Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis
Status:
Completed
Trial end date:
2015-10-01
Target enrollment:
Participant gender:
Summary
This study is being conducted to explore the potential benefits of a twelve-month doxycycline
(at the best tolerated dose of 200 mg/day) and tauroursodeoxycholic acid (750 mg/day)
treatment on disease progression in patients affected by transthyretin amyloidosis,
including: 1) patients not eligible for liver transplantation; 2) patients eligible for liver
transplantation, as a "bridge" therapy between the time of diagnosis and surgery, with the
aim of stabilizing the disease; 3) patients showing disease progression after liver
transplantation performed since at least 1 year.
It is a phase II, therapeutic exploratory, two-part, 18-month, single centre, prospective
study.
Part I is a 12-month, open label treatment period in which doxycycline (200 mg/day,
continuously) and tauroursodeoxycholic acid (750 mg/day continuously) are administered to 40
consenting subjects with transthyretin amyloidosis. Part II is a withdrawal period in which
subjects will be monitored for disease progression. During part I, subjects will be evaluated
at baseline (study Day 0), and then after 3, 6, 9 and 12 months of doxycycline plus
tauroursodeoxycholic acid treatment or at premature treatment discontinuation; during part
II, they will be assessed at months 15 and 18. Monthly phone contacts and blood tests will be
performed to monitor potential adverse events.