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Safety and Effect of Doxycycline in Patients With Amyloidosis

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2015 by Boston University.
Recruitment status was:  Active, not recruiting
Information provided by (Responsible Party):
John L. Berk, Boston University Identifier:
First received: August 21, 2012
Last updated: January 2, 2015
Last verified: January 2015

August 21, 2012
January 2, 2015
July 2012
June 2015   (Final data collection date for primary outcome measure)
Composite measures specific to the organ system affected by amyloidosis at study entry [ Time Frame: 12 months ]

Amyloid nephropathy: 24 hour urine protein excretion, creatinine clearance

Amyloid cardiomyopathy: cardiac biomarkers (BNP, Troponin I), echo parameters (IVSd, longitudinal strain, diastolic indices [e/e']), ECG

Amyloid peripheral neuropathy: Neurologic Impairment Score-Lower Limb (NIS-LL), modified body mass index (mBMI)

Amyloid autonomic neuropathy: postural blood pressures, heart rate variability, mBMI

Localized amyloidosis:

  1. airway -- PFTs, CT imaging, endoscopic visualization
  2. gastrointestinal -- endoscopic visualization
  3. bladder -- CT imaging, cystoscopy, urodynamics
  4. skin -- direct measures of disease
Same as current
Complete list of historical versions of study NCT01677286 on Archive Site
  • Quality of Life [ Time Frame: 12 months ]
    Quality of Life (SF-36)
  • Kumamoto neurologic score [ Time Frame: 12 months ]
    Motor, sensory, autonomic measures of neuropathy
Same as current
Not Provided
Not Provided
Safety and Effect of Doxycycline in Patients With Amyloidosis
A Phase II Study 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.

Not Provided
Phase 2
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Drug: Doxycycline 100 mg po bid x 12 months
100mg by mouth twice daily for 1 year.
Other Name: CAS: 564-25-0; ATC code: J01AA02 A01AB22; PubChem: CID 11256
Experimental: 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.
Intervention: Drug: Doxycycline 100 mg po bid x 12 months

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
December 2015
June 2015   (Final data collection date for primary outcome measure)

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%
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Not Provided
John L. Berk, Boston University
Boston University
Not Provided
Principal Investigator: John L Berk, M.D. Boston University
Boston University
January 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP