Status:
COMPLETED
Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis
Lead Sponsor:
Fondazione IRCCS Policlinico San Matteo di Pavia
Conditions:
Transthyretin Amyloidosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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...
Eligibility Criteria
Inclusion
- Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens;
- Molecular definition of the transthyretin (TTR) mutation or immunohistochemical staining of amyloid fibrils with anti-TTR antibody;
- ECOG performance status (PS) 0, 1, 2;
- New York Heart Association (NYHA) class ≤III
- Systolic blood pressure ≥100 mmHg (standing)
- Must have symptomatic organ involvement with amyloid to justify therapy; must have evidence of neuropathy and/or cardiomyopathy progression after liver transplantation performed since at least one year.
- Contraception for women of childbearing potential. Medically approved contraception could include abstinence. A negative serum pregnancy test is required prior to initiation of treatment with study medication.
Exclusion
- Liver transplantation in the previous 12 months or liver transplantation anticipated in less than 6 months;
- ALT and/or AST ≥ 2 x Upper Normal Limit (UNL);
- Alkaline phosphatase ≥ 2 x UNL;
- Creatinine clearance \< 30 ml/min;
- Any other lab values that in the opinion of the investigator might place the subject at unacceptable risk for participation in the study;
- Echocardiographic ejection fraction \< 50%;
- Other neuropathies, due to vitamin B12 deficiency, alcoholism, hypothyroidism, uremia, diabetes mellitus, vasculitides;
- History of poor compliance;
- History of hypersensitivity to any of the ingredients of the study therapies;
- Use of any investigational drug, device (or biologic) within 4 weeks prior to study entry or during the study.
Key Trial Info
Start Date :
July 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2015
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT01171859
Start Date
July 1 2010
End Date
October 1 2015
Last Update
February 25 2016
Active Locations (1)
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1
Amyloid Research and Treatment Centre, Biotechnology Research Laboratories
Pavia, Pavia, Italy, 27100