AmyloidosisNCT05375279
A PILOT Study on LSGB vs EMB in the Diagnosis of Cardiac Transthyretin Amyloidosis
An observational study of Amyloidosis, run by Unity Health Toronto at 1 site in Canada.
Trial summary
Transthyretin (TTR) is a plasma protein mainly synthesized in the liver, recognized as a transporter of thyroxine and retinol-binding protein. Unstable changes in two types of TTR (wild type or variant) become misfolded, aggregate, and ultimately forms amyloid fibrils. Read more about this study
- Who is it for?
- Ages 18 to 96 years
- Conditions the study is for
- Amyloidosis
- What is being tested?
- Lip Salivary Gland Biopsy
- Type of treatment
- Surgery or procedure
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- 15 Jun 2022to 31 Mar 2023
- 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 to 96 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).
Who may be able to join
- Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).
Who cannot join
- Patients who had negative amyloid deposits in the endomyocardial biopsy.
Trial locations and contacts
1 site in 1 country.
Canada1 site
London Health Sciences Centre
London, Ontario, N6A 5A5
Heather Hern, 519-685-8500 ext. 32818, heather.hern@lhsc.on.ca
Ryan Davey, MD, FRCPC
About this study
Official title: A PILOT Study on Labial Salivary Gland Biopsy Potentially to Replace Endomyocardial Biopsy in the Diagnosis of Cardiac Transthyretin Amyloidosis
Transthyretin (TTR) is a plasma protein mainly synthesized in the liver, recognized as a transporter of thyroxine and retinol-binding protein. Unstable changes in two types of TTR (wild type or variant) become misfolded, aggregate, and ultimately forms amyloid fibrils. Amyloid Transthyretin Cardiac amyloidosis (ATTR-CA) is an infiltrative cardiomyopathy caused by extracellular deposition of insoluble transthyretin (TTR) amyloid fibrils in the heart muscle. Cardiac amyloidosis (CA) has been recognized as a common cause of heart failure with preserved ejection fraction (HFpEF) among elderly persons, with increasing incidence.
There are different ways of diagnosing ATTR-CA. These include cardiac magnetic resonance imaging, nuclear scintigraphy, and tissue biopsy, the gold standard. Tissues biopsy extracted from the adipose, lip salivary gland (LSG), and heart muscle (endomyocardial biopsy or EMB).
Read more
Tissue diagnosis is the prerequisite of provincial support of the disease-modifying agent. However, with the convenience, ease, less risk of bleeding, and high sensitivity, LSG may offer an alternative to the more invasive EMB to diagnose suspected CA.
To test the hypothesis that LSGB can replace EMB in tissue diagnosis of ATTR-CM.
This Pilot study is designed to evaluate two invasive diagnostic methodologies: LSGB and the EMB. A total of 20 patients who underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will be invited to participate. In addition, patients who signed the informed consent form will be scheduled for LSGB within two weeks.
Read the detailed description
We propose broad eligibility criteria to increase the generalizability and feasibility of the proposed study. Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy) are eligible to participate in the study.The exclusion criteria are predominantly patients who had negative amyloid deposits in the endomyocardial biopsy. There are no exclusions based on gender, race or ethnicity in this trial.
LSG biopsy will be performed under local anesthesia (2% lidocaine). The lip will be everted, and a 3 to 4 mm longitudinal incision will be made in the labial mucosa in front of the mandibular canine tooth. Three minor salivary glands will be exposed and removed. No silk suture will be used. [18] The LSG specimen will match the EMB specimen. LSG biopsy tissues will be studied using the method of Congo red stain.[18] The participants will be scheduled for LSGB at the London Health Sciences Centre within two weeks of signing the informed consent.
- Conditions
- Amyloidosis
- Being tested
- Lip Salivary Gland Biopsy
- Keywords
- Amyloidosis, Cardiomyopathy
- Medical subject headings
- Amyloidosis, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lip Salivary Gland BiopsyProcedure or surgery
Obtaining few salivary glands from the partipants' lip that will be studied using the Congo Red staining method.
| Study group | Role | What they receive |
|---|---|---|
| LSGB vs EMB | Patients who already underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will undergo LSGB. Receives: Procedure: Lip Salivary Gland Biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
LSG biopsy with positive amyloid deposits will be observed in a higher proportion in patients with positive EMB.
Proportions of patients who will test positive and negative will be determined.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2022
- Study start15 Jun 2022 (estimated)
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Mar 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- More trials run by Unity Health TorontoThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Unity Health Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Gordon Moe, Unity Health Toronto
- Investigators
- Gordon W Moe, MD, FRCPC, FACC, Principal investigator, Unity Health Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05375279
- Sponsor's protocol ID
- 70095215
- Status last verified
- May 2022
- First submitted
- 10 May 2022
- Last updated
- 23 May 2022
- 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 23 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A PILOT Study on Labial Salivary Gland Biopsy Potentially to Replace Endomyocardial Biopsy in the Diagnosis of Cardiac Transthyretin Amyloidosis
- ClinicalTrials.gov ID
- NCT05375279
- Acronym / sponsor ID
- 70095215
- Conditions
- Amyloidosis
- Keywords
- Amyloidosis, Cardiomyopathy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Unity Health TorontoUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 15 Jun 2022 (estimated)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Mar 2023 (estimated)
- First posted
- 16 May 2022 (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.
Transthyretin (TTR) is a plasma protein mainly synthesized in the liver, recognized as a transporter of thyroxine and retinol-binding protein. Unstable changes in two types of TTR (wild type or variant) become misfolded, aggregate, and ultimately forms amyloid fibrils. Amyloid Transthyretin Cardiac amyloidosis (ATTR-CA) is an infiltrative cardiomyopathy caused by extracellular deposition of insoluble transthyretin (TTR) amyloid fibrils in the heart muscle. Cardiac amyloidosis (CA) has been recognized as a common cause of heart failure with preserved ejection fraction (HFpEF) among elderly persons, with increasing incidence.
There are different ways of diagnosing ATTR-CA. These include cardiac magnetic resonance imaging, nuclear scintigraphy, and tissue biopsy, the gold standard. Tissues biopsy extracted from the adipose, lip salivary gland (LSG), and heart muscle (endomyocardial biopsy or EMB).
Tissue diagnosis is the prerequisite of provincial support of the disease-modifying agent. However, with the convenience, ease, less risk of bleeding, and high sensitivity, LSG may offer an alternative to the more invasive EMB to diagnose suspected CA.
To test the hypothesis that LSGB can replace EMB in tissue diagnosis of ATTR-CM.
This Pilot study is designed to evaluate two invasive diagnostic methodologies: LSGB and the EMB. A total of 20 patients who underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will be invited to participate. In addition, patients who signed the informed consent form will be scheduled for LSGB within two weeks.
Detailed description
We propose broad eligibility criteria to increase the generalizability and feasibility of the proposed study. Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy) are eligible to participate in the study.The exclusion criteria are predominantly patients who had negative amyloid deposits in the endomyocardial biopsy. There are no exclusions based on gender, race or ethnicity in this trial.
LSG biopsy will be performed under local anesthesia (2% lidocaine). The lip will be everted, and a 3 to 4 mm longitudinal incision will be made in the labial mucosa in front of the mandibular canine tooth. Three minor salivary glands will be exposed and removed. No silk suture will be used. [18] The LSG specimen will match the EMB specimen. LSG biopsy tissues will be studied using the method of Congo red stain.[18] The participants will be scheduled for LSGB at the London Health Sciences Centre within two weeks of signing the informed consent.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LSGB vs EMB | – | Procedure: Lip Salivary Gland BiopsyPatients who already underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will undergo LSGB. |
Who may qualify
- Ages
- 18 to 96 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).
Key inclusion criteria (1 listed)
- Patients who are confirmed diagnosis of Amyloid Transthyretin Cardiomyopathy - wild type (ATTRwt) through Echocardiography, Cardiac Magnetic Resonance Imaging, pyrophosphate scan, and genetic testing and had an endomyocardial biopsy (or will undergo endomyocardial biopsy).
Key exclusion criteria (1 listed)
- Patients who had negative amyloid deposits in the endomyocardial biopsy.
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 |
|---|---|---|
| Lip Salivary Gland BiopsyObtaining few salivary glands from the partipants' lip that will be studied using the Congo Red staining method. | Procedure or surgery | LSGB vs EMB |
Primary outcome
LSG biopsy with positive amyloid deposits will be observed in a higher proportion in patients with positive EMB.
Time frame: 6 months
Proportions of patients who will test positive and negative will be determined.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2022.
- Individual participant dataSharing planned: No
Related trials for Amyloidosis
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Recruiting
Intracoronary Cryotherapy Effect on Stabilization of Vulnerable Plaque in Patients With Stable Angina or ACS - A Traditional Feasibility Study
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Enrolling by invitationPhase 2
Tau PET Outcomes With Anti-amyloid Immunotherapies
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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.
Gordon W Moe, MD, FRCPC, FACC principal investigator
Unity Health Toronto
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 Amyloidosis, run by Unity Health Toronto at 1 site in Canada.
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 23 May 2022. 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 Canada. 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 Gordon W. Moe, MD, FRCPC, FACC (416-864-5192, Gordon.Moe@unityhealth.to), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
LSG biopsy with positive amyloid deposits will be observed in a higher proportion in patients with positive EMB. (time frame: 6 months). 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 23 May 2022.