Safety and Effect of Doxycycline in Patients With Amyloidosis

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-07-31

Study Completion Date

2015-12-15

Brief Summary

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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.

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Detailed Description

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In transgenic animal models of disease, 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.

The aim of this single-center, 12-month open label, prospective phase II study was to determine a) the safety and tolerability of prolonged full dose doxycycline in patients with amyloidosis, and b) the effect of doxycycline treatment on amyloid-induced organ dysfunction.

We enrolled 25 patients with measurable organ dysfunction caused by amyloid deposition who were not receiving active treatment to control their amyloid production. All 25 subjects received doxycycline 100 mg by both twice daily for up to 12 months depending on their tolerance of the antibiotic. The primary endpoint, defined by the organ most affected by amyloid, was measured at baseline, 6 and 12 months along with safety laboratory values.

Conditions

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Amyloidosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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doxycycline 100 mg po bid x 12 months

Open-label doxycycline 100 mg twice daily by mouth will be administered to subjects for 12 months.

Group Type EXPERIMENTAL

Doxycycline 100 mg po bid x 12 months

Intervention Type DRUG

100mg by mouth twice daily for 1 year.

Interventions

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Doxycycline 100 mg po bid x 12 months

100mg by mouth twice daily for 1 year.

Intervention Type DRUG

Other Intervention Names

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CAS: 564-25-0; ATC code: J01AA02 A01AB22; PubChem: CID 11256

Eligibility Criteria

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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%

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No