Amyloidosis CardiacNCT04985734
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy
A study testing Specific diagnostic work-up for ATTR-CM patients in people with Amyloidosis Cardiac and Amyloid Polyneuropathy, run by University Hospital, Antwerp at 1 site in Belgium.
Trial summary
The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a state-of-the-art diagnostic work-up; the investigators believe that the identification of patients… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis
- What is being tested?
- Specific diagnostic work-up for ATTR-CM patients
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteBelgium
- Study dates
- 1 Oct 2021to 1 Jul 2025
- Phase
- Interventional
- 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
Who may be able to join
- Adult patients (> 18 years old) with diagnosis of idiopathic peripheral neuropathy
Who cannot join
- Patients with alternative established diagnosis of heart disease (ischemic heart disease, primary valvular heart disease, cardiomyopathies, pericardial disease)
- Patients with end stage renal disease undergoing dialysis
- Patients with other medical conditions recognized to impair the 1-year prognosis of patients
Trial locations and contacts
1 site in 1 country.
Belgium1 site
Emeline van Craenenbroek
Edegem, Antwerp, 2610
Victor S Issa, victorsarli.issa@uza.br
Emelin Van Craenenbroek, Prof
About this study
Official title: Study on the Occurrence of Transthyretin Amyloidosis Cardiomyopathy in Patients With Idiopathic Peripheral Neuropathy
The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a state-of-the-art diagnostic work-up; the investigators believe that the identification of patients with ATTR-CM in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiation of appropriate therapy with impact on prognosis of patients.
Read the detailed description
Background. Different studies have described the value of amyloidosis screening in different clinical settings. The investigators hypothesize that in a relevant yet undetermined proportion of patients with idiopathic peripheral neuropathy a concomitant diagnosis of transthyretin amyloidosis cardiomyopathy can be made with a modern diagnostic work-up based on Technetium-scintigraphy as the primary screening tool. Further the investigators believe that the identification of these patients can lead to early initiation of therapy and modification of the prognosis of the disease.
Objective. To study the occurrence of transthyretin amyloidosis cardiomyopathy in patients with a diagnosis of idiopathic peripheral neuropathy using Tc-scinfigraphy as the primary screening tool.
Methods. This will be a prospective longitudinal cohort of adult patients with the diagnosis of idiopathic peripheral neuropathy. All patients will be submitted to a cardiologic evaluation that will include clinical evaluation, laboratory tests, electrocardiogram and echocardiography. For the diagnosis of amyloidosis all patients will be submitted to 99mTc-Methyl-Diphosphonate scintigraphy. Those with a positive result (grade 2 to 3 cardiac uptake) will be considered as having diagnosis of transthyretin amyloidosis cardiomyopathy and will be further submitted to genetic testing for transthyretin mutation; patients with abnormal scintigraphic results that do not meet current semi-quantitative diagnostic criteria will be submitted to additional investigation, that will include magnetic resonance heart imaging and fat biopsy. All patients will receive standard care for heart failure and transthyretin amyloidosis cardiomyopathy and will be follow-up every 6 months with clinical parameters, quality life questionnaire, ergospirometer and echocardiogram Perspectives. The investigators believe that the identification of patients with transthyretin amyloidosis cardiomyopathy in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiate appropriate therapy with impact on prognosis of patients.
- Conditions
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure, Cardiomyopathy Non-Dilated, Cardiomyopathies, Secondary
- Being tested
- Specific diagnostic work-up for ATTR-CM patients
- Keywords
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure, Cardiomyopathy
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Heart Failure, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Specific diagnostic work-up for ATTR-CM patientsDiagnostic test
Technetium-scintigraphy
| Study group | Role | What they receive |
|---|---|---|
| Patients under diagnostic work-up | Experimental | Not described Receives: Diagnostic Test: Specific diagnostic work-up for ATTR-CM patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
Diagnosis yield
Diagnosis yield of Tc-scintigraphy as a screening tool for ATTR-CM, as measured by the percentage of patients with a confirmed diagnosis of ATTR-CM in relation to the whole population of patients studied
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2021
- Study start1 Oct 2021 (estimated)
- Primary completion1 Jan 2025 (estimated)
- Study completion1 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloidosis CardiacStudies of the same condition, open and under way.
- More trials run by University Hospital, AntwerpThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Cohort of patients
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Antwerp
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04985734
- Sponsor's protocol ID
- EDGE1850
- Status last verified
- Jul 2021
- First submitted
- 17 May 2021
- Last updated
- 2 Aug 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study on the Occurrence of Transthyretin Amyloidosis Cardiomyopathy in Patients With Idiopathic Peripheral Neuropathy
- ClinicalTrials.gov ID
- NCT04985734
- Acronym / sponsor ID
- EDGE1850
- Conditions
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure, Cardiomyopathy Non-Dilated, Cardiomyopathies, Secondary
- Keywords
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure, Cardiomyopathy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- University Hospital, AntwerpUniversity, hospital or other
- Study start
- 1 Oct 2021 (estimated)
- Primary completion
- 1 Jan 2025 (estimated)
- Study completion
- 1 Jul 2025 (estimated)
- First posted
- 2 Aug 2021 (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.
The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a state-of-the-art diagnostic work-up; the investigators believe that the identification of patients with ATTR-CM in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiation of appropriate therapy with impact on prognosis of patients.
Detailed description
Background. Different studies have described the value of amyloidosis screening in different clinical settings. The investigators hypothesize that in a relevant yet undetermined proportion of patients with idiopathic peripheral neuropathy a concomitant diagnosis of transthyretin amyloidosis cardiomyopathy can be made with a modern diagnostic work-up based on Technetium-scintigraphy as the primary screening tool. Further the investigators believe that the identification of these patients can lead to early initiation of therapy and modification of the prognosis of the disease.
Objective. To study the occurrence of transthyretin amyloidosis cardiomyopathy in patients with a diagnosis of idiopathic peripheral neuropathy using Tc-scinfigraphy as the primary screening tool.
Methods. This will be a prospective longitudinal cohort of adult patients with the diagnosis of idiopathic peripheral neuropathy. All patients will be submitted to a cardiologic evaluation that will include clinical evaluation, laboratory tests, electrocardiogram and echocardiography. For the diagnosis of amyloidosis all patients will be submitted to 99mTc-Methyl-Diphosphonate scintigraphy. Those with a positive result (grade 2 to 3 cardiac uptake) will be considered as having diagnosis of transthyretin amyloidosis cardiomyopathy and will be further submitted to genetic testing for transthyretin mutation; patients with abnormal scintigraphic results that do not meet current semi-quantitative diagnostic criteria will be submitted to additional investigation, that will include magnetic resonance heart imaging and fat biopsy. All patients will receive standard care for heart failure and transthyretin amyloidosis cardiomyopathy and will be follow-up every 6 months with clinical parameters, quality life questionnaire, ergospirometer and echocardiogram Perspectives. The investigators believe that the identification of patients with transthyretin amyloidosis cardiomyopathy in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiate appropriate therapy with impact on prognosis of patients.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Cohort of patients
| Arm | Type | Receives |
|---|---|---|
| Patients under diagnostic work-up | Experimental | Diagnostic Test: Specific diagnostic work-up for ATTR-CM patients |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Adult patients (> 18 years old) with diagnosis of idiopathic peripheral neuropathy
Key exclusion criteria (3 listed)
- Patients with alternative established diagnosis of heart disease (ischemic heart disease, primary valvular heart disease, cardiomyopathies, pericardial disease)
- Patients with end stage renal disease undergoing dialysis
- Patients with other medical conditions recognized to impair the 1-year prognosis of patients
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 |
|---|---|---|
| Specific diagnostic work-up for ATTR-CM patientsTechnetium-scintigraphy | Diagnostic test | Patients under diagnostic work-up |
Primary outcome
Diagnosis yield
Time frame: 1 year
Diagnosis yield of Tc-scintigraphy as a screening tool for ATTR-CM, as measured by the percentage of patients with a confirmed diagnosis of ATTR-CM in relation to the whole population of patients studied
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2021.
- Individual participant dataSharing planned: Undecided
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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?
A study testing Specific diagnostic work-up for ATTR-CM patients in people with Amyloidosis Cardiac and Amyloid Polyneuropathy, run by University Hospital, Antwerp at 1 site in Belgium.
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 3 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 2 Aug 2021. 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 Belgium. 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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnosis yield (time frame: 1 year). 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 2 Aug 2021.