Electrophysiological data
Time frame: within 6 months of inclusion (at the time of the electromyogram)
Estimation of the frequency of the presence of neuropathy in our study population in order to compare it to a reference population
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2026.
- Individual participant dataSharing planned: No
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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.
Guilhem SOLE, MD principal investigator
Université Hospital, Bordeaux
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing electromyogram in people with Wild-type Amyloid Cardiopathy, run by University Hospital, Bordeaux at 3 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 4 inclusion and 2 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: finished, as of the sponsor's last update on 2 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Presence of a clinical and/or electrophysiological neuropathy (time frame: within 6 months of inclusion (at the time of the electromyogram)). 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 2 Jun 2026.