NCT04186559

Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers

Clinical Protocol to Evaluate Use of Topical Pentoxifylline Gel on Behcet's Disease Genital Ulcers

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Silk Road Therapies, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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

Interventions

Type	Name	Description
DRUG	Topical Pentoxifylline Gel (Vehicle +PTX)	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.
DRUG	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.

Timeline

• Start date: 2024-02-01
• Primary completion: 2024-11-01
• Completion: 2024-12-01
• First posted: 2019-12-04
• Last updated: 2023-08-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study