Although Behçet's Disease (BD) has no FDA-approved therapies, numerous clinical reports suggest that oral ulcers of BD may resolve when treated with systemic ingested pentoxifylline (PTX). The investigators here propose to investigate the therapeutic potential of PTX dissolved in muco-adherent formulation and directly applied to the oral lesions. This 60 patient proof of concept trial is designed to meet regulatory requirements for safety concerns while at the same time exploring the potential efficacy and clinical utility of this product. The investigators hypothesize that application of topical PTX will accelerate the healing of these lesions in a clinically meaningful way, and further hypothesize that topical PTX can become a valuable adjunct to any other systemic therapy for BD.
Behçet's Clinic at Cerrahpaşa Medical Faculty at the University of Istanbul has a long history of supportive care for BD patients and is recognized as the world leader in this regard. According to the EULAR 2018 recommended guidelines of Behcet's Disease, the first line of therapy recommended against recurring oral ulcers is colchicine. However, a significant portion of the participants undergoing colchicine therapy does not have sufficient response and require subsequent immune modulatory therapy. By comparing participants receiving colchicine as part of supportive care to those who also receive topical pentoxifylline (PTX), the investigators seek to prove the concept that topical PTX may have clinical value. Additionally, the investigators seek to demonstrate the safety and practicality of using topical PTX in BD patients. If the PTX gel therapy provides a sufficient response in participants, these participants might not need to undergo immune modulatory therapies. The risks of PTX are well characterized and previously reported adverse events were primarily gastrointestinal. The investigators expect fewer GI symptoms to result from the topical PTX application. The investigators anticipate the benefits of PTX can be optimized by a topical application, but require a trial to test that hypothesis.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
41
Topical gel version of pentoxifylline to be administered intraorally.
Colchicine therapy is provided to patients with oral ulcers as part of supportive care routinely offered by Behcet's Clinic-Istanbul.
Istanbul Universitesi-Cerrahpaşa Tıp Fakültesi, İç Hastalıkları Anabilim Dalı-Romatoloji
Istanbul, Turkey (Türkiye)
Mean change in the speed of healing of the index oral ulcer
The size of the index oral ulcer will be measured at study enrollment and during the two week trial at seven time points (Please see Schedule of Activities in the protocol for more detail). The change in the size of the index ulcer will be compared between two study arms.
Time frame: The participants will be assessed at seven time points starting at enrollment when the index ulcer is assigned until the complete healing(disappearance) of the index oral ulcer. This time frame is estimated to take two weeks (please see SoA in protocol).
Mean change in the total number of oral ulcers
The total number of oral ulcer will be measured at study enrollment and during the two week trial at seven time points (Please see Schedule of Activities in the protocol for more detail). The total number of oral ulcers will be compared between two study arms.
Time frame: The participants will be assessed at seven time points starting at the enrollment visit until the end of study visit. This time frame is estimated to take two weeks.
Mean change in patient reported outcome pain scores
Pain scores will be compared between two study arms as measuring pain offers an opportunity to correlate simple measurements of oral mucosal disruption with patient's fundamental disease experience.
Time frame: The participants will be assessed at sixteen time points starting at the enrollment visit until the final follow up visit.This time frame is estimated to take 45 days. Please see Schedule of Activities(SoA) in the protocol.
Behcet's Disease-Quality of Life Assessment Questionnaire
Participant's quality of life will be assessed by a Behcet's Disease specific questionnaire prepared by key opinion leaders in the field of Behcet's Disease research at the University of Leeds. Please see the "Behcet's Disease Quality of Life Measure" in the appendix of the protocol for more details.
Time frame: The participants will be assessed at eight time points starting at the enrollment visit until the final follow up visit.This time frame is estimated to take 45 days. Please see Schedule of Activities(SoA) in the protocol.
Behcet's Disease Activity Index
Behcet's Disease Activity Index is prepared by International Society for Behcet's Disease to guide the doctor in assessing the disease activity.
Time frame: The participants will be assessed at eight time points starting at the enrollment visit until the final follow up visit.This time frame is estimated to take 45 days. Please see Schedule of Activities(SoA) in the protocol.
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