Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis (Dox/Urso)

Umeå University

Status and phase

Completed

Phase 2

Conditions

Transthyretin Amyloidosis

Cardiomyopathy

Treatments

Drug: Doxycycline

Drug: Ursodeoxycholic acid

Study type

Interventional

Funder types

Other

Identifiers

NCT02016365

EudraCT No:2011-005236-25

Details and patient eligibility

About

The primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.

Enrollment

55 patients

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Cardiomyopathy with septal thickness > 15 mm and/or S-NT-ProBNP > 300 ng/
Age >50 years
Male and females after menopause. Menopause is defined as 6 to 12 months of amenorrhea in a woman over 45 years of age.
Written informed consent to be obtained prior to any study procedure
Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens, and typing of amyloid deposits as TTR and identification of amyloid fibril type.
Molecular definition of the TTR mutation or immunohistochemical staining of amyloid fibrils with anti TTR antibody
New York Heart Association (NYHA) class <III
Systolic blood pressure >100 mmHg (standing)
Must have symptomatic organ involvement with amyloid to justify therapy

Exclusion criteria

Liver transplantation in the previous 6 months or liver transplantation anticipated in less than 6 months;
ALT and/or AST > 2 x upper normal limit (UNL);
Creatinine clearance < 30 ml/min (Cockcroft -Gault Formula)
Any other lab values, illness or condition that in the opinion of the investigator might place the subject at unacceptable risk for participation in the study;
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.