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
- Are there results?
- Not yet: the study is under way
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":
Show all 16Show fewer
- 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
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phenotyping and Characterization of Danish Wild-type Transthyretin Amyloidosis Cardiomyopathy Patients: A Cross-sectional Study
- ClinicalTrials.gov ID
- NCT06291805
- Acronym / sponsor ID
- 1-10-72-189-23
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Quality of Life
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Steen Hvitfeldt PoulsenUniversity, hospital or other
- Collaborators
- Novo Nordisk A/S
- Study start
- 20 Feb 2024 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 4 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- 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
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Investigation of cardiac amyloidosis severity in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy.
Time frame: Through study completion, 2 years.
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).
Investigation of the relations between cardiac amyloid severity and patient quality of life in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy.
Time frame: Through study completion, 2 years.
Quality of life will be addressed using KCCQ summary score, EQ-5D Utility score and VAS ranging score and ATTR-QOL total score.
Assessment of transthyretin and pathogenic fragments and relation to cardiac amyloid severity in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy
Time frame: Through study completion, 2 years.
Transthyretin (TTR) will be measured as follows: total TTR, misfolded TTR and fragmented TTR in plasma and misfolded TTR and fragmented TTR in urine.
1 secondary outcome
Validation of a new amyloid specific questionnaire (ATTR-QoL) in comparison with Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time frame: Through study completion, 2 years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2025.
- Individual participant dataSharing planned: No
Related trials for Transthyretin Amyloidosis
Not yet recruitingObservational study
Single-arm, Multi-center Study Evaluating the Acceptance and Feasibility of a Digital Remote Monitoring Tool Configured for Patients With ATTR-CM: ACO-Monitor
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Not yet recruitingPhase 2
Effect of Trimetazidine on Coronary Flow in Transthyretin Cardiac Amyloidosis
Transthyretin Cardiac Amyloidosis
Not yet recruitingObservational study
PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
Active, not recruitingPhase 3
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Transthyretin Amyloid Cardiomyopathy
Enrolling by invitationPhase 3
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloidosis and Transthyretin Amyloid Cardiomyopathy, run by Steen Hvitfeldt Poulsen at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sie Kronborg Fensman, MD (+45 30 48 88 85, siefensman@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Investigation of cardiac amyloidosis severity in patients with Wild-type Transthyretin Amyloidosis Cardiomyopathy. (time frame: Through study completion, 2 years.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2025.