Pathophysiological impacts of tafamidis on cardiac transthyretin amyloidosis

JRCT ID: jRCT1041200007

Registered date:28/04/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Transthyretin amyloidosis
• Date of first enrollment: 02/10/2020
• Target sample size: 20
• Study type: Interventional
• Intervention(s): To prescribe tafamidis for 1 year in patients with ATTR-CM and to evaluate the pathophysiological impacts of tafamidis on cardiac transthyretin amyloidosis

Outcome(s)

• Primary Outcome: The change on the area of amyloid deposition in the myocardial biopsy specimen from right ventricular septum before and 1 year after treatment with tafamidis

Secondary Outcome

(1) the way of amyloid deposition and interstitial fibrosis obtained by myocardial biopsy from ventricular septum
(2) cardiac function by echocardiography, cardiac MRI, and conductance catheter
(3) six minutes walk test, activity monitoring, peak oxygen uptake and quality of life
(4) heart failure severity
(5) blood tests (BNP, troponin T, etc)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(1)heart failue patients with transthyretin amyloidosis
(2)patients with transthyretin amyloidosis confirmed by myocardial biopsy
(3)patients with LV thickness equal and/or greater than 12 mm
(4)patients wth age equal and/or greater than 20
(5)men and women
(6)patients with NYHA 1 to 3
(7) patients with LV hypertrophy (and diastolic dysfunction) caused by amyloid deposition

Exclude criteria

(1) patients with severe valcular disease
(2) patients with NYHA4 heart failure
(3) patients with acute coronary syndrome
(4) patients with cardiogenic shock
(5) patients with severe liver failure
(6) patients with stage 5 kidney disease
(7) patients with infectious disease
(8) patients with a a life expectancy of less than 1 year
(9) pregnant patients
(10) patients who are unable to walk

Related Information

• Primary Sponsor: Fujimoto Naoki
• Source(s) of Monetary Support: Boehringer Ingelheim Seiyaku