The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05950867
Recruitment Status : Recruiting
First Posted : July 18, 2023
Last Update Posted : November 7, 2023
Sponsor:
Information provided by (Responsible Party):
Veronique Bissay, Universitair Ziekenhuis Brussel

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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).


Condition or disease Intervention/treatment Phase
Polyneuropathy Amyloidosis Diagnostic Test: ECG + echocardiography Other: Answering questionnaires Not Applicable

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.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Screening
Official Title: Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause: a Prospective Monocentric Study (CAP-TTR)
Actual Study Start Date : July 28, 2023
Estimated Primary Completion Date : June 1, 2026
Estimated Study Completion Date : June 1, 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
1 arm including patients with CAP and/or SFN

1 arm including patients with chronic axonal length-dependent polyneuropathy and/or small-fiber neuropathy.

All participants will be screened with ECG and echocardiography. All participants will be asked to complete questionnaires about there polyneuropathy and cardiological symptoms.

 Diagnostic Test: ECG + echocardiography
Electrocardiogram and echocardiography

Other: Answering questionnaires
Answering questionnaires about polyneuropathy symptoms (NTSS -6, COMPASS31, NIS, mPND) and also cardialogical symptoms (KCCQ-12).


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause. [ Time Frame: 36 months ]

    Prevalence of cardiac amyloidosis by doing a cardiac screening for wild-type cardiac amyloidosis with performing an echocardiography.

    Echocardiography criteria: LV wall (>12mm), left ventricular ejection fraction (%), Apical sparing pattern (y/n), diastolic dysfunction (y/n), left atrial volume (ml/m2).

    Left atrial volume in ml and LA volume in ml/m2, whereby m2 is the body surface area based on weight and height (Mosteller formula).


  2. Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [ Time Frame: 36 months ]

    Prevalence of cardiac amyloidosis by doing a cardiac screening for wild-type cardiac amyloidosis with performing an ECG.

    ECG criteria: atrial fibrillation (y/n), QRS-duration, low voltage (y/n)


  3. Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [ Time Frame: 36 months ]

    Prevalence of cardiac amyloidosis by doing a cardiac screening for wild-type cardiac amyloidosis with performing a questionnaire (Kansas City Cardiomyopathy Questionnaire).

    KCCQ-12 has 4 domains (Physical Limitation Score, Symptom Frequency Score, Quality of Life Score, Social Limitation Score) and one Summary Score. Scores are scaled 0-100, where 0 denotes the lowest reportable health status and 100 the highest.

    This descriptive score will be used as a functional parameter without a cut-off.



Secondary Outcome Measures :
  1. Secondary outcome: severity and evolution [ Time Frame: 36 months ]
    Considering the rather small study population and the rarity of wtTTR amyloidosis, statistical analyses cannot be used without massively overfitting the results, which will not be reproducible. The investigators consequently opt for descriptive statistics to compare the group of polyneuropathy patients with wt-Ca and those without wt-CA. Therefore a few parameters are examined. The first scale used therefore is the modified Polyneuropathy Disability score (mPND). This scale is a questionnaire with a few questions about the complaints of the polyneuropathy in the daily life. O means no complaints of the polyneuropathy in daily life, IV means a major impact on the daily life of the patient.

  2. Secondary outcome: severity and evolution [ Time Frame: 36 months ]

    Considering the rather small study population and the rarity of wtTTR amyloidosis, statistical analyses cannot be used without massively overfitting the results, which will not be reproducible.

    The investigators consequently opt for descriptive statistics to compare the group of polyneuropathy patients with wt-Ca and those without wt-CA. Therefore a few parameters are examined. The second scale used therefore is the Neuropathy Impairment Score (NIS).This scale is a questionnaire with a few questions about the complaints of the polyneuropathy in the daily life. The score is between 0 and 244. O means less complaints in the daily life, 244 means major impact in the daily life of the patient.


  3. Secondary outcome: severity and evolution [ Time Frame: 36 months ]

    Considering the rather small study population and the rarity of wtTTR amyloidosis, statistical analyses cannot be used without massively overfitting the results, which will not be reproducible.

    The investigators consequently opt for descriptive statistics to compare the group of polyneuropathy patients with wt-Ca and those without wt-CA. Therefore a few parameters are examined. The third scale used therefore is the Neuropathy Total Symptom Score - Health Care professional administered version (NTSS-6). This scale is a questionnaire with a few items about the symptoms of the polyneuropathy in the daily life (aching pain, burning pain, prickling sensation, numbness, lancinating pain and allodynia). Depending on the presence of the symptoms in time and strength, a score will show up. On each symptom you can score a maximum of 3,66. So the total score for this scale is 21,96 if the patients has major complaints in daily life.


  4. Secondary outcome: severity and evolution [ Time Frame: 36 months ]

    Considering the rather small study population and the rarity of wtTTR amyloidosis, statistical analyses cannot be used without massively overfitting the results, which will not be reproducible.

    The investigators consequently opt for descriptive statistics to compare the group of polyneuropathy patients with wt-Ca and those without wt-CA. Therefore a few parameters are examined. The fourth scale used therefore is the Composite Autonomic Symptom score (COMPASS-31). This scale is a questionnaire with a 31 questions about the complaints in the daily life and also if there complaints/symptoms on the autonomic system. A few questions are asked about different domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal system, bladder, pupillomotor).The total score is minimal 0 and maximal 75 raw counted (100 weighted).Each domain had a weighting factor to be count depending on the answers of the patient.


  5. Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [ Time Frame: 36 months ]
    The red flags for wild-type TTR cardiac amyloidosis are among others carpal tunnel syndrome (CTS), wich can be detected by performing an electromyography (EMG).

  6. Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [ Time Frame: 36 months ]
    The red flags for wild-type TTR cardiac amyloidosis are among others spinal canal stenosis.

  7. Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [ Time Frame: 36 months ]
    The red flags for wild-type TTR cardiac amyloidosis are among others trigger finger.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   60 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05950867


Contacts
Layout table for location contacts
Contact: Véronique Bissay, MD,Phd +32 2 477 68 01 veronique.bissay@uzbrussel.be

Locations
Layout table for location information
Belgium
UZ Brussel Recruiting
Jette, Belgium Capital City, Belgium, 1090
Contact: Veronique Bissay, MD, PhD    +32 2 477 68 01    veronique.bissay@uzbrussel.be   
Contact: Steven Droogmans, MD, PhD    +32 2 477 60 09    steven.droogmans@uzbrussel.be   
Principal Investigator: Véronique Bissay, MD, Phd         
Sub-Investigator: Steven Droogmans, MD, Phd         
Sponsors and Collaborators
Universitair Ziekenhuis Brussel
Investigators
Layout table for investigator information
Principal Investigator: Veronique Bissay, MD, Phd UZ Brussel - VUB
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Publications:

Layout table for additonal information
Responsible Party: Veronique Bissay, Principal Investigator, Universitair Ziekenhuis Brussel
ClinicalTrials.gov Identifier: NCT05950867    
Other Study ID Numbers: EC-2023-115
First Posted: July 18, 2023    Key Record Dates
Last Update Posted: November 7, 2023
Last Verified: November 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Polyneuropathies
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
 Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases