Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis

NCT ID: NCT02016365

Last Updated: 2016-05-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 55 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-02-29
• Study Completion Date: 2014-12-31

Brief Summary

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.

Conditions

Transthyretin Amyloidosis; Cardiomyopathy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Doxycycline and UDCA

Doxycycline (200 mg/day intermittently) and UDCA (750 mg/day continuously)

Group Type: EXPERIMENTAL

Doxycycline

Intervention Type: DRUG

200 mg/day (100 mg twice daily, orally) for 4 weeks with a pause of 2 weeks in combination with UDCA

Ursodeoxycholic acid

Intervention Type: DRUG

750 mg/day (500 mg +250mg orally) continuously

Interventions

Doxycycline

200 mg/day (100 mg twice daily, orally) for 4 weeks with a pause of 2 weeks in combination with UDCA

Intervention Type: DRUG

Ursodeoxycholic acid

750 mg/day (500 mg +250mg orally) continuously

Intervention Type: DRUG

Other Intervention Names

Doxyferm; Ursofalk

Eligibility Criteria

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.

• Minimum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Umeå University

OTHER

Sponsor Role: lead

Responsible Party

Ole B Suhr, Professor, MD, PhD

Professor, MD, PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Ole B Suhr, MD PhD Prof

Role: PRINCIPAL_INVESTIGATOR

Dept of Clinical Medicine and public Health, Umeå University

Locations

Dept of Clinical Medicin, Ptieå Hospital

Piteå, , Sweden

Dept of clinical medicin, Skellefteå Hospital

Skellefteå, , Sweden

Dept of Clinical Medicine, Umeå University Hospital

Umeå, , Sweden

Countries

Sweden

Other Identifiers

EudraCT No:2011-005236-25

Identifier Type: -

Identifier Source: org_study_id