Safety and Effect of Doxycycline in Patients With Amyloidosis
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ClinicalTrials.gov Identifier: NCT01677286 |
Recruitment Status :
Completed
First Posted : September 3, 2012
Results First Posted : June 6, 2017
Last Update Posted : August 1, 2017
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Tracking Information | |||||||
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First Submitted Date ICMJE | August 21, 2012 | ||||||
First Posted Date ICMJE | September 3, 2012 | ||||||
Results First Submitted Date ICMJE | March 19, 2017 | ||||||
Results First Posted Date ICMJE | June 6, 2017 | ||||||
Last Update Posted Date | August 1, 2017 | ||||||
Actual Study Start Date ICMJE | July 2012 | ||||||
Actual Primary Completion Date | May 22, 2015 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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:
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Change History | |||||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||||
Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Safety and Effect of Doxycycline in Patients With Amyloidosis | ||||||
Official Title ICMJE | A Phase II Study of Doxycycline in Patients With Amyloidosis | ||||||
Brief 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. |
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Detailed Description | 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. |
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Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Amyloidosis | ||||||
Intervention ICMJE | 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
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Study Arms ICMJE | 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
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE |
25 | ||||||
Original Estimated Enrollment ICMJE |
60 | ||||||
Actual Study Completion Date ICMJE | December 15, 2015 | ||||||
Actual Primary Completion Date | May 22, 2015 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT01677286 | ||||||
Other Study ID Numbers ICMJE | H-31546 | ||||||
Has Data Monitoring Committee | No | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | John L. Berk, Boston University | ||||||
Original Responsible Party | John L. Berk, Boston University, Clinical Director, Amyloid Treatment & Research Program | ||||||
Current Study Sponsor ICMJE | Boston University | ||||||
Original Study Sponsor ICMJE | Same as current | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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PRS Account | Boston University | ||||||
Verification Date | July 2017 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |