Spesolimab in Pyoderma Gangrenosum

NCT06092216 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: STUDY-23-00611
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to assess the feasibility of using spesolimab in participants with moderate to severe pyoderma gangrenosum. Pyoderma gangrenosum is a rare, inflammatory, autoimmune condition which results in ulceration of skin. The study will also investigate the body's immune response to the spesolimab (when the body detects and defends itself against substances that appear unknown and harmful).

Conditions

Pyoderma Gangrenosum

Outcome Measures

Primary Outcomes (1)

• Change in Global Pyoderma Gangrenosum (GPG) Severity Score

  Change in GPG severity score at week 16 from baseline in target lesion 0\. Completely clear; evidence of cribriform scarring, re-epithelization and possible residual hyperpigmentation. 0% ulceration apparent, lesion is dry. 1. Almost clear; \<25% of active ulceration present; \> 90% granulation tissue present with mild pink, slightly elevated borders. Some evidence of re-epithelization. Minimal to no purulent drainage at presentation; 2. Mild; \<50% of active ulceration with perceptible border elevation with mild red border. Evidence of granulation tissue without any re-epithelization of skin. Few drops purulence appreciated upon examination. 3. Moderate; \<75% active ulceration with marked red, rolled borders and significant purulence. Some evidence of granulation tissue with multiple purulent drops and significant purulence on ulcer bed at presentation 4. Severe; 100% active ulcer with violaceous, raised rolled borders. Necrotic tissue may be present.

  Baseline and Week 16

Secondary Outcomes (5)

• Number of Lesions With Complete Re-epithelization of PG Lesions

  Up to Week 28

• Absolute Change in Patient-reported Pain Severity (Pain-VAS) Score

  Baseline and at Week 28

• Absolute Change in Dermatology Life Quality Index (DLQI)

  Baseline and at Week 28

• Number of Recurrence of PG Lesions (GPG >0) After Achieving Complete Re-epithelialization (GPG Score 0) and Spesolimab Cessation

  Last dose (Week 26 or 28) up to 16 week post-spesolimab last dose

• Number of Lesions With Global Pyoderma Gangrenosum (GPG) Severity Score of 1

  Last dose (Week 26 or 28) up to 16 week post-spesolimab last dose

Study Arms (1)

Spesolimab

EXPERIMENTAL

900 mg of spesolimab intravenously (IV)

Drug: Spesolimab

Interventions

Spesolimab DRUG

900 mg of spesolimab intravenously (IV) administered every 4 weeks at Visits 2, 3, 4, 5, 6, 7, 8, 9. If at visit 4 dosing schedule changes to every 3 weeks, Spesolimab will be administered at Visits 2, 3, 4, 5, 6, 8, 9, 10 and 11.

Spesolimab

Eligibility Criteria

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

You may qualify if:

• Male or female subjects ≥ 18 years of age at the time of signing the informed consent document
• Subject is able to understand and voluntarily sign an informed consent document prior to participation in any study assessments or procedures
• Subject is able to adhere to the study visit schedule and other protocol requirements.
• Subject has clinically diagnosed ulcerative PG with PARACELSUS score greater than or equal to 10
• Subject has at least one clinically measurable ulcerative PG lesion on body that has failed to respond to at least one prior therapy such as (but not limited to) topical corticosteroids, intralesional triamcinolone, prednisone, cyclosporine, IL-23 inhibitor, IL-17 inhibitors, IL-1 inhibitors, or TNF-α- blocker therapy
• Subject has moderate to severe PG as determined by a GPG severity score of ≥3
• Subject is judged to be in otherwise good overall health as judged by the investigator, based on medical history, limited physical examination, and laboratory testing. (NOTE: The definition of good health means a subject does not have uncontrolled significant co-morbid conditions).
• Females of childbearing potential (FCBP) must have a negative pregnancy test at Screening and Baseline. While on investigational product and for at least 28 days after taking the last dose of investigational product, FCBP who engage in activity in which conception is possible must use one of the approved contraceptive options described below:
• Option 1: Any one of the following highly effective contraceptive methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy.
• Or option 2: Male or female condom (latex condom or nonlatex condom NOT made out of natural \[animal\] membrane \[for example, polyurethane\]). PLUS one additional barrier method:
• (a) diaphragm with spermicide
• (b) cervical cap with spermicide;
• or (c) contraceptive sponge with spermicide.
• The female subject's chosen form of contraception must be effective by the time the female subject presents for her Baseline visit (for example, hormonal contraception should be initiated at least 28 days before first spesolimab infusion at Baseline).

You may not qualify if:

• Subject has a persistent or recurring bacterial infection requiring systemic antibiotics, or clinically significant viral or fungal or helminth parasitic infections, within 2 weeks of the Screening Visit. Any treatment of such infections must have been completed at least 2 weeks prior to the Screening Visit and no new/recurrent infections should have occurred prior to the Baseline Visit.
• Subject with current or history of positive human immunodeficiency virus (HIV), or congenital or acquired immunodeficiency (i.e. Common Variable Immunodeficiency \[CVID\]), hepatitis B or C, or active or untreated latent tuberculosis.
• Subject has clinically significant (as determined by the investigator) renal, hepatic, hematologic, intestinal, endocrine, pulmonary, cardiovascular, neurological, psychiatric, immunologic, or other major uncontrolled diseases that will affect the health of the subject during the study, or interfere with the interpretation of study results. Uncontrolled disease defined as hospitalization within 1 month of screening visit or determined by specialist (rheumatologist, gastroenterologist) consulted prior to study start.
• Subject has presence of acute demyelinating neuropathy
• Major surgery (according to the investigator's assessment) performed within 12 weeks prior to receiving first dose of spesolimab or planned during trial such as hip replacement, aneurysm removal, stomach ligation, or otherwise determined by investigator
• Subject has a suspected or active lymphoproliferative disorder or malignancy
• Subject was treated previously with spesolimab or another IL-36R inhibitor biologic
• Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treatment basal or squamous cell carcinoma of the skin or in situ carcinoma of uterine cervix.
• Subject has received a live attenuated vaccine ≤ 30 days prior to study initiation.
• History of adverse systemic or allergic reactions to any component of the study drug.
• Female subject who is pregnant or breast feeding

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead
• Boehringer Ingelheim: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Pyoderma Gangrenosum

Interventions

spesolimab

Condition Hierarchy (Ancestors)

Pyoderma; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular; Skin Ulcer

Results Point of Contact

• Title: Giselle Singer
• Organization: Icahn School of Medicine at Mount Sinai

giselle.singer@mssm.edu

212-241-3288

Study Officials

• Saakshi Khattri, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

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

Model Details: Open-label study

Study Record Dates

• First Submitted: October 16, 2023
• First Posted: October 23, 2023
• Study Start: September 23, 2023
• Primary Completion: January 14, 2025
• Study Completion: January 14, 2025
• Last Updated: March 31, 2026
• Results First Posted: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)