Behcet SyndromePhase 2NCT04186559
Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers
A Phase 2 study of Behcet Syndrome and Behcet Disease, run by Silk Road Therapies, Inc. at 1 site in United States.
Trial summary
Behçet's Disease (BD) is a complex, chronic, relapsing, multi-system inflammatory disorder that is characterized by oral ulcers, genital ulcers in addition to ocular and skin symptoms. Genital ulcers are the second most common symptom of BD and occur in 57-93% of BD patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Behcet Syndrome, Behcet Disease
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2024to Dec 2024
- 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 (BD)
- Presents with at least one active genital lesion accessible to measurement
- Adult (>18 years) male or a non-pregnant, non-lactating female
- Has signed an IRB 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
- Subject's treating physician confirm that the subject has previously had a genital ulcer due to Behcet's Disease
Who cannot join
- Has a severe, acute, or chronic systemic disease other than BD 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 for which they are immune-compromised
- Has received PTX in any form over the previous 5 days prior to enrollment
- 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 experienced recent cerebral and/or retinal hemorrhage or in patients who have previously exhibited intolerance to this product or methylxanthines such as caffeine, theophylline, and theobromine
- Is receiving apremilast or anti-TNF agents such as; Rituximab, Infliximab, Adalimumab, Etanercept (azathioprine, cyclosporine, colchicine, mycophenolate mofetil, interferon and topical or systemic steroid use does not disqualify from enrollment- however, topical steroids should not be used during the treatment period)
- Has concomitant administration of strong CYP1A2 inhibitors (including e.g. ciprofloxacin or fluvoxamine) and other drugs that may increase the exposure to PTX
Trial locations and contacts
1 site in 1 country.
United States1 site
Silk Road Therapies
Washington D.C., District of Columbia, 20008
Timothy Cote, MD, 202-738-6336, timcote1960@gmail.com
Andrew J Sulich, MD
About this study
Official title: Clinical Protocol to Evaluate Use of Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers
Behçet's Disease (BD) is a complex, chronic, relapsing, multi-system inflammatory disorder that is characterized by oral ulcers, genital ulcers in addition to ocular and skin symptoms. Genital ulcers are the second most common symptom of BD and occur in 57-93% of BD patients. The genital ulcers of BD clearly cause considerable morbidity for those affected. This will be one of the first trials of a topical product designed for accelerated healing of BD genital ulcers. There is no cure or FDA-approved therapies for BD genital ulcers and the treatment of recurrent genital ulcers that are associated with BD is palliative.
Recent study evaluating the effect of topical PTX on BD oral ulcers suggests that topical pentoxifylline (PTX) might have an immediate impact on BD oral ulcer healing leading to accelerated ulcer clearance, which results in lower daily ulcer numbers for the patients along with reduced pain scores. Investigators hypothesize that application of topical PTX will accelerate the healing of BD genital ulcers in a similar 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
In contrast to the recent study on BD oral ulcers, in which very carefully selected patients were enrolled (i.e. no immunosuppressive therapies and a new lesion of no more than 48hrs), this trial will be more "Real World" and enroll all patients with genital ulcers of BD, allowing randomization to eliminate biases between treatment and control groups. The advantages of this more open approach are twofold. First, this approach more directly measures the benefit of PTX gel therapy on the population that is more likely to use the therapy if it becomes approved. Secondly, those broader criteria will allow faster enrollment for a disease manifestation that occurs considerably less frequently than oral ulcers.
This randomized proof of concept trial investigating the effect of topical PTX on BD genital ulcers will be double-blind, placebo controlled with a crossover component, and it will follow the patients during the treatment period of 30 days or until the healing of genital ulcers. Specifically, patients who receive placebo as their initial course of treatment would receive active drug in a second course of treatment in which they would be re-enrolled upon recurrence of another crop of genital ulcers. Conversely, patients who receive active drug in their initial course of treatment would receive placebo drug once they are re-enrolled for the second course of treatment.
For all patients in either group, investigators will collect photographs of the external genital ulcer area, the size and location of the genital ulcers, number of ulcers, pain scores, and concomitant medications. This data will be collected for the following month or until the healing of genital ulcers. Additionally, adverse event experiences will be solicited. Data analyses will be exploratory comparisons of differences in safety and efficacy endpoints between BD patients who did and did not receive topical PTX.
- Conditions
- Behcet Syndrome, Behcet Disease
- Being tested
- Topical Pentoxifylline Gel (Vehicle +PTX), Topical Placebo Gel (Vehicle)
- Medical subject headings
- Behcet Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Topical Pentoxifylline Gel (Vehicle +PTX)Drug
Also called Trental
Patients will be given 1 tube containing 20 ml of topical PTX gel (containing 1,000mg of PTX) to be applied no fewer than four times per day with complete consumption of the entire tube each day.
Topical Placebo Gel (Vehicle)Drug
Patients will be given 1 tube containing 20 ml of topical placebo gel to be applied no fewer than four times per day with complete consumption of the entire tube each day.
| Study group | Role | What they receive |
|---|---|---|
| Topical pentoxifylline (PTX) gel | Experimental | As part of a randomized, placebo-controlled trial with a crossover component, patients who receive topical PTX gel in their initial course of treatment will receive topical placebo gel once they are re-enrolled for their second course of treatment upon recurrence of another crop of genital ulcers. Receives: Drug: Topical Pentoxifylline Gel (Vehicle +PTX); Drug: Topical Placebo Gel (Vehicle) |
| Topical placebo gel | Placebo comparator | As part of a randomized, placebo-controlled trial with a crossover component, patients who receive topical placebo gel in their initial course of treatment will receive topical PTX gel once they are re-enrolled for their second course of treatment upon recurrence of another crop of genital ulcers. Receives: Drug: Topical Pentoxifylline Gel (Vehicle +PTX); Drug: Topical Placebo Gel (Vehicle) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean time to healing of genital ulcers
The size of the genital ulcers will be measured at study enrollment and during the 30-day treatment period or until the healing of the genital ulcer(s). The change in the size of the genital ulcers will be compared between two study arms. Genital ulcer size will be calculated by measuring the ulcer area via the image processing of ulcer photographs. Time to healing will be calculated as the number of days to reach genital ulcer area of zero. Shorter time to ulcer area of zero(complete healing of genital ulcers) would be a better outcome. Accordingly, longer time to genital ulcer area of zero would be a worse outcome.
Measured: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days.There will be a final follow-up 30 days after the treatment.
Mean change in total number of genital ulcers
Total number of genital ulcers will be measured at study enrollment and during the 30-day treatment period or until the healing of the genital ulcer(s). The change in the total number of genital ulcers will be compared between two study arms. Upon complete healing of genital ulcers, ulcer number of zero would be expected (better outcome). Accordingly, if genital ulcers are not healed, there would be greater total number of genital ulcers (worse outcome).
Measured: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days.There will be a final follow-up 30 days after the treatment.
Mean change in patient reported outcome pain scores in numeric rating scale
Pain scores will be compared between two study arms as measuring pain offers an opportunity to correlate simple measurements of genital ulcers with patient's fundamental disease experience. Pain scores will be evaluated by using the numeric rating scale with the range of 0 (no pain) to 10 (maximum pain).
Measured: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days. There will be a final follow-up 30 days after the treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2019
- Study startFeb 2024 (estimated)
- Primary completionNov 2024 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Behcet SyndromeStudies 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
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Silk Road Therapies, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andew J Sulich, MD, Principal investigator, Shores Rheumatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04186559
- Sponsor's protocol ID
- P145440V01A
- Status last verified
- Aug 2023
- First submitted
- 2 Dec 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: Study protocol, informed consent form, clinical study report, 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 to Evaluate Use of Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers
- ClinicalTrials.gov ID
- NCT04186559
- Acronym / sponsor ID
- P145440V01A
- Conditions
- Behcet Syndrome, Behcet Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Silk Road Therapies, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2024 (estimated)
- Primary completion
- Nov 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 4 Dec 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.
Behçet's Disease (BD) is a complex, chronic, relapsing, multi-system inflammatory disorder that is characterized by oral ulcers, genital ulcers in addition to ocular and skin symptoms. Genital ulcers are the second most common symptom of BD and occur in 57-93% of BD patients. The genital ulcers of BD clearly cause considerable morbidity for those affected. This will be one of the first trials of a topical product designed for accelerated healing of BD genital ulcers. There is no cure or FDA-approved therapies for BD genital ulcers and the treatment of recurrent genital ulcers that are associated with BD is palliative.
Recent study evaluating the effect of topical PTX on BD oral ulcers suggests that topical pentoxifylline (PTX) might have an immediate impact on BD oral ulcer healing leading to accelerated ulcer clearance, which results in lower daily ulcer numbers for the patients along with reduced pain scores. Investigators hypothesize that application of topical PTX will accelerate the healing of BD genital ulcers in a similar clinically meaningful way, and further hypothesize that topical PTX can become a valuable adjunct to any other systemic therapy for BD.
Detailed description
In contrast to the recent study on BD oral ulcers, in which very carefully selected patients were enrolled (i.e. no immunosuppressive therapies and a new lesion of no more than 48hrs), this trial will be more "Real World" and enroll all patients with genital ulcers of BD, allowing randomization to eliminate biases between treatment and control groups. The advantages of this more open approach are twofold. First, this approach more directly measures the benefit of PTX gel therapy on the population that is more likely to use the therapy if it becomes approved. Secondly, those broader criteria will allow faster enrollment for a disease manifestation that occurs considerably less frequently than oral ulcers.
This randomized proof of concept trial investigating the effect of topical PTX on BD genital ulcers will be double-blind, placebo controlled with a crossover component, and it will follow the patients during the treatment period of 30 days or until the healing of genital ulcers. Specifically, patients who receive placebo as their initial course of treatment would receive active drug in a second course of treatment in which they would be re-enrolled upon recurrence of another crop of genital ulcers. Conversely, patients who receive active drug in their initial course of treatment would receive placebo drug once they are re-enrolled for the second course of treatment.
For all patients in either group, investigators will collect photographs of the external genital ulcer area, the size and location of the genital ulcers, number of ulcers, pain scores, and concomitant medications. This data will be collected for the following month or until the healing of genital ulcers. Additionally, adverse event experiences will be solicited. Data analyses will be exploratory comparisons of differences in safety and efficacy endpoints between BD patients who did and did not receive topical PTX.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Topical pentoxifylline (PTX) gel | Experimental | Drug: Topical Pentoxifylline Gel (Vehicle +PTX), Drug: Topical Placebo Gel (Vehicle)As part of a randomized, placebo-controlled trial with a crossover component, patients who receive topical PTX gel in their initial course of treatment will receive topical placebo gel once they are re-enrolled for their second course of treatment upon recurrence of another crop of genital ulcers. |
| Topical placebo gel | Placebo comparator | Drug: Topical Pentoxifylline Gel (Vehicle +PTX), Drug: Topical Placebo Gel (Vehicle)As part of a randomized, placebo-controlled trial with a crossover component, patients who receive topical placebo gel in their initial course of treatment will receive topical PTX gel once they are re-enrolled for their second course of treatment upon recurrence of another crop of genital ulcers. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Meets the International Study Group criteria for Behcet's Disease (BD)
- Presents with at least one active genital lesion accessible to measurement
- Adult (>18 years) male or a non-pregnant, non-lactating female
- Has signed an IRB 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
Key exclusion criteria (6 listed)
- Has a severe, acute, or chronic systemic disease other than BD 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 for which they are immune-compromised
- Has received PTX in any form over the previous 5 days prior to enrollment
- 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 experienced recent cerebral and/or retinal hemorrhage or in patients who have previously exhibited intolerance to this product or methylxanthines such as caffeine, theophylline, and theobromine
- Is receiving apremilast or anti-TNF agents such as; Rituximab, Infliximab, Adalimumab, Etanercept (azathioprine, cyclosporine, colchicine, mycophenolate mofetil, interferon and topical or systemic steroid use does not disqualify from enrollment- however, topical steroids should not be used during the treatment period)
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 |
|---|---|---|
| Topical Pentoxifylline Gel (Vehicle +PTX)Also: TrentalPatients will be given 1 tube containing 20 ml of topical PTX gel (containing 1,000mg of PTX) to be applied no fewer than four times per day with complete consumption of the entire tube each day. | Drug | Topical pentoxifylline (PTX) gel, Topical placebo gel |
| Topical Placebo Gel (Vehicle)Patients will be given 1 tube containing 20 ml of topical placebo gel to be applied no fewer than four times per day with complete consumption of the entire tube each day. | Drug | Topical pentoxifylline (PTX) gel, Topical placebo gel |
Primary outcome
Mean time to healing of genital ulcers
Time frame: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days.There will be a final follow-up 30 days after the treatment.
The size of the genital ulcers will be measured at study enrollment and during the 30-day treatment period or until the healing of the genital ulcer(s). The change in the size of the genital ulcers will be compared between two study arms. Genital ulcer size will be calculated by measuring the ulcer area via the image processing of ulcer photographs. Time to healing will be calculated as the number of days to reach genital ulcer area of zero. Shorter time to ulcer area of zero(complete healing of genital ulcers) would be a better outcome. Accordingly, longer time to genital ulcer area of zero would be a worse outcome.
Mean change in total number of genital ulcers
Time frame: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days.There will be a final follow-up 30 days after the treatment.
Total number of genital ulcers will be measured at study enrollment and during the 30-day treatment period or until the healing of the genital ulcer(s). The change in the total number of genital ulcers will be compared between two study arms. Upon complete healing of genital ulcers, ulcer number of zero would be expected (better outcome). Accordingly, if genital ulcers are not healed, there would be greater total number of genital ulcers (worse outcome).
Mean change in patient reported outcome pain scores in numeric rating scale
Time frame: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days. There will be a final follow-up 30 days after the treatment.
Pain scores will be compared between two study arms as measuring pain offers an opportunity to correlate simple measurements of genital ulcers with patient's fundamental disease experience. Pain scores will be evaluated by using the numeric rating scale with the range of 0 (no pain) to 10 (maximum pain).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
- Individual participant dataSharing planned: Yes. Study protocol, informed consent form, clinical study report, individual participant data collected during the trial, after deidentification will be available to other researchers.
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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.
Andew J Sulich, MD principal investigator
Shores Rheumatology
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 of Behcet Syndrome and Behcet Disease, run by Silk Road Therapies, Inc. at 1 site in United States.
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 7 inclusion and 6 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: status not confirmed, 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 United States. 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Semsi B Derkunt, PHD (631-219-6223, bderkunt@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean time to healing of genital ulcers (time frame: The participants will be assessed at thirteen time points (or until the complete healing of genital ulcers) for each treatment period. Each treatment period is estimated to take 30 days.There will be a final follow-up 30 days after the treatment.). 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; AzTrials last refreshed its copy on 7 Nov 2026.