Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers

NCT04186559 | Unknown Phase 2 FDA Drug

• 1 other identifier: P145440V01A
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Behcet Syndrome; Behcet Disease

Outcome Measures

Primary Outcomes (3)

• 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.

  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).

  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).

  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.

Study Arms (2)

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.

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.

Drug: Topical Pentoxifylline Gel (Vehicle +PTX); Drug: Topical Placebo Gel (Vehicle)

Interventions

Topical Pentoxifylline Gel (Vehicle +PTX) DRUG

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.

Also known as: Trental

Topical pentoxifylline (PTX) gel; Topical placebo gel

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.

Topical pentoxifylline (PTX) gel; Topical placebo gel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Silk Road Therapies, Inc.: lead

Study Sites (1)

Silk Road Therapies

Washington D.C., District of Columbia, 20008, United States

Location

MeSH Terms

Conditions

Behcet Syndrome

Interventions

Pentoxifylline

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases; Uveitis, Anterior; Panuveitis; Uveitis; Uveal Diseases; Eye Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular

Intervention Hierarchy (Ancestors)

Theobromine; Xanthines; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Andew J Sulich, MD

  Shores Rheumatology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Semsi B Derkunt, PHD

CONTACT

631-219-6223; bderkunt@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2019
• First Posted: December 4, 2019
• Study Start: February 1, 2024
• Primary Completion: November 1, 2024
• Study Completion: December 1, 2024
• Last Updated: August 3, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Beginning 3 months following publication. No end date.
• Access Criteria: Proposals should be directed to bderkunt@gmail.com. Anyone who provides a methodologically sound proposal will be approved to perform any type of analysis to achieve the aims in the proposals. To gain access, data requestors will need to sign a data access agreement. Data will be made available indefinitely at (link to be included).

Locations

US (1)