Safety and Effect of Doxycycline in Patients With Amyloidosis

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.