Clinical trial NCT05950867: Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.
Official title: Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause: a Prospective Monocentric Study (CAP-TTR)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Polyneuropathy
Amyloidosis
Lead sponsor: Universitair Ziekenhuis Brussel
Sponsor class: OTHER
First posted: 2023-07-18
Enrollment: 150
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SCREENING

Brief summary: To investigate to what extent chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) is part of early non-cardiac manifestations of wild-type TTR cardiac amyloidosis (wtTTR-CA).

Consequently, explore whether this could ultimately lead to faster diagnosis and clinical outcome of wild-type TTR cardiac amyloidosis (wtTTR-CA).

Detailed description: Patients with chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) without well-defined cause will be recruited after a neurological standard routine work-up with NCS (Nerve conduction study) test, EMG, and Sudoscan®, previously performed at the neurology department of UZ-Brussel in normal clinical setting. All participants will be invited to the Neurology and Cardiology department for one visit on one day, for the following assessments:

Following exams will be performed:

* assessment of symptoms, severity, and duration of the polyneuropathy and the use of NTSS-6 and COMPASS31 score for mapping somatosensory and autonomic symptoms
* evaluation of objective polyneuropathy signs, using following scales: mPND, NIS
* Kansas City Cardiomyopathy Questionnaire (KCCQ)
* Electrocardiogram (ECG)
* Echocardiography

The following retrospective data from the medical file will be analyzed:

* assessment of medical history, medical treatment, and demographic data
* assessment of laboratory results (and, if applicable, other exams) extracted from the medical file and previously performed in the context of polyneuropathy workup
* assessment of previously performed NCV/EMG data and Sudoscan®, extracted from the medical file of the participants.

Eligibility criteria: Inclusion Criteria:

* Patients with chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) without well-defined etiology.
* Age: \>= 60 years
* Male and female gender
* Written informed consent

Exclusion Criteria:

* Known cause of polyneuropathy
* Other types of peripheral neuropathy than chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN).
* Patients younger than 60 years

Primary outcomes:
measure: Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause.
timeFrame: 36 months
measure: Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause
timeFrame: 36 months
measure: Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause
timeFrame: 36 months
Secondary outcomes:
measure: Secondary outcome: severity and evolution
timeFrame: 36 months
measure: Secondary outcome: severity and evolution
timeFrame: 36 months
measure: Secondary outcome: severity and evolution
timeFrame: 36 months
measure: Secondary outcome: severity and evolution
timeFrame: 36 months
measure: Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis
timeFrame: 36 months
measure: Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis
timeFrame: 36 months
measure: Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis
timeFrame: 36 months
Other outcomes:
Locations:
city: Jette
country: Belgium
facility: UZ Brussel
state: Belgium Capital City
Study officials:
affiliation: UZ Brussel - VUB
name: Veronique Bissay, MD, Phd
role: PRINCIPAL_INVESTIGATOR
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