Baricitinib in the Treatment of Adults With Pyoderma Gangrenosum (PG)

NCT04901325 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: Baricitinib for PG Treatment
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

An Open-Label, Proof-Of-Concept, Study of Baricitinib for the Treatment of Pyoderma Gangrenosum

Conditions

Pyoderma Gangrenosum; Skin Diseases; Wound Heal; Pyoderma; Skin Ulcer

Keywords

Baricitinib; JAK Kinase Inhibitors

Outcome Measures

Primary Outcomes (1)

• 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.

  Week 24

Secondary Outcomes (13)

• Physician Global Assessment (PGA)

  Week 24

• Percent change in lesion surface area

  Week 0 and 24

• Mean change in lesion surface area

  Week 0 and 24

• Mean change in Physician Global Assessment (PGA)

  Week 0 and 24

• Sustained healing

  Week 36

Other Outcomes (1)

• Evaluation of cytokine gene expression

  Week 0 and week 24

Study Arms (1)

Baricitinib for PG

EXPERIMENTAL

Subjects with PG will be treated with 4 mg once daily of baricitinib for 24 weeks in addition to starting stable dose (at least 2 weeks) of prednisone at 30 mg daily. Prednisone will be tapered based on a pre-established algorithm assessed by investigator.

Drug: Baricitinib

Interventions

Baricitinib DRUG

Subjects with PG will be treated with 4 mg once daily of baricitinib for 24 weeks.

Also known as: Olumiant

Baricitinib for PG

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 minimum wound size of 4 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.
• Female age 18-99; either of non-childbearing potential or of childbearing potential who test negative for pregnancy and agree to use at least two reliable methods of birth control or remain abstinent during the study and for at least 1 week following the last dose of baricitinib.
• Classic PG defined as deep ulceration with undermining violaceous borders.
• Are candidate for systemic therapy. All participants will be taking and clinically stable 30 mg (same ulcer size) of prednisone fort least two weeks at the start of the study. Patients must have discontinued immunosuppressive therapies (cyclosporine, azathioprine, mycophenolate mofetil, methotrexate, apremilast, dapsone) due to inadequate response or intolerance for at least 4 weeks prior to beginning the study drug.
• Undergoing at least once a week standard of care wound care at home or wound care facility.
• Willingness to travel to Oregon Health and Science University (OHSU) for all study visits, or living \>30 miles from OHSU and willing/able to participate in remote videoconferencing visits with access to a computer with internet and webcam capabilities.

You may not qualify if:

• 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.Patients currently on treatment for active TB with drugs such as strong OAT-3 inhibitors will be excluded from study)
• Clinically serious infection or received intravenous antibiotics for an infection, within 4 weeks of randomization.
• Active viral infection that, based on the investigator's clinical assessment, makes the subject and unsuitable candidate for the study.
• Positive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus.
• Symptomatic herpes zoster infection within 12 weeks of screening or recurrent or disseminated (even a single episode) herpes zoster.
• Symptomatic herpes simplex at the time of randomization or disseminated (even a single episode) herpes simplex
• History of disseminated opportunistic infections (e.g., listeriosis and histoplasmosis).
• Have a history of venous thromboembolism (VTE), or are considered at high risk for VTE as deemed by the investigator, or have 2 or more of the following risk factors for VTE:
• Aged \>65 years.
• Body mass index (BMI) \>35 kg/m2.
• Oral contraceptive use and current smoker.
• Creatinine Clearance \<30 mL/min
• Wound care debridement of any PG ulcer within 2 weeks.
• Intralesional corticosteroids within 4 weeks of screening.

Sponsors & Collaborators

• Oregon Health and Science University: lead

Study Sites (1)

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Related Publications (9)

• Braswell SF, Kostopoulos TC, Ortega-Loayza AG. Pathophysiology of pyoderma gangrenosum (PG): an updated review. J Am Acad Dermatol. 2015 Oct;73(4):691-8. doi: 10.1016/j.jaad.2015.06.021. Epub 2015 Aug 5.

  PMID: 26253362 BACKGROUND

• Alves de Medeiros AK, Speeckaert R, Desmet E, Van Gele M, De Schepper S, Lambert J. JAK3 as an Emerging Target for Topical Treatment of Inflammatory Skin Diseases. PLoS One. 2016 Oct 6;11(10):e0164080. doi: 10.1371/journal.pone.0164080. eCollection 2016.

  PMID: 27711196 BACKGROUND

• Shanmugam VK, McNish S, Shara N, Hubley KJ, Kallakury B, Dunning DM, Attinger CE, Steinberg JS. Chronic leg ulceration associated with polycythemia vera responding to ruxolitinib (Jakafi((R))). J Foot Ankle Surg. 2013 Nov-Dec;52(6):781-5. doi: 10.1053/j.jfas.2013.07.003. Epub 2013 Aug 14.

  PMID: 23953278 BACKGROUND

• Nasifoglu S, Heinrich B, Welzel J. Successful therapy for pyoderma gangrenosum with a Janus kinase 2 inhibitor. Br J Dermatol. 2018 Aug;179(2):504-505. doi: 10.1111/bjd.16468. Epub 2018 May 21. No abstract available.

  PMID: 29451690 BACKGROUND

• Kochar B, Herfarth N, Mamie C, Navarini AA, Scharl M, Herfarth HH. Tofacitinib for the Treatment of Pyoderma Gangrenosum. Clin Gastroenterol Hepatol. 2019 Apr;17(5):991-993. doi: 10.1016/j.cgh.2018.10.047. Epub 2018 Nov 4.

  PMID: 30404036 BACKGROUND

• Palanivel JA, Macbeth AE, Levell NJ. Pyoderma gangrenosum in association with Janus kinase 2 (JAK2V617F) mutation. Clin Exp Dermatol. 2013 Jan;38(1):44-6. doi: 10.1111/j.1365-2230.2012.04375.x. Epub 2012 May 21.

  PMID: 22607468 BACKGROUND

• Ortega-Loayza AG, Nugent WH, Lucero OM, Washington SL, Nunley JR, Walsh SW. Dysregulation of inflammatory gene expression in lesional and nonlesional skin of patients with pyoderma gangrenosum. Br J Dermatol. 2018 Jan;178(1):e35-e36. doi: 10.1111/bjd.15837. Epub 2017 Dec 5. No abstract available.

  PMID: 28734003 BACKGROUND

• Liu D, Ahmet A, Ward L, Krishnamoorthy P, Mandelcorn ED, Leigh R, Brown JP, Cohen A, Kim H. A practical guide to the monitoring and management of the complications of systemic corticosteroid therapy. Allergy Asthma Clin Immunol. 2013 Aug 15;9(1):30. doi: 10.1186/1710-1492-9-30.

  PMID: 23947590 BACKGROUND

• Smolen JS, Genovese MC, Takeuchi T, Hyslop DL, Macias WL, Rooney T, Chen L, Dickson CL, Riddle Camp J, Cardillo TE, Ishii T, Winthrop KL. Safety Profile of Baricitinib in Patients with Active Rheumatoid Arthritis with over 2 Years Median Time in Treatment. J Rheumatol. 2019 Jan;46(1):7-18. doi: 10.3899/jrheum.171361. Epub 2018 Sep 15.

  PMID: 30219772 BACKGROUND

MeSH Terms

Conditions

Pyoderma Gangrenosum; Skin Diseases; Pyoderma; Skin Ulcer

Interventions

baricitinib

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases; Skin Diseases, Vascular

Study Officials

• Alex G Ortega-Loayza, MD, MCR

  Oregon Health & Science University, Department of Dermatology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Dermatology, School of Medicine

Study Record Dates

• First Submitted: May 17, 2021
• First Posted: May 25, 2021
• Study Start: October 3, 2023
• Primary Completion: January 5, 2025
• Study Completion: May 7, 2025
• Last Updated: May 13, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)