Transthyretin AmyloidosisAMYLOIDOZANCT05814380
The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.
An observational study of Transthyretin Amyloidosis, run by Katarzyna Holcman at 1 site in Poland.
Trial summary
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant… 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
- What is being tested?
- 99mTc-DPD scintigraphy and Collection of blood samples and echocardiography
- Type of treatment
- Surgery or procedure
- How many people
- 100 participants
- Where
- 1 sitePoland
- Study dates
- 4 May 2020to 1 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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: 100 consecutive patients
Who may be able to join
- over 18 years of age,
- providing written informed consent,
- grade 1-3 cardiac retention of 99mTc-DPD in scintigraphic study or a first-degree relative of a patient with ATTR
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
Poland1 site, 1 recruiting
- Recruiting
Department of Cardiac and Vascular Diseases, John Paul II Hospital
Krakow, Lesser Poland Voivodeship, 31-202
Katarzyna Holcman, MD, PhD, katarzyna.holcman@gmail.com
About this study
Official title: The Comparisons of Regional Scintigraphic DPD Uptake Between Patients With Hereditary and Wild Type Cardiac Transthyretin Amyloidosis
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.
Read the detailed description
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.
This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.
This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.
The collected data will be analyzed statistically to verify research hypotheses. Approval from the local Bioethical Committee will be obtained before carrying out the study. All procedures performed are going to be in accordance with the ethical standards of the 1964 Helsinki declaration and its later amendments, or comparable ethical standards.
- Conditions
- Transthyretin Amyloidosis
- Being tested
- 99mTc-DPD scintigraphy, Collection of blood samples and echocardiography
- Keywords
- transthyretin amyloidosis, SPECT
- 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.
99mTc-DPD scintigraphyRadiation
99mTc-DPD scintigraphy
Collection of blood samples and echocardiographyDiagnostic test
Collection of blood samples from a peripheral vein and TTR sequencing. Transthoracic echocardiography.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | grade 1-3 cardiac retention of 99mTc-DPD in scintigraphy Receives: Radiation: 99mTc-DPD scintigraphy; Diagnostic Test: Collection of blood samples and echocardiography | |
| Group 2 | first-degree relative of a patient with ATTR Receives: Radiation: 99mTc-DPD scintigraphy; Diagnostic Test: Collection of blood samples and echocardiography |
What is being measured
The primary outcome is the main question the study is designed to answer.
regional left ventricle 99mTc-DPD uptake
To compare the regional left ventricle 99mTc-DPD uptake among patients with hereditary and wild-type cardiac transthyretin amyloidosis.
Measured: day 1
Secondary outcomes (2)
right ventricular 99mTc-DPD accumulation
To assess prevalence of right ventricular 99mTc-DPD accumulation among patients with hereditary and wild-type cardiac transthyretin amyloidosis.
Measured: day 1
99mTc-DPD cardiac uptake among asymptomatic hereditary transthyretin amyloidosis variants carriers
To evaluate the presence and extent of 99mTc-DPD cardiac uptake among asymptomatic hereditary transthyretin amyloidosis variants carriers.
Measured: day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2023
- Study start4 May 2020
- Primary completion1 Mar 2024 (estimated)
- Study completion1 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- 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
- Family based
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Katarzyna Holcman
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Katarzyna Holcman, John Paul II Hospital, Krakow
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05814380
- Sponsor's protocol ID
- ID#57165999
- Acronym
- AMYLOIDOZA
- Status last verified
- Apr 2023
- First submitted
- 3 Apr 2023
- Last updated
- 19 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Holcman K, Rubis P, Cmiel B, Zabek A, Boczar K, Stepien-Wroniecka A, Graczyk K, Mroz K, Dziewiecka E, Mateusz W, Szczepara S, Kurek M, Gutkowska K, Podolec P, Kostkiewicz M. The left ventricular mechanical dispersion as a marker of disease severity in transthyretin cardiac amyloidosis. Sci Rep. 2025 Nov 27;15(1):42372. doi: 10.1038/s41598-025-26329-x.
PubMed 41309713Holcman K, Rubis P, Podolec P, Ostrowska M, Stepien-Wroniecka A, Graczyk K, Mroz K, Zabek A, Boczar K, Dziewiecka E, Winiarczyk M, Cmiel B, Kurek M, Kostkiewicz M. Transthyretin-related amyloid cardiomyopathy: A single-center experience in southern Poland, an endemic area. Kardiol Pol. 2025;83(9):1039-1052. doi: 10.33963/v.phj.107207. Epub 2025 Jul 11.
PubMed 40643127Holcman K, Rubis P, Cmiel B, Szot W, Stepien-Wroniecka A, Graczyk K, Mroz K, Dziewiecka E, Mateusz W, Szczepara S, Kurek M, Keska M, Podolec P, Kostkiewicz M. [99mTc]Tc-DPD SPECT/CT evaluation of right and left ventricular involvement in cardiac transthyretin amyloidosis. Int J Cardiol. 2025 Jul 15;431:133227. doi: 10.1016/j.ijcard.2025.133227. Epub 2025 Apr 2.
PubMed 40185370Holcman K, Rubis P, Cmiel B, Stepien A, Graczyk K, Mroz K, Szot W, Dziewiecka E, Winiarczyk M, Kurek M, Keska M, Podolec P, Kostkiewicz M. Pre-symptomatic scintigraphic and genetic cascade screening in cardiac transthyretin amyloidosis. Eur J Nucl Med Mol Imaging. 2025 Apr;52(5):1840-1852. doi: 10.1007/s00259-024-06966-6. Epub 2024 Nov 14.
PubMed 39537877
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Comparisons of Regional Scintigraphic DPD Uptake Between Patients With Hereditary and Wild Type Cardiac Transthyretin Amyloidosis
- ClinicalTrials.gov ID
- NCT05814380
- Acronym / sponsor ID
- AMYLOIDOZA / ID#57165999
- Conditions
- Transthyretin Amyloidosis
- Keywords
- transthyretin amyloidosis, SPECT
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Katarzyna HolcmanUniversity, hospital or other
- Study start
- 4 May 2020 (actual)
- Primary completion
- 1 Mar 2024 (estimated)
- Study completion
- 1 Mar 2024 (estimated)
- First posted
- 14 Apr 2023 (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.
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.
Detailed description
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis. The disease is caused by misfolding of the liver-derived precursor protein transthyretin as a result of an acquired wild-type variant (ATTRwt) or as a hereditary mutant variant (ATTRm). Application of single-photon emission computed tomography (SPECT) provides greater anatomic resolution, enabling the assessment of amyloid burden within individual left ventricle segments.
This study aims to describe the pattern of regional myocardial distribution of 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) SPECT uptake among patients with ATTRwt and ATTRm. It will investigate the clinical, biochemical and echocardiographic, including left ventricle longitudinal strain profile in ATTRwt and ATTRm. Moreover, we will evaluate the presence and extent of DPD cardiac uptake among asymptomatic ATTRm variants carriers.
This is a prospective multi-center observational study. The study, after obtaining prior written informed consent, will include consecutive patients who have Grade 1-3 cardiac DPD retention in scintigraphy. In addition, first-degree relatives of patients with ATTRm are going to be enrolled. Patients are going to undergo TTR gene sequencing to assess the presence of pathogenic variants associated with ATTRm. Both planar scintigraphy, SPECT and speckle-tracking echocardiography will be reviewed and interpreted using visual and quantitative approaches.
The collected data will be analyzed statistically to verify research hypotheses. Approval from the local Bioethical Committee will be obtained before carrying out the study. All procedures performed are going to be in accordance with the ethical standards of the 1964 Helsinki declaration and its later amendments, or comparable ethical standards.
Study design
- Observational model
- Family based
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Radiation: 99mTc-DPD scintigraphy, Diagnostic Test: Collection of blood samples and echocardiographygrade 1-3 cardiac retention of 99mTc-DPD in scintigraphy |
| Group 2 | – | Radiation: 99mTc-DPD scintigraphy, Diagnostic Test: Collection of blood samples and echocardiographyfirst-degree relative of a patient with ATTR |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- 100 consecutive patients
Key inclusion criteria (3 listed)
- over 18 years of age,
- providing written informed consent,
- grade 1-3 cardiac retention of 99mTc-DPD in scintigraphic study or a first-degree relative of a patient with ATTR
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 99mTc-DPD scintigraphy99mTc-DPD scintigraphy | Radiation | Group 1, Group 2 |
| Collection of blood samples and echocardiographyCollection of blood samples from a peripheral vein and TTR sequencing. Transthoracic echocardiography. | Diagnostic test | Group 1, Group 2 |
Primary outcome
regional left ventricle 99mTc-DPD uptake
Time frame: day 1
To compare the regional left ventricle 99mTc-DPD uptake among patients with hereditary and wild-type cardiac transthyretin amyloidosis.
2 secondary outcomes
right ventricular 99mTc-DPD accumulation
Time frame: day 1
99mTc-DPD cardiac uptake among asymptomatic hereditary transthyretin amyloidosis variants carriers
Time frame: day 1
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2023.
- Derived from this study: PubMed 41309713Holcman K, Rubis P, Cmiel B, Zabek A, Boczar K, Stepien-Wroniecka A, Graczyk K, Mroz K, Dziewiecka E, Mateusz W, Szczepara S, Kurek M, Gutkowska K, Podolec P, Kostkiewicz M. The left ventricular mechanical dispersion as a marker of disease severity in transthyretin cardiac amyloidosis. Sci Rep. 2025 Nov 27;15(1):42372. doi: 10.1038/s41598-025-26329-x.
- Derived from this study: PubMed 40643127Holcman K, Rubis P, Podolec P, Ostrowska M, Stepien-Wroniecka A, Graczyk K, Mroz K, Zabek A, Boczar K, Dziewiecka E, Winiarczyk M, Cmiel B, Kurek M, Kostkiewicz M. Transthyretin-related amyloid cardiomyopathy: A single-center experience in southern Poland, an endemic area. Kardiol Pol. 2025;83(9):1039-1052. doi: 10.33963/v.phj.107207. Epub 2025 Jul 11.
- Derived from this study: PubMed 40185370Holcman K, Rubis P, Cmiel B, Szot W, Stepien-Wroniecka A, Graczyk K, Mroz K, Dziewiecka E, Mateusz W, Szczepara S, Kurek M, Keska M, Podolec P, Kostkiewicz M. [99mTc]Tc-DPD SPECT/CT evaluation of right and left ventricular involvement in cardiac transthyretin amyloidosis. Int J Cardiol. 2025 Jul 15;431:133227. doi: 10.1016/j.ijcard.2025.133227. Epub 2025 Apr 2.
- Derived from this study: PubMed 39537877Holcman K, Rubis P, Cmiel B, Stepien A, Graczyk K, Mroz K, Szot W, Dziewiecka E, Winiarczyk M, Kurek M, Keska M, Podolec P, Kostkiewicz M. Pre-symptomatic scintigraphic and genetic cascade screening in cardiac transthyretin amyloidosis. Eur J Nucl Med Mol Imaging. 2025 Apr;52(5):1840-1852. doi: 10.1007/s00259-024-06966-6. Epub 2024 Nov 14.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
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, run by Katarzyna Holcman at 1 site in Poland.
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 3 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 19 Apr 2023. 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 Poland. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Katarzyna Holcman, MD, PhD (+48 12 614 22 87, katarzyna.holcman@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
regional left ventricle 99mTc-DPD uptake (time frame: day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05814380, which also lists 4 linked publications.
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 19 Apr 2023.