National, multicenter, epidemiological, longitudinal protocol to investigate the hATTR prevalence in an at-risk population for Hereditary Transthyretin Amyloidosis (hATTR) and subjects diagnosed with hATTR, to monitor the clinical status in TTR positive subjects and to establish hATTR biomarker/s
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Informed consent is obtained from the participant
* The participant is 18 years of age or older
* The participant has no diagnosis of alcoholism according to international guidelines
* The participant has not undergone chemotherapy for any carcinoma
AND
The participant is at risk for hATTR due to two or more the factors listed below:
* cardiomyopathy or polyneuropathy with no obvious etiology atypical Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) or Motor Neuron Disease (MND)
* autonomic dysfunction
* hypertrophic cardiomyopathy or heart failure with preserved ejection fraction Left Ventricular Hypertrophy (LVH)
* bilateral carpal tunnel syndrome
* spinal stenosis or spinal radiculopathy
* gait disorders
* ocular changes involving vitreous opacities
* unexplained weight loss \>5kg
* renal abnormalities
* family history of hATTR
* based on imaging or biopsy suspected for the wild type TTR (ATTR) and not genetically tested for hATTR
OR • The participant is diagnosed with hATTR
OR
• The participant is a 1st or 2nd degree relative of the TTR positive subject
Exclusion Criteria
* Informed consent is not obtained from the participant
* The participant is younger than 18 years of age
* The participant has a diagnosis of alcoholism according to International guidelines
* The participant has undergone chemotherapy for any carcinoma
* The participant is not at risk for hATTR
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Analysis of prevalance of hATTR mutations among a cohort of participants at risk for hATTR.
Timeframe: 4 years
2
To monitor clinical status in TTR positive subjects.