Behçet Disease Affecting Oral MucosaPhase 2NCT03888846
Topical Pentoxifylline Gel on Behcet's Disease Oral Ulcers
A Phase 2 study testing Pentoxifylline and Colchicine in people with Behçet Disease Affecting Oral Mucosa, run by Ipekyolu Ilac Ltd. Sti at 1 site in Turkey (Türkiye).
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Behçet Disease Affecting Oral Mucosa
- What is being tested?
- Pentoxifylline and Colchicine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 25 Mar 2019to 30 Sep 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Meets the International Study Group criteria for Behcet's Disease
- Presents with at least one active lesions accessible to measurement, the largest of which is to be designated as the index ulcer
- Index oral ulcer to be assessed should be in the easily accessible areas of the oral mucosa, and the oral ulcer first began within 48 hrs prior to enrollment
- Adult (>18 years) male or a non-pregnant, non-lactating female
- Has signed an Ethics Committee (EC) approved subject consent form
- Has completed all screening procedures satisfactorily, is deemed to be an acceptable subject and is otherwise eligible for entry into the study
- Is willing and able to comply with the protocol
- Is being treated with colchicine
Who cannot join
- Has a severe, acute, or chronic systemic disease other than Behcet's Disease such as congestive heart failure, hepatic failure, renal failure, systemic lupus erythematosus, Stevens-Johnson syndrome, ulcerative colitis, cancer, leukemia, diabetes, AIDS, or any other condition that is severely compromising the immune system
- Has received pentoxifylline in any form over the previous 60 days prior to enrollment
- Has experienced recent cerebral and/or retinal hemorrhage or in patients who have previously exhibited intolerance to pentoxifylline or methylxanthines such as caffeine, theophylline, and theobromine
- Has concomitant administration of strong CYP1A2 inhibitors (including e.g. ciprofloxacin or fluvoxamine) and other drugs that may increase the exposure to pentoxifylline
- Is receiving immune suppressing or modulating therapy (e.g., apremilast) or topical corticosteroids within 2 weeks prior to enrollment
- Is not being treated with colchicine
- Is under active treatment for dental conditions, such that multiple dental office visits would be required during the study period, or presents with oral conditions which are not thought to be related to Behcet's Disease and in the judgment of a qualified dentist, will require treatment during the study period
- Is suffering from any medical condition other than Behcet's Disease known to cause oral ulcerations, such as erosive lichen planus, benign mucous membrane pemphigoid, Systemic Lupus Erythematous, Crohn's disease, Reiter's syndrome, or AIDS. Has an eating disorder and/or psychiatric illness requiring treatment. Has a history of, or is currently exhibiting, any disease or condition, which, in the opinion of the principal investigator, would place the subject at increased risk during study therapy. Has any abnormality in hematological or biochemical variable, which, in the opinion of the principal investigator, would place the subject at increased risk during study therapy
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Istanbul Universitesi-Cerrahpaşa Tıp Fakültesi, İç Hastalıkları Anabilim Dalı-Romatoloji
Istanbul, 34098
About this study
Official title: Clinical Protocol for Administration of Topical Pentoxifylline Gel on Behcet's Disease Oral Ulcers
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.
Read the detailed description
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.
- Conditions
- Behçet Disease Affecting Oral Mucosa
- Being tested
- Pentoxifylline, Colchicine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PentoxifyllineDrug
Also called Trental
Topical gel version of pentoxifylline to be administered intraorally.
ColchicineDrug
Also called Colchicum-Dispert
Colchicine therapy is provided to patients with oral ulcers as part of supportive care routinely offered by Behcet's Clinic-Istanbul.
| Study group | Role | What they receive |
|---|---|---|
| Colchicine | Other | Colchicine therapy as part of supportive care routinely offered in Behcet's Clinic-Istanbul Receives: Drug: Colchicine |
| Topical Pentoxifylline Gel and Colchicine | Experimental | Topical Pentoxifylline Gel administration in addition to the colchicine therapy as part of supportive care routinely offered in Behcet's Clinic-Istanbul Receives: Drug: Pentoxifylline; Drug: Colchicine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Secondary outcomes (2)
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.
Measured: 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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2019
- Study start25 Mar 2019
- Primary completion31 Aug 2019
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Behçet Disease Affecting Oral MucosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipekyolu Ilac Ltd. Sti
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Istanbul University - Cerrahpasa
- Responsible party
- Sponsor
- Investigators
- Gulen Hatemi, MD, Principal investigator, Istanbul Universitesi-Cerrahpasa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03888846
- Sponsor's protocol ID
- P104375V04
- Status last verified
- Aug 2023
- First submitted
- 21 Mar 2019
- Last updated
- 3 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after deidentification will be available to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Protocol for Administration of Topical Pentoxifylline Gel on Behcet's Disease Oral Ulcers
- ClinicalTrials.gov ID
- NCT03888846
- Acronym / sponsor ID
- P104375V04
- Conditions
- Behçet Disease Affecting Oral Mucosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Ipekyolu Ilac Ltd. StiIndustry (pharma, biotech, devices)
- Collaborators
- Istanbul University - Cerrahpasa
- Study start
- 25 Mar 2019 (actual)
- Primary completion
- 31 Aug 2019 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 25 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Colchicine | Other | Drug: ColchicineColchicine therapy as part of supportive care routinely offered in Behcet's Clinic-Istanbul |
| Topical Pentoxifylline Gel and Colchicine | Experimental | Drug: Pentoxifylline, Drug: ColchicineTopical Pentoxifylline Gel administration in addition to the colchicine therapy as part of supportive care routinely offered in Behcet's Clinic-Istanbul |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Meets the International Study Group criteria for Behcet's Disease
- Presents with at least one active lesions accessible to measurement, the largest of which is to be designated as the index ulcer
- Index oral ulcer to be assessed should be in the easily accessible areas of the oral mucosa, and the oral ulcer first began within 48 hrs prior to enrollment
- Adult (>18 years) male or a non-pregnant, non-lactating female
- Has signed an Ethics Committee (EC) approved subject consent form
Key exclusion criteria (8 listed)
- Has a severe, acute, or chronic systemic disease other than Behcet's Disease such as congestive heart failure, hepatic failure, renal failure, systemic lupus erythematosus, Stevens-Johnson syndrome, ulcerative colitis, cancer, leukemia, diabetes, AIDS, or any other condition that is severely compromising the immune system
- Has received pentoxifylline in any form over the previous 60 days prior to enrollment
- Has experienced recent cerebral and/or retinal hemorrhage or in patients who have previously exhibited intolerance to pentoxifylline or methylxanthines such as caffeine, theophylline, and theobromine
- Has concomitant administration of strong CYP1A2 inhibitors (including e.g. ciprofloxacin or fluvoxamine) and other drugs that may increase the exposure to pentoxifylline
- Is receiving immune suppressing or modulating therapy (e.g., apremilast) or topical corticosteroids within 2 weeks prior to enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PentoxifyllineAlso: TrentalTopical gel version of pentoxifylline to be administered intraorally. | Drug | Topical Pentoxifylline Gel and Colchicine |
| ColchicineAlso: Colchicum-DispertColchicine therapy is provided to patients with oral ulcers as part of supportive care routinely offered by Behcet's Clinic-Istanbul. | Drug | Colchicine, Topical Pentoxifylline Gel and Colchicine |
Primary outcome
Mean change in the speed of healing of the index oral ulcer
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).
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.
Mean change in the total number of oral ulcers
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.
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.
Mean change in patient reported outcome pain scores
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.
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.
2 secondary outcomes
Behcet's Disease-Quality of Life Assessment Questionnaire
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
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after deidentification will be available to other researchers.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gulen Hatemi, MD principal investigator
Istanbul Universitesi-Cerrahpasa
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pentoxifylline and Colchicine in people with Behçet Disease Affecting Oral Mucosa, run by Ipekyolu Ilac Ltd. Sti at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change in the speed of healing of the index oral ulcer (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).). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2023.