Transthyretin Cardiomyopathy (TTR-CM)NCT06714019
PL_GNT01_ISR_Grant 53234273
An observational study of Transthyretin Cardiomyopathy (TTR-CM) and Transthyretin (TTR) Amyloid Cardiomyopathy, run by National Institute of Cardiology, Warsaw, Poland at 1 site in Poland.
Trial summary
We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Cardiomyopathy (TTR-CM), Transthyretin (TTR) Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- How many people
- 100 participants
- Where
- 1 sitePoland
- Study dates
- 11 Feb 2019to Nov 2025
- How long will I take part?
- 12 Months
- 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
- No healthy volunteers
- Adult, Older adult
Study population: All individuals with wild-type TTR amyloidosis and patients with a confirmed TTR mutation with or without a diagnosis of TTR amyloidosis will be eligible to be enrolled in the registry. Enrolment is voluntary and dependent on each subject's written informed consent.
Who may be able to join
- Adults over 18 years old with confirmed diagnosis of TTR amyloidosis
Who cannot join
- Refusal to participate in the study. Light-chain amyloidosis.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: WI241665 2018 GLOBAL ASPIRE TTR Amyloidosis _ Transthyretin Amyloidosis National Registry - a Prospective Non-interventional, Longitudinal, Observational Multicentre Study
We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country. The main objective of the proposed study is to determine the occurrence of TTR amyloidosis and describe clinical profile of patients in the population of our country.
Read the detailed description
Demographic information, TTR genotype, medical history, family history of the disease, and transplant history are assessed at baseline. On return visits, signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.
Specific Aims and Hypotheses
Our working hypotheses are:
- TTR amyloidosis affects patients in the population of our country;
- there are specific TTR mutations in the population of our country;
- there is genotype-phenotype relationship in hereditary TTR amyloidosis;
- there are risk factors for TTR amyloidosis in the population of our country. These hypotheses will be tested in our specific aims. In aim 1. we will describe the occurrence of TTR amyloidosis in the population of our country, including the hereditary and acquired forms of the disease.
In aim 2. we will determine and characterize high frequency TTR mutations in the population of our country.
In aim 3. we will determine a clinical profile of patients and we will try to enhance understanding of the natural history of TTR amyloidosis, including the variability, progression of the disease, and predisposing factors. We will evaluate patients' quality of life.
In aim 4. we will search for genotype-phenotype relationship in hereditary TTR amyloidosis.
In aim 5. we will evaluate effects of liver transplantation and other treatments on disease progression in our patients. We will advance knowledge of the disease to optimize the assessment, treatment and monitoring of patients.
In aim 6. we will formulate novel hypotheses for further prospective studies. We will form a community of medical experts on amyloidosis (cardiologists, neurologist, internal medicine physicians, as well as other specialists) to create in the future national centre of amyloidosis in our country that would offer the highest standard of care and gather clinical data on this rare disease.
- Conditions
- Transthyretin Cardiomyopathy (TTR-CM), Transthyretin (TTR) Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Keywords
- transthyretin, transthyretin amyloidosis, ATTR, Transthyretin amyloid cardiomyopathy
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Transthyretin-Related
What is being measured
The primary outcome is the main question the study is designed to answer.
disease progression
Signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.
Measured: From enrollment for at least 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2024
- Study start11 Feb 2019
- Primary completionOct 2025 (estimated)
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Cardiomyopathy (TTR-CM)Studies 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- National Institute of Cardiology, Warsaw, Poland
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Monika Gawor-Prokopczyk, National Institute of Cardiology, Warsaw, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06714019
- Sponsor's protocol ID
- 53234273
- Status last verified
- May 2024
- First submitted
- 27 Nov 2024
- Last updated
- 3 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- 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 3 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- WI241665 2018 GLOBAL ASPIRE TTR Amyloidosis _ Transthyretin Amyloidosis National Registry - a Prospective Non-interventional, Longitudinal, Observational Multicentre Study
- ClinicalTrials.gov ID
- NCT06714019
- Acronym / sponsor ID
- 53234273
- Conditions
- Transthyretin Cardiomyopathy (TTR-CM), Transthyretin (TTR) Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Keywords
- transthyretin, transthyretin amyloidosis, ATTR, Transthyretin amyloid cardiomyopathy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- National Institute of Cardiology, Warsaw, PolandUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 11 Feb 2019 (actual)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Nov 2025 (estimated)
- First posted
- 3 Dec 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.
We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country. The main objective of the proposed study is to determine the occurrence of TTR amyloidosis and describe clinical profile of patients in the population of our country.
Detailed description
Demographic information, TTR genotype, medical history, family history of the disease, and transplant history are assessed at baseline. On return visits, signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.
Specific Aims and Hypotheses
Our working hypotheses are:
- TTR amyloidosis affects patients in the population of our country;
- there are specific TTR mutations in the population of our country;
- there is genotype-phenotype relationship in hereditary TTR amyloidosis;
- there are risk factors for TTR amyloidosis in the population of our country. These hypotheses will be tested in our specific aims. In aim 1. we will describe the occurrence of TTR amyloidosis in the population of our country, including the hereditary and acquired forms of the disease.
In aim 2. we will determine and characterize high frequency TTR mutations in the population of our country.
In aim 3. we will determine a clinical profile of patients and we will try to enhance understanding of the natural history of TTR amyloidosis, including the variability, progression of the disease, and predisposing factors. We will evaluate patients' quality of life.
In aim 4. we will search for genotype-phenotype relationship in hereditary TTR amyloidosis.
In aim 5. we will evaluate effects of liver transplantation and other treatments on disease progression in our patients. We will advance knowledge of the disease to optimize the assessment, treatment and monitoring of patients.
In aim 6. we will formulate novel hypotheses for further prospective studies. We will form a community of medical experts on amyloidosis (cardiologists, neurologist, internal medicine physicians, as well as other specialists) to create in the future national centre of amyloidosis in our country that would offer the highest standard of care and gather clinical data on this rare disease.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 12 Months
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All individuals with wild-type TTR amyloidosis and patients with a confirmed TTR mutation with or without a diagnosis of TTR amyloidosis will be eligible to be enrolled in the registry. Enrolment is voluntary and dependent on each subject's written informed consent.
Key inclusion criteria (1 listed)
- Adults over 18 years old with confirmed diagnosis of TTR amyloidosis
Key exclusion criteria (1 listed)
- Refusal to participate in the study. Light-chain amyloidosis.
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
disease progression
Time frame: From enrollment for at least 12 month
Signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2024.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Cardiomyopathy (TTR-CM) and Transthyretin (TTR) Amyloid Cardiomyopathy, run by National Institute of Cardiology, Warsaw, Poland 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 1 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 3 Dec 2024. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
disease progression (time frame: From enrollment for at least 12 month). 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 3 Dec 2024.