Effects of glavonoid for transthyretin amyloidosis

JRCT ID: jRCTs071230091

Registered date:20/11/2023

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Subjects with pathologically diagnosed transthyretin amyloidosis excluded from insurance cover
• Date of first enrollment: 20/11/2023
• Target sample size: 40
• Study type: Interventional

Intervention(s)

Allocation
Subject group: Gravonoid group
Control subject group: Placebo group

Outcome(s)

Primary Outcome

Clinical endpoints for efficacy
1) Physical examination
2) Nutrition
3) Liver function
4) Glycemic state
5) Lipid profile
6) Renal function
7) Myocardial damage marker
8) Neuropathy marker
9) Aging marker
10) Amyloidosis score (0-100 points)
11) Polyneuropathy Disability score
12) Carpal tunnel syndrome score (0-30 points)
13) Cardiac evaluation

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Subjects with pathologically confirmed deposition of transthyretin amyloid protein.
2) Age of 18 years or older at screening and written informed consent to participate in this study.
3) Subjects who do not meet the following indication criteria for tafamidis (*).
* Indication criteria for tafamidis.
1. Wild-type transthyretin-derived cardiac amyloidosis
a. Medical history of hospitalization for heart failure or a heart failure condition requiring treatment with a diuretic for improvement.
b. Presence of amyloid deposits in biopsy tissue (cardiac or non-cardiac).
c. Transthyretin precursor protein identification by immunohistochemistry.
d. Evidence of cardiac involvement on echocardiography with an end-diastolic interventricular septal wall thickness >12mm.

2. Variant transthyretin-derived cardiac amyloidosis
a. Cardiomyopathy symptoms and a transthyretin gene mutation associated with cardiomyopathy.
b. Medical history of hospitalization for heart failure or a heart failure condition requiring treatment with a diuretic for improvement.
c. Evidence of cardiac involvement on echocardiography with an end-diastolic interventricular septal wall thickness >12mm.
d. Presence of amyloid deposits in biopsy tissue (cardiac or non-cardiac).

Exclude criteria

1) Subjects with a history of hypersensitivity to glavonoid.
2) Subjects who have been taking tafamidis, diflunisal, or glavonoid 8 weeks prior to the initiation of screening.
3) Subjects receiving treatment with patisiran or vutrisiran.
4) Subjects with confirmed transthyretin gene abnormality and polyneuropathy.
5) Pregnant subjects.
6) Subjects with significant hepatic disorders (ALT or AST levels more than 3 times higher than normal values) or liver cirrhosis.
7) Subjects judged to be inappropriate to participate in this study by investigators.

Related Information

• Primary Sponsor: Tahara Nobuhiro
• Source(s) of Monetary Support: Department of Amyloidosis Research, Nagasaki International University