Pyoderma GangrenosumPhase 2NCT06092216
Spesolimab in Pyoderma Gangrenosum
A Phase 2 study testing Spesolimab in people with Pyoderma Gangrenosum, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pyoderma Gangrenosum
- What is being tested?
- Spesolimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Sep 2023to 14 Jan 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 14Show fewer
- 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).
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: Characterizing Pyoderma Gangrenosum Lesion Regression and Remission by IL-36 Receptor Targeting With Spesolimab
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).
Read the detailed description
To date, there is no gold standard for treatment of PG. Patients with pyoderma gangrenosum suffer from severe pain and poor quality of life due to frequent dressing changes and disfiguring lesions. More importantly, rapidly progressing ulcers present an important risk for infection, morbidity, and mortality for patients. Spesolimab is humanized antagonistic monoclonal IgG1 antibody that blocks human IL36R signalling and subsequent downstream pro-inflammatory pathways. The IL-36 receptor blocker was recently approved for generalized pustular psoriasis (GPP). The research team hypothesize that targeting IL-36 in refractory, ulcerative postoperative PG may result in regression and resolution of a patient's lesions.
There are, at minimum, a total of 14 or 15 visits which will include physical exams, blood testing and infectious disease testing, completing questionnaires, and photographs and assessment of skin affected by PG. Spesolimab will be administered via a 90-minute infusion every 3 or 4 weeks (depending on response at Visit 4) at 8 or 9 Visits during the study.
- Conditions
- Pyoderma Gangrenosum
- Being tested
- Spesolimab
- Medical subject headings
- Pyoderma Gangrenosum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SpesolimabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Spesolimab | Experimental | 900 mg of spesolimab intravenously (IV) Receives: Drug: Spesolimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Global Pyoderma Gangrenosum (GPG) Severity Score
Change in GPG severity score at week 16 from baseline in target lesion
- Completely clear; evidence of cribriform scarring, re-epithelization and possible residual hyperpigmentation. 0% ulceration apparent, lesion is dry.
- 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;
- 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.
- 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
- Severe; 100% active ulcer with violaceous, raised rolled borders. Necrotic tissue may be present.
Measured: Baseline and Week 16
Secondary outcomes (5)
Number of Lesions With Complete Re-epithelization of PG Lesions
Number of lesions with Complete re-epithelization of PG lesions = GPG Score 0
GPG Score 0 - Completely clear; evidence of cribriform scarring, re-epithelization and possible residual hyperpigmentation. 0% ulceration apparent and lesion is dry
Measured: Up to Week 28
Absolute Change in Patient-reported Pain Severity (Pain-VAS) Score
Absolute change at Week 28 from baseline in patient-reported pain severity (Pain-VAS).
Patient Pain Visual Analogue Scale (VAS):
Patients will be asked to report pain scores at each visit. Patients will report scores on a scale of 0 to 10.
0 signifies no pain and 10 signifies the worst pain imaginable. Higher scores indicate increased levels of pain.
Measured: Baseline and at Week 28
Absolute Change in Dermatology Life Quality Index (DLQI)
Absolute Change at week 28 from baseline in DLQI.
The DLQI is a validated questionnaire consisting of 10 questions that has been used in many randomized controlled trials in dermatology.
Scoring of each question is as follows:
Very much - 3 A lot - 2 A little - 1 Not at all - 0 Not relevant - 0 Question unanswered - 0
The DLQI is calculated by summing the score of each question with total scale from 0-30. Higher score represents a quality of life that is more impaired.
Definition of DLQI Scores 0-1 = no effect at all on patient's life 2-5 = small effect on patient's life 6-10 = moderate effect on patient's life 11-20 = very large effect on patient's life 21-30 = extremely large effect on patient's life
A change in DLQI score of at least 4 points is considered clinically important. Such change suggests that there has actually been a meaningful change in that patient's quality of life since the previous measurement of his/her/their DLQI scores.
Measured: Baseline and at Week 28
Number of Recurrence of PG Lesions (GPG >0) After Achieving Complete Re-epithelialization (GPG Score 0) and Spesolimab Cessation
GPG Severity Score Scale:
- Completely clear; evidence of cribriform scarring, re-epithelization and possible residual hyperpigmentation. 0% ulceration apparent, lesion is dry
- 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
- 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 exam.
- 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
- Severe; 100% active ulcer with violaceous, raised rolled borders. Necrotic tissue may be present. No evidence of granulation tissue. Extensive purulent drops on ulcer bed
Measured: 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
Lesions with GPG score of 1
Scale:
- Completely clear; evidence of cribriform scarring, re-epithelization and possible residual hyperpigmentation. 0% ulceration apparent and lesion is dry
- Almost clear; <25% of active ulceration present; more than 90% granulation tissue present with mild pink, slightly elevated borders. Some evidence of re-epithelization. Minimal to no purulent drainage at presentation
- 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.
- 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
- Severe; 100% active ulcer with violaceous, raised rolled borders. Necrotic tissue may be present. No evidence of granulation tissue
Measured: Last dose (Week 26 or 28) up to 16 week post-spesolimab last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2023
- Study start23 Sep 2023
- Primary completion14 Jan 2025
- Study completion14 Jan 2025
Why it stopped: The funding sponsor terminated our study and will conduct a multicenter national study in its place.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pyoderma GangrenosumStudies of the same condition, open and under way.
- More trials run by Icahn School of Medicine at Mount SinaiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label study
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Sponsor type
- University, hospital or other
- Collaborators
- Boehringer Ingelheim
- Responsible party
- Principal investigator, Saakshi Khattri, Icahn School of Medicine at Mount Sinai
- Investigators
- Saakshi Khattri, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06092216
- Sponsor's protocol ID
- STUDY-23-00611
- Status last verified
- Mar 2026
- First submitted
- 16 Oct 2023
- Last updated
- 31 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Data will be analyzed as aggregated data.
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characterizing Pyoderma Gangrenosum Lesion Regression and Remission by IL-36 Receptor Targeting With Spesolimab
- ClinicalTrials.gov ID
- NCT06092216
- Acronym / sponsor ID
- STUDY-23-00611
- Conditions
- Pyoderma Gangrenosum
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The funding sponsor terminated our study and will conduct a multicenter national study in its place.
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Icahn School of Medicine at Mount SinaiUniversity, hospital or other
- Collaborators
- Boehringer Ingelheim
- Study start
- 23 Sep 2023 (actual)
- Primary completion
- 14 Jan 2025 (actual)
- Study completion
- 14 Jan 2025 (actual)
- First posted
- 23 Oct 2023 (actual)
- Results
- Posted 31 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
To date, there is no gold standard for treatment of PG. Patients with pyoderma gangrenosum suffer from severe pain and poor quality of life due to frequent dressing changes and disfiguring lesions. More importantly, rapidly progressing ulcers present an important risk for infection, morbidity, and mortality for patients. Spesolimab is humanized antagonistic monoclonal IgG1 antibody that blocks human IL36R signalling and subsequent downstream pro-inflammatory pathways. The IL-36 receptor blocker was recently approved for generalized pustular psoriasis (GPP). The research team hypothesize that targeting IL-36 in refractory, ulcerative postoperative PG may result in regression and resolution of a patient's lesions.
There are, at minimum, a total of 14 or 15 visits which will include physical exams, blood testing and infectious disease testing, completing questionnaires, and photographs and assessment of skin affected by PG. Spesolimab will be administered via a 90-minute infusion every 3 or 4 weeks (depending on response at Visit 4) at 8 or 9 Visits during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label study
| Arm | Type | Receives |
|---|---|---|
| Spesolimab | Experimental | Drug: Spesolimab900 mg of spesolimab intravenously (IV) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Spesolimab900 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. | Drug | Spesolimab |
Primary outcome
Change in Global Pyoderma Gangrenosum (GPG) Severity Score
Time frame: Baseline and Week 16
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.
5 secondary outcomes
Number of Lesions With Complete Re-epithelization of PG Lesions
Time frame: Up to Week 28
Absolute Change in Patient-reported Pain Severity (Pain-VAS) Score
Time frame: Baseline and at Week 28
Absolute Change in Dermatology Life Quality Index (DLQI)
Time frame: Baseline and at Week 28
Number of Recurrence of PG Lesions (GPG >0) After Achieving Complete Re-epithelialization (GPG Score 0) and Spesolimab Cessation
Time frame: 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
Time frame: Last dose (Week 26 or 28) up to 16 week post-spesolimab last dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. Data will be analyzed as aggregated data.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Saakshi Khattri, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Spesolimab in people with Pyoderma Gangrenosum, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Global Pyoderma Gangrenosum (GPG) Severity Score (time frame: Baseline and Week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2026.