Overview

This trial is active, not recruiting.

Condition amyloidosis
Treatment doxycycline 100 mg po bid x 12 months
Phase phase 2
Sponsor Boston University
Start date July 2012
End date June 2015
Trial size 60 participants
Trial identifier NCT01677286, H-31546

Summary

The tetracycline antibiotic doxycycline disrupts A beta amyloid fibrils (AB) in Alzheimer's disease, transthyretin (ATTR) amyloid fibrils in familial amyloidotic polyneuropathy, and immunoglobulin light chain (AL) amyloid fibrils in transgenic mouse models of disease. If untreated, amyloid deposits impair organ function, affecting the morbidity and mortality of patients.

This single-center, twelve-month, open-label, prospective, pilot phase II study aims to determine whether doxycycline reduces amyloid deposits and improves organ function in patients with systemic or localized amyloidosis.

The investigators plan to enroll patients with measurable amyloid disease according to internationally-accepted diagnostic criteria. Patients must have stable organ function at enrollment. Eligible subjects not receiving active treatments for amyloidosis affecting their kidneys, heart, aerodigestive tracts, peripheral or autonomic nervous system(s), lungs, eyes, skin, bladder, or breasts will undergo evaluations at baseline, 6 months, and 12 months - or more frequently as clinically indicated.

Over 45 years experience indicates doxycycline is a safe, well tolerated antibiotic. The investigators will use standard grading systems to assess doxycycline response following twelve months of treatment.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Open-label doxycycline 100 mg twice daily by mouth will be administered to subjects for 12 months.
doxycycline 100 mg po bid x 12 months CAS: 564-25-0; ATC code: J01AA02 A01AB22; PubChem: CID 11256
100mg by mouth twice daily for 1 year.

Primary Outcomes

Measure
Composite measures specific to the organ system affected by amyloidosis at study entry
time frame: 12 months

Secondary Outcomes

Measure
Quality of Life
time frame: 12 months
Kumamoto neurologic score
time frame: 12 months

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Age 18 or older - Biopsy-proven amyloidosis - Biochemical or clinical evidence of amyloid induced end-organ dysfunction Exclusion Criteria: - Concurrent use of other tetracyclines - Ongoing active treatment for amyloidosis - Pregnancy or unwillingness to use contraception by women of childbearing age - Doxycycline drug allergy/hypersensitivity - ECOG performance status > 3 - NYHA class > 3 - Renal insufficiency (estimated creatinine clearance < 25 ml/min) - Transaminitis (AST or ALT > 5 times upper limit of normal) - Diabetes mellitus or hemoglobin A1C > 6.2%

Additional Information

Official title A Phase II Study of Doxycycline in Patients With Amyloidosis
Principal investigator John L Berk, M.D.
Trial information was received from ClinicalTrials.gov and was last updated in January 2015.
Information provided to ClinicalTrials.gov by Boston University.