Topical Ruxolitinib in the Treatment of Adults With Pyoderma Gangrenosum

ConditionPyoderma Gangrenosum

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18-99

SponsorOregon Health and Science University

About this trial

This is a single-center, open-label, single-arm pilot study evaluating topical ruxolitinib (OPZELURA) as a treatment for adults with Pyoderma Gangrenosum (PG), a chronic inflammatory skin condition that causes painful ulcers. Approximately 12 participants will apply ruxolitinib cream twice daily for 24 weeks while continuing standard wound care, with the goal of determining whether the drug helps target ulcers heal completely without the need for systemic immunosuppressive therapy. Participants will be followed for a total of 28 weeks, with the option to continue treatment in a 48-week open-label extension if they respond well and remain safe on the drug. The study is sponsored by Oregon Health \& Science University and funded by Incyte Corporation.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Willingness to comply with study procedures/requirements

Capable of giving informed consent

Diagnosis of at least one PG ulcer by clinical, histological and laboratory assessments with a maximum wound size of 10 cm2.

Male age 18-99 who agree to not father a child or donate sperm while on study and at least 1 week following last dose of the study drug. If subject is a sexually active male and could cause a pregnancy, subject must be sure that female partner(s) are using birth control that works well or not have sex.

Disqualifiers

Patients with pyoderma gangrenosum who have more than 3 ulcers measuring total >30 cm2

Have history of malignancy or lymphoproliferative disease; or have signs or symptoms suggestive of possible lymphoproliferative disease, including lymphadenopathy or splenomegaly; or have active primary or recurrent malignant disease; or have been in remission from clinically significant malignancy for < 5years.

Patients with cervical carcinoma in situ, basal cell carcinoma or squamous cell carcinomas who have had active disease within 3 years of screening for this study.

Active, untreated, acute or chronic infection (such as untreated tuberculosis), or immunocompromised to an extent that such that participation in the study would pose an unacceptable risk to the subject. (Treated infections such as latent tuberculosis after completion of the appropriate therapy are not excluded.)

Trial design

Design model

Single group

Treatments tested in this trial

topical ruxolitinib 1.5% cream
Drug
topical ruxolitinib 1.5% cream applied to wound bed twice daily for 24 weeks.

Treatment groups

12 Participants

are divided into 1 treatment group

Group A: TreatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Complete healing of target ulcer

The proportion of patients with complete re-epithelization, defined as 100% re-epithelialization without any drainage, of the target ulcer at week 24.

Time frame

Up to 24 weeks

Secondary outcomes

PGA between 0-1

1\. Assessing the proportion of patients that show target ulcer healing in response to study treatment as measured by achieving PGA between 0 and 1 after treatment with baricitinib at week-24. This scale has been used in previous trials: 0 = total resolution of target ulcer with no signs of active PG 1= almost completely healed target ulcer with only minimal signs of active PG 2 = evidence of target ulcer healing which involves at least 50% of ulcer/ulcer margin 3 = evidence of target ulcer healing which involves less than 50% of ulcer/margin 4 = no evidence of target healing ulcer

Time frame

Through week 24

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

To determine the safety of ruxolitinib for the treatment of subjects with PG who are candidates for systemic therapy, by the number of participants who experienced treatment-related adverse events while on study.

Time frame

Through week 24

Impact of skin disease on quality of life using Skindex Mini

A 3-item questionnaire assessing three domains of quality of life impact; symptoms, emotions, and function related to skin disease on a scale of 0 (never bothered) to 5 (always bothered)

Time frame

By week 28

Quality of life via Participant Global Assessment

Measures an individual's overall impression of their health, disease severity, or treatment response on a scale of clear to severe.

Time frame

By week 28

Other outcomes

Cytokine markers changed due to treatment

Evaluation of cytokine gene expression before and after treatment in skin and blood samples

Time frame

Through week 24

Evaluate imaging structures using HFUS

Use high-frequency ultrasound imaging to predict healing via the presence of hyperechoic oval structures, hair tracts, and U-shaped vessels.

Time frame

Through week 24

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Oregon Health and Science University

Lead sponsor

Incyte Corporation

Collaborator

Source ClinicalTrials.gov

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