NCT03481972

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 102 (actual)
• Sponsor: Fondazione IRCCS Policlinico San Matteo di Pavia · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• TTR Cardiac Amyloidosis

Interventions

Type	Name	Description
DRUG	Doxycycline and tauroursodeoxycholic acid	doxycicline and tauroursodeoxycholic acid
DRUG	Standard of care	Standard of care

Timeline

• Start date: 2018-04-11
• Primary completion: 2023-01-18
• Completion: 2023-06-22
• First posted: 2018-03-29
• Last updated: 2024-07-11

Locations

1 site across 1 country: Italy