Effects of glavonoid for transthyretin amyloidosis

Phase 2

• Conditions: Subjects with pathologically diagnosed transthyretin amyloidosis excluded from insurance cover; transthyretin amyloidosis

• Registration Number: JPRN-jRCTs071230091
• Lead Sponsor: Tahara Nobuhiro

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-11-20
• Last Update Posted: 22 January 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 40

Inclusion 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).

Exclusion 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

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