Overview

A Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy Versus Standard Supportive Therapy Alone in Cardiac Amyloidosis Caused by Transthyretin

Status:
Active, not recruiting

Trial end date:
2021-04-01

Target enrollment:

Participant gender:

Summary

Cardiac amyloidosis caused by transthyretin either mutated (in ATTRm amyloidosis) or wildtype (in ATTRwt, formerly senile, amyloidosis) is a rare disease but is diagnosed with increasing frequency thanks to the availability of non-invasive scintigraphy-based means. Cardiac ATTR amyloidosis is characterized by progressive heart failure with a median survival of less than 4 years, and there is no standard treatment for this disease. It was proved that the marketed antibiotic doxycycline (Doxy) disrupts amyloid fibrils in vitro and in animal models synergistically with tauroursodeoxycholic acid (TUDCA). Based on these pre-clinical data, a clinical trial of Doxy/TUDCA in ATTR Amyloidosis (NCT01171859) was conducted. Treatment was well tolerated and was able to prevent progression of cardiac dysfunction.

Phase:

Phase 3

Details

Lead Sponsor:

IRCCS Policlinico S. Matteo

Treatments:

Doxycycline
Taurochenodeoxycholic Acid
Tauroursodeoxycholic acid
Ursodoxicoltaurine