Transthyretin AmyloidosisNCT06291805
Phenotyping and Characterization of wtATTR-CM (TRACE 1)
An observational study of Transthyretin Amyloidosis and Transthyretin Amyloid Cardiomyopathy, run by Steen Hvitfeldt Poulsen at 1 site in Denmark.
Trial summary
Descriptive cross-sectional study on 100 consecutive ATTRwt-CM patients reflecting all NAC stages aiming primarily to investigate ATTRwt-CM patient's quality of life (QoL) measures and their relation to ATTRwt-CM severity. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Wild-Type Transthyretin-Related (ATTR)Amyloidosis
- How many people
- 120 participants
- Where
- 1 siteDenmark
- Study dates
- 20 Feb 2024to Dec 2025
- Phase
- Observational
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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Including variating severity of wtATTR-CM by including 40 patients with NAC stage 1, 40 patients with NAC stage 2, and 20 patients with NAC stage 3. Furthermore, including a healthy control group in order to examine normal values of mis- and frag-TTR
Who may be able to join
- Patients > 18 years diagnosed with ATTRwt-CM by:
- endomyocardial biopsy
- DPD scintigraphy with Perugini grade 2-3 where variant amyloidosis is ruled out due to genetic testing.
- Informed oral and written consent
- Patients > 18 years
- Informed oral and written consent
Who cannot join
- AL amyloidosis (light-chain amyloidosis).
- Myelomatosis
- Waldenström macroglobulinemia
- Group 2: Control group
- Known cardiovascular disease including ischemic heart disease, heart failure, atrial fibrillation, presence of a pacemaker, or malignant hypertension. Well-controlled hypertension is acceptable.
- Suspicion of cardiac amyloidosis assessed through clinical history, physical examination, ECG, and echocardiography focusing on "red flags":
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- Echocardiography with:
- Myocardial hypertrophy (septum >11 mm)
- Apical sparing in LV-GLS
- Infiltrative changes in the right ventricle free wall, thickened atrioventricular valves, or thickened atrial septum
- Symptoms of polyneuropathy
- Low voltage on ECG or discrepancy between left ventricular thickness and ECG amplitude indicative of low voltage
- Atrioventricular block (AV block)
- Bilateral carpal tunnel syndrome
- Surgery for spinal stenosis
- Elevated troponin I or NT-pro-BNP
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Denmark1 site, 1 recruiting
- Recruiting
Aarhus University Hospital
Aarhus, Arrhus N, 8200
Sie Kronborg Fensman, +45 30 48 88 85, siefensman@gmail.com
About this study
Official title: Phenotyping and Characterization of Danish Wild-type Transthyretin Amyloidosis Cardiomyopathy Patients: A Cross-sectional Study
Descriptive cross-sectional study on 100 consecutive ATTRwt-CM patients reflecting all NAC stages aiming primarily to investigate ATTRwt-CM patient's quality of life (QoL) measures and their relation to ATTRwt-CM severity. Secondarily aiming to investigate the possibility to measure misTTR and fragTTR in plasma and urine and to detect fragTTR in endomyocardial biopsies from ATTRwt-CM patients. To investigate whether misTTR and fragTTR levels are correlated with ATTRwt-CM severity.
Read the detailed description
Hypothesis:
- We hypothesize that more severe wild-type amyloidosis cardiomyopathy (ATTRwt-CM) according to clinical, biochemical, and diagnostic imaging parameters are correlated with worse quality of life (QoL) for patients.
- We expect misfolded (misTTR) and/or fragmented transthyretin (fragTTR) to be measurable in plasma and/or urine and fragTTR to be detectable in endomyocardial biopsies from patients with ATTRwt-CM. We expect the values of misTTR and fragTTR to be correlated with the severity of ATTRwt-CM according to clinical, biochemical, and diagnostic imaging parameters. We expect the level of fragTTR from endomyocardial biopsies to be correlated with plasma levels of fragTTR.
Method:
ATTRwt-CM patients: Prospective inclusion of 100 consecutive ATTRwt-CM patients reflecting all NAC stages (40 patients from NAC disease stage 1, 40 patients from NAC disease stage II and 20 patients from NAC disease stage III). Patients will be recruited from the out-patient amyloidosis clinic at Aarhus University hospital. Patients will be thoroughly clinically assessed.
Control patients:
A control cohort of 20 age- and gender-matched heart-healthy patients will be included for comparison of total/mis-/fragTTR values.
The investigating into QoL, bio markers and the analyses on cardiac MR imaging markers will hopefully provide us with tools to evaluate and monitor disease progression and response to treatment.
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Quality of Life
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| wtATTR-CM patients (n = 100) | Clinical history, physical examination, ECG, advanced echocardiography, blood and urine samples, cardiac magnetic resonance imaging (CMRI), and QoL questionaires (KCCQ, EQ-5Q-5L and ATTR-QoL)). Subgroup with these patient (n = 10): A endomyocardial biopsy. | |
| Control (n = 20) | Blood and urine samples. To rule out cardiac disease: clinical history, physical examination, ECG and advanced echocardiography. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigation of cardiac amyloidosis severity in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy.
Amyloid severity will be addressed using primarily NAC-stages and NYHA-class. Furthermore, we will use NT-pro-BNP, findings on echo (stroke volume, ejectionfraction and global longitudional strain) and finding on CMRI (LV mass, T1 myocardial values and extracellular volume).
Measured: Through study completion, 2 years.
Investigation of the relations between cardiac amyloid severity and patient quality of life in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy.
Quality of life will be addressed using KCCQ summary score, EQ-5D Utility score and VAS ranging score and ATTR-QOL total score.
Measured: Through study completion, 2 years.
Assessment of transthyretin and pathogenic fragments and relation to cardiac amyloid severity in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy
Transthyretin (TTR) will be measured as follows: total TTR, misfolded TTR and fragmented TTR in plasma and misfolded TTR and fragmented TTR in urine.
Measured: Through study completion, 2 years.
Secondary outcomes (1)
Validation of a new amyloid specific questionnaire (ATTR-QoL) in comparison with Kansas City Cardiomyopathy Questionnaire (KCCQ)
Comparing ATTR-QOL total score to KCCQ summary score and EQ-5D Utility score and VAS ranging score.
Measured: Through study completion, 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2024
- Study start20 Feb 2024
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Steen Hvitfeldt Poulsen
- Sponsor type
- University, hospital or other
- Collaborators
- Novo Nordisk A/S
- Responsible party
- Sponsor-investigator, Steen Hvitfeldt Poulsen, Aarhus University Hospital Skejby
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06291805
- Sponsor's protocol ID
- 1-10-72-189-23
- Status last verified
- Aug 2025
- First submitted
- 30 Jan 2024
- Last updated
- 15 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No