RecruitingNCT05760287
Cardiovascular
Transthyretin Amyloidosis
Also known as ATTR amyloidosis, hATTR, familial amyloid polyneuropathy, TTR amyloidosis
Transthyretin Amyloidosis is caused by misfolding and extracellular deposition of amyloid fibrils derived from transthyretin, a liver-produced transport protein, in multiple organs including the heart, peripheral nerves, and carpal tunnel.
ORPHA:85451 ↗Gene TTRPrevalence Wild-type: estimated 1–5 per 10,000 over age 65; hereditary: varies by variantOnset Adulthood; hereditary form typically 30–60; wild-type after 60Autosomal dominant (hereditary); acquired (wild-type)
87
studies recruiting now
as of 7 Sept 2026
346
studies registered in total
as of 7 Sept 2026
3
countries with a recruiting site
as of 7 Sept 2026
7 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT07752732
Non-Interventional Study on the Prevalence of Cardiac Amyloidosis in Patients With Higher-Grade Aortic Valve Stenosis - Evaluation Using Echocardiography, Computed Tomography, Tc99-SPECT/CT and Cardiac Magnetic Resonance Imaging (CMR)
RecruitingNot applicableNCT05577819
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
RecruitingNCT05489549
Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers
RecruitingNCT07205666
The Eplontersen Pregnancy and Lactation Outcomes Study
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 87 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Transthyretin Amyloidosis
Transthyretin Amyloidosis is caused by misfolding and extracellular deposition of amyloid fibrils derived from transthyretin, a liver-produced transport protein, in multiple organs including the heart, peripheral nerves, and carpal tunnel. Hereditary ATTR (hATTR), caused by autosomal dominant TTR mutations such as Val30Met and Val122Ile, presents with polyneuropathy and/or cardiomyopathy depending on the variant, while wild-type ATTR (ATTRwt) causes cardiomyopathy exclusively and is substantially underdiagnosed in elderly men with heart failure with preserved ejection fraction. Disease-modifying therapies including TTR stabilisers (tafamidis) and RNA-silencing agents (patisiran, vutrisiran) have transformed the management landscape.
Common clinical features
Cardiac restrictive physiology with heart failure symptomsPeripheral sensorimotor neuropathy (in hATTR)Autonomic neuropathy (orthostatic hypotension, GI dysmotility)Carpal tunnel syndrome, often bilateral and preceding diagnosis by yearsLumbar spinal stenosisIncreased left ventricular wall thickness with preserved or reduced EFDiscordant low-voltage ECG relative to echocardiographic wall thicknessIncreased myocardial uptake on technetium pyrophosphate bone scan
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Patisiran
Before you apply
Things trial teams commonly ask about for Transthyretin Amyloidosis. Not eligibility rules; those are set by each study.
- Tissue biopsy with amyloid typing (Congo red staining and immunohistochemistry or mass spectrometry) or positive bone scan with genetic TTR testing is required to confirm ATTR vs. AL amyloidosis, a critical distinction for trial eligibility.
- Current or prior use of tafamidis or RNA-silencing therapy may influence eligibility for interventional trials; a detailed treatment history with dates is essential.
- Functional capacity (6-minute walk test distance and NYHA class) and biomarkers (NT-proBNP, troponin) are standard inclusion and stratification criteria; recent results should be available.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).