An Open-Label, Proof-Of-Concept, Study of Baricitinib for the Treatment of Pyoderma Gangrenosum
This is a Phase II study that will be open label and include a total of 20 patients who will receive the investigational product. PG will be defined by the investigator and a second reviewer on the basis of results from clinical, histological and laboratory assessments. These patients will undergo 24 weeks of baricitinib dosed daily and stable dose of prednisone dosed daily with follow-up until week 36.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Subjects with PG will be treated with 4 mg once daily of baricitinib for 24 weeks.
Oregon Health and Science University
Portland, Oregon, United States
Healing
defined as 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: Week 24
Physician Global Assessment (PGA)
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-36 52. This scale has been used in previous trials: 1. 0 = total resolution of target ulcer with no signs of active PG 2. 1= almost completely healed target ulcer with only minimal signs of active PG 3. 2 = evidence of target ulcer healing which involves at least 50% of ulcer/ulcer margin 4. 3 = evidence of target ulcer healing which involves less than 50% of ulcer/margin 5. 4 = no evidence of target healing ulcer
Time frame: Week 24
Percent change in lesion surface area
The percent change in surface area of target lesion of PG (two-dimensional surface in cm²) using digital photography and acetate tracing at Week 0 and Week 24
Time frame: Week 0 and 24
Mean change in lesion surface area
The mean change in surface area of target lesion of PG (two-dimensional surface in cm²) using digital photography and acetate tracing
Time frame: Week 0 and 24
Mean change in Physician Global Assessment (PGA)
The mean change in Physician global assessment (PGA) 5-point scale at week 0 to week 24
Time frame: Week 0 and 24
Sustained healing
The proportion of patients with target ulcer that remains healed by week 36
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Time frame: Week 36
Decrease in ulcer area size
The proportion of patients with decrease in ulcer area size of at least 50% after treatment at week 24
Time frame: Week 24
Time to healing
Time to which sterile dressings are not required.
Time frame: Over 36-week period of study.
Time to recurrence (weeks)
Interval between target lesion healing and further episodes of PG at any site through the study.
Time frame: 36 weeks
Number of treatment failures
Treatment intolerance, number of patients switching into standard of care or target lesion unhealed.
Time frame: By week 24
Adverse reactions to medications
Possibly-, probably- or related throughout the study.
Time frame: Over 24-week period of study.
Quality of life change (as measured by the Dermatology Life Quality Index)
Proportion of subjects achieving a 4-point change in quality of life measured by the Dermatology Life Quality Index (DLQI) at week 24, and mean change in DLQI score at week 24. The DLQI is a validated tool for inflammatory skin conditions. It is a 10-question survey, scored 0 - 30 points. For inflammatory skin conditions, a 4-point change in DLQI score is considered clinically important.
Time frame: Week 24
Mean change in quality of life (measured by Dermatology Life Quality Index)
Mean change in DLQI score from week 0 to week-24.
Time frame: Week 0 and 24
Skin pain scale
The proportion of patients with a 2 point decrease in the point numeric pain rating scale (NRS) at week 0 and week-24. The pain NRS is a subject-administered, 11-point horizontal scale anchored at 0 and 10, with 0 representing "no pain" and 10 representing "worst pain imaginable."
Time frame: Week 0 to 24