Treatment with doxycycline and tauroursodeoxycholic acid (Doxy/TUDCA) plus standard supportive therapy 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 - AmY-DoT

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-001621-41-IT

Enrollment

102

Registered

2020-11-04

Start date

2017-11-30

Completion date

Unknown

Last updated

2021-08-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cardiac amyloidosis caused by transthyretin MedDRA version: 20.0 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders

Interventions

Trade Name: BASSADO - 100 MG COMPRESSE 10 COMPRESSE Product Name: Doxiciclina Product Code: [NA] Pharmaceutical Form: Tablet INN or Proposed INN: DOXICICLINA CAS Number: 564-25-0 Current Sponsor code:

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Diagnosis of ATTRwt amyloidosis, or ATTRm p.Ile68Leu and p.Val122Ile; - 18 years or older; - cardiac involvement (mean left ventricular wall thickness >12 mm in the absence of other causes); - history of occurrence of at least 1 event of symptomatic heart failure; - stable diuretic dosage for at least 2 weeks before treatment initiation; - agree to practice effective methods of contraception where necessary - voluntary written consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 66

Exclusion criteria

Exclusion criteria: - Non-ATTR amyloidosis - pregnant or nursing women - uncontrolled bacterial, viral, fungal, HIV, HBV, or HCV infection; - presence of other active malignancy with - known allergy to tetracycline; - treatment with drugs potentially affecting doxycycline absorption - significant acute gastrointestinal symptoms; - active peptic ulceration and/or esophageal reflux disease

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy (superiority) of Doxy/TUDCA in patients with cardiac ATTR amyloidosis,;Secondary Objective: -To compare overall survival at 18 and 30 months of patients receiving Doxy/TUDCA in addition to standard supportive therapy with that of patients receiving standard supportive therapy alone. - To assess safety of Doxy/TUDCA by measuring the rate and grade of adverse events. - To assess changes in biochemical, echocardiographic, and clinical markers of disease severity during and after treatment. - To assess the impact of re-treatment with Doxy/TUDCA in patients who experience a major cardiac event after Doxy/TUDCA. - To assess discontinuation on biochemical, echocardiographic, and clinical markers of disease severity. - To assess the impact of treatment with Doxy/TUDCA in patients originally enrolled in the control arm who experience a “major cardiac event” and are then given Doxy/TUDCA on biochemical, echocardiographic, and clinical markers of disease severity.;Primary end point(s): Major cardiac events;Timepoint(s) of evaluation of this end point: 18 months	—

Measure

Time frame

Main Objective: To evaluate the efficacy (superiority) of Doxy/TUDCA in patients with cardiac ATTR amyloidosis,;Secondary Objective: -To compare overall survival at 18 and 30 months of patients receiving Doxy/TUDCA in addition to standard supportive therapy with that of patients receiving standard supportive therapy alone. - To assess safety of Doxy/TUDCA by measuring the rate and grade of adverse events. - To assess changes in biochemical, echocardiographic, and clinical markers of disease severity during and after treatment. - To assess the impact of re-treatment with Doxy/TUDCA in patients who experience a major cardiac event after Doxy/TUDCA. - To assess discontinuation on biochemical, echocardiographic, and clinical markers of disease severity. - To assess the impact of treatment with Doxy/TUDCA in patients originally enrolled in the control arm who experience a “major cardiac event” and are then given Doxy/TUDCA on biochemical, echocardiographic, and clinical markers of disease severity.;Primary end point(s): Major cardiac events;Timepoint(s) of evaluation of this end point: 18 months

—

Secondary

Measure	Time frame
Secondary end point(s): Overall survival; Adverse events equal or higher grade 3 (CTCAE v 5.0); Changes in BNP, NT-proBNP, cTnI, mLVW, EF, MCF, GLS, 6MWT;Timepoint(s) of evaluation of this end point: 18 and 30 months; At each visit; After 6, 12, 18 months during treatment and at 6, 12 months after the end of treatment	—

Countries

Italy

Contacts

Public ContactCentro per lo studio e la cura dell

Fondazione IRCCS Policlinico San Matteo

segreteria.amiloidosi@smatteo.pv.it0382502994

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026