A study to test whether spesolimab helps people with a skin condition called pyoderma gangrenosum

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

13 Feb 2025 → ongoing

Decision date (initial)

2024-12-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-514306-31-00

WHO UTN

U1111-1306-8055

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Safety

The primary objective is to demonstrate superiority of spesolimab over placebo in the difference in the proportion of trial participants with achievement of complete closure (100% pyoderma gangrenosum (PG) area reduction, PGAR-100) of the target PG ulcer at any time up to Week 26.

Secondary objectives 1

1. Secondary objectives are to demonstrate superiority of spesolimab against placebo for the (key) secondary endpoints.

Conditions and MedDRA coding

Pyoderma gangrenosum

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency

Plan to share IPD

Yes

IPD plan description

Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed “Document Sharing Agreement”. Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined on the website. Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program. Access Criteria: For study documents – upon signing of a ‚Document Sharing Agreement‘. For study data – 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Adult trial participants, aged ≥18 years (if local legislation for age of consent differs, then local legislation will be followed) at screening.
2. Signed and dated written informed consent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission to the trial.
3. A confirmed diagnosis of ulcerative pyoderma gangrenosum (PG) (≥10 points on the PARACELSUS score) that requires systemic therapy in the opinion of the investigator. The diagnosis needs to be confirmed by an Adjudication Committee. Trial participants with mixed PG subtypes are eligible as long as the target lesion is of the ulcerative subtype.
4. At least one measurable (defined as measuring ≥5 cm2) PG ulcer. In trial participants with more than one PG ulcer, the target PG ulcer will be selected by the investigator and confirmed by external Adjudication Committee.
5. At the time of the Screening Visit, a maximum duration of 6 months since the target ulcer in the current PG episode was diagnosed. Target ulcers >6 months since diagnosis are allowed if they are active and progressing, as judged by the investigator and confirmed by an Adjudication Committee.
6. Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria and instructions on the duration of use is provided in the participant information and in Section 4.2.2.3 of the protocol.

Exclusion criteria 9

1. Trial participants with non-PG lesions.
2. Trial participants with a target PG ulcer measuring >80 cm2.
3. Trial participants with chronic, non-inflamed PG wounds or ulcers that are not responsive to immunosuppressive therapy, as determined by an Adjudication Committee.
4. Presence of active ulcer infection at the Screening Visit (unless treated and resolved prior to administration of the first dose of trial medication) based on investigator assessment.
5. Presence of persistent or recurring bacterial infection requiring systemic antibiotic therapy; or clinically significant viral, fungal, or parasitic infections within 2 weeks prior to the Screening Visit. Any such infection must be resolved, with treatment completed ≥2 weeks prior to the Screening Visit. No new/recurrent infections should have occurred prior to Visit 2.
6. Active or latent tuberculosis (TB) • Participants with active TB are excluded • Participants with latent TB may be included if treatment of latent TB, as per local guidelines, is initiated prior to randomization and completed during the course of the trial.
7. Chronic or acute infections including Human immunodeficiency virus (HIV) infections and viral hepatitis (including occult hepatitis); the corresponding laboratory tests will be performed during screening. A trial participant can be re-screened if the trial participant was treated and is cured from the acute infection.
8. Severe, progressive, or uncontrolled hepatic disease, defined as >3x Upper Limit of Normal (ULN) elevation in Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) or alkaline phosphatase, or >2x ULN elevation in total bilirubin.
9. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Achievement of complete closure (PGAR-100 (100% pyoderma gangrenosum area reduction)) of the target PG ulcer at any time up to Week 26 and confirmed at the next consecutive visit (at least 2 weeks later)

Secondary endpoints 7

1. Key secondary endpoint: Achievement of PGAR-100 of the target PG ulcer at Week 26 confirmed at the next consecutive visit (at least 2 weeks later)
2. Achievement of 50% area reduction (PGAR-50) of the target PG ulcer at any time up to Week 26
3. Achievement of ≥ 3 point reduction in NRS Pain score from baseline at Week 26
4. Achievement of a DLQI of ≤ 5 at Week 26
5. Achievement of PGAR-100 of any measurable PG ulcer (≥5 cm2 at baseline) at any time up to Week 26 and confirmed at the next consecutive visit (at least 2 weeks later)
6. Achievement of PGAR-100 of all measurable PG ulcers (≥5 cm2 at baseline) at any time up to Week 26 and confirmed at the next consecutive visit (at least 2 weeks later)
7. Time to recurrence among trial participants who had achieved complete response (CR, complete closure of all PG ulcers) at Week 26 up to Week 52. Recurrence is defined as emergence of the disease (PG ulcer[s]) at the previous ulcer sites(s) or emergence of any new PG ulcer(s)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50, Sant Cugat Del Valles Sant Cugat Del Valles

City

Barcelona

Postcode

08174

Country

Spain

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

11 EU/EEA countries · 33 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 176 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications