Clinical trial NCT06338696: Thailand ATTR-CM Registry
Official title: Thailand Transthyretin Amyloid Cardiomyopathy Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Keywords: Transthyretin amyloid cardiomyopathy (ATTR-CM)
Heart Failure
Cardiomyopathy
Lead sponsor: Mahidol University
Sponsor class: OTHER
Collaborators: name: Pfizer
First posted: 2024-04-01
Enrollment: 105
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients diagnosed with ATTR-CM,Transthyretin cardiomyopathy, who have either symptomatic or asymptomatic.

Brief summary: The investigators of this registry aim to study the natural history, clinical presentation, characteristics, and imaging findings of patients diagnosed with ATTR amyloidosis in Thailand longitudinally. This will enable the investigators to study this disease in depth. With a better understanding, the investigators can aim to develop early screening programs for at-risk patients, raising awareness among non-cardiologists. As amyloid-specific therapies have now become available in Thailand, the findings of this registry can be helpful for epidemiological studies in Thailand.


Eligibility criteria: Inclusion Criteria:

* Age \> 18 years.
* All patients diagnosed with ATTR-CM (Transthyretin cardiomyopathy) who have either symptomatic or asymptomatic must have evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of ≥ 12 mm and with at least one of the following criteria:
* Positive scintigraphy by 99mTC-DPD \[99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid\] or 99mTC-PYP \[Pyrophosphate\] or 99mTC-HMDP \[hydroxymethylene diphosphate\] with Perugini grade ≥ 2 and/or H/CL (heart to contralateral lung) ratio \> 1.5
* Positive scintigraphy by 99mTC-DPD \[99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid\] or 99mTC-PYP \[Pyrophosphate\] or 99mTC-HMDP \[hydroxymethylene diphosphate\] with Perugini grade 1 and positive cardiac biopsy tissue confirmed which Congo red stain ≥ 4 sites
* Positive scintigraphy by 99mTC-DPD \[99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid\] or 99mTC-PYP \[Pyrophosphate\] or 99mTC-HMDP \[hydroxymethylene diphosphate\] with Perugini grade 1 with positive non-cardiac biopsy tissue confirmed by Congo red stain
* Abnormalities on CMR (Cardiac MRI) \[Subendocardium LGE (late gadolinium enhancement) and/or elevated native T1 value and/or increased extracellular volume (ECV \> 0.4)\] with positive non-cardiac and/or cardiac tissue biopsy confirmed by Congo red stain

Exclusion Criteria:

* Diagnosed with amyloidosis caused by proteins other than transthyretin (light chain Amyloidosis, etc.)
* Diagnosed with secondary amyloidosis

Primary outcomes:
measure: Rate of death
timeFrame: 3 years
measure: Rate of heart failure hospitalization
timeFrame: 3 years
Secondary outcomes:
measure: Transthyretin (ATTR) amyloidosis medication
timeFrame: 3 years
Other outcomes:
Locations:
city: Bangkok
country: Thailand
facility: Faculty of Medicine at Chulalongkorn University
city: Bangkok
country: Thailand
facility: Faculty of Medicine Ramathibodi hospital, Mahidol University
city: Bangkok
country: Thailand
facility: Faculty of Medicine Siriraj Hospital, Mahidol University
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Chiang Mai
country: Thailand
facility: Faculty of Medicine Chiang Mai University
city: Khon Kaen
country: Thailand
facility: Queen Sirikit Heart Center of The Northeast
city: Pathum Thani
country: Thailand
facility: Faculty of Medicine, Thammasat University
city: Songkhla
country: Thailand
facility: Prince of Songkla University
Study officials:
affiliation: Division of Cardiology, Department of Medicine, Siriraj Hospital, Mahidol University
name: Rungroj Krittayaphong, MD, FESC, FACC
role: PRINCIPAL_INVESTIGATOR
References: