Clinical trial NCT06624670: A Study to Test Whether Spesolimab Helps People With a Skin Condition Called Pyoderma Gangrenosum
Official title: A Multi-centre, Randomised, Placebo-controlled, Double-blind, Parallel-group Trial to Evaluate Safety and Efficacy of Spesolimab (BI 655130) in Adult Patients With Ulcerative Pyoderma Gangrenosum (PG) Who Require Systemic Therapy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Pyoderma Gangrenosum
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2024-10-03
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to find out whether a medicine called spesolimab helps people with pyoderma gangrenosum (PG). The main aim is to see whether spesolimab leads to closure of PG ulcers. This study is open to adults with ulcerative PG with at least 1 ulcer that measures between 5 cm\^2 to 80 cm\^2 in size.

This study has 2 parts. In Part 1, participants are put into groups randomly, which means by chance. 1 group gets spesolimab and the other group gets placebo. Placebo injections look like spesolimab injections, but do not contain any medicine. Every participant has a 2 in 3 chance of getting spesolimab. For the first 8 weeks, participants also take corticosteroid medicine by mouth.

In Part 2, participants are put into groups again. Participants without open ulcers have an equal chance of getting spesolimab or placebo. Participants with open skin ulcers will get spesolimab.

In both parts, participants receive spesolimab or placebo as an infusion into a vein every 4 weeks.

Participants are in the study for about 1.5 years. During this time, they visit the study site 20 times. At study visits, doctors check the participant's skin for signs of PG. The doctors also regularly check participants' health and take note of any unwanted effects. The results of the groups are compared to see whether the treatment works.


Eligibility criteria: Inclusion criteria:

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 cm\^2) 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 the protocol.

Exclusion criteria:

1. Trial participants with non-PG lesions.
2. Trial participants with a target PG ulcer measuring \>80 cm\^2.
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.

Further exclusion criteria apply.

Primary outcomes:
measure: 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)
timeFrame: Up to Week 28.
Secondary outcomes:
measure: 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)
timeFrame: Up to Week 28.
measure: Achievement of 50% area reduction (PGAR-50) of the target PG ulcer at any time up to Week 26
timeFrame: Up to Week 26.
measure: Achievement of ≥ 3 point reduction in NRS Pain score from baseline at Week 26
timeFrame: At baseline and at Week 26.
measure: Achievement of a DLQI of ≤ 5 at Week 26
timeFrame: At Week 26.
measure: Achievement of PGAR-100 of any measurable PG ulcer (≥5 cm^2 at baseline) at any time up to Week 26 and confirmed at the next consecutive visit (at least 2 weeks later)
timeFrame: Up to Week 28.
measure: Achievement of PGAR-100 of all measurable PG ulcers (≥5 cm^2 at baseline) at any time up to Week 26 and confirmed at the next consecutive visit (at least 2 weeks later)
timeFrame: Up to Week 28.
measure: 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
timeFrame: Up to Week 52.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Medical Dermatology Specialists Phoenix
state: Arizona
city: Orange
country: United States
facility: University of California Irvine
state: California
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Indianapolis
country: United States
facility: Dawes Fretzin Clinical Research Group, LLC-Indianapolis -68995
state: Indiana
city: New Orleans
country: United States
facility: Tulane University Hospital and Clinic
state: Louisiana
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Clinics Heater Road
state: New Hampshire
city: Lake Success
country: United States
facility: Dermatology at Lake Success
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Fargo
country: United States
facility: Red River Research Partners, LLC
state: North Dakota
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Lewisville
country: United States
facility: Epiphany Dermatology
state: Texas
city: Murray
country: United States
facility: University of Utah Health
state: Utah
city: Caba
country: Argentina
facility: Centro de Investigaciones y Prevencion Cardiovascular CIPREC
city: Caba
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: Rosario
country: Argentina
facility: Instituto de Especialidades de la Salud Rosario
city: Darlinghurst
country: Australia
facility: Skin and Cancer Foundation
state: New South Wales
city: St Leonards
country: Australia
facility: Royal North Shore Hospital-St Leonards-20807
state: New South Wales
city: Melbourne
country: Australia
facility: Alfred Hospital
state: Victoria
city: Graz
country: Austria
facility: Medical University of Graz State Hospital - University Hospital Graz
city: Salzburg
country: Austria
facility: LKH Salzburg University Hospital
city: Leuven
country: Belgium
facility: UZ Leuven
city: Brasilia
country: Brazil
facility: Chronos Pesquisa Clinica
city: Santo André
country: Brazil
facility: Faculdade de Medicina do ABC
city: Sao Paulo
country: Brazil
facility: Hospital Alemao Oswaldo Cruz
city: Edmonton
country: Canada
facility: Rejuvenation Dermatology Clinic
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta Hospital (University of Alberta)
state: Alberta
city: Montreal
country: Canada
facility: McGill University Health Centre (MUHC)
state: Quebec
city: Quebec
country: Canada
facility: Centre de Recherche Saint-Louis
city: Beijing
country: China
facility: Peking University People's Hospital
city: Beijing
country: China
facility: Peking University Third Hospital
city: Changchun
country: China
facility: The First Hospital of Jilin University
city: Changsha
country: China
facility: The Second Xiangya Hospital Of Central South University
city: Chengdu
country: China
facility: West China Hospital
city: Hangzhou
country: China
facility: The First Affiliated Hospital, Zhejiang University
city: Hangzhou
country: China
facility: The Second Affiliated Hospital Zhejiang University School of Medicine
city: Hangzhou
country: China
facility: Hangzhou Third People's Hospital
city: Jinan
country: China
facility: Shandong Provincial Hospital of Dermatology
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shenzhen
country: China
facility: The University of Hong Kong-Shenzhen Hospital
city: Wuhan
country: China
facility: Wuhan Union Hospital
city: Xi'an
country: China
facility: Second Affiliated Hospital of Xi'an JiaoTong University
city: Helsinki
country: Finland
facility: HUS Tulehduskeskus /Ihosairauksien linja
city: Antony
country: France
facility: HOP Privé Antony
city: Nice Cedex 3
country: France
facility: HOP l'Archet
city: Paris
country: France
facility: HOP Saint-Louis
city: Berlin
country: Germany
facility: Charite Universitätsmedizin Berlin KöR
city: Erlangen
country: Germany
facility: Universitätsklinikum Erlangen
city: Essen
country: Germany
facility: Universitätsklinikum Essen AöR
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg AÖR
city: Milano
country: Italy
facility: Fondazione IRCCS Ca'Granda-Ospedale Maggiore Policlinico
city: Pisa
country: Italy
facility: Azienda Ospedaliera Universitaria Pisana
city: Roma
country: Italy
facility: Istituto Dermatologico S. Maria e S. Gallicano IRCCS - IFO
city: Roma
country: Italy
facility: AOU Policlinico Umberto I
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli IRCCS
city: Rozzano (MI)
country: Italy
facility: Istituto Clinico Humanitas
city: Torino
country: Italy
facility: AO Città della Salute e Scienza
city: Aichi, Toyoake
country: Japan
facility: Fujita Health University Hospital
city: Hokkaido, Sapporo
country: Japan
facility: Hokkaido University Hospital
city: Hyogo, Nishinomiya
country: Japan
facility: Hyogo College of Medicine Hospital
city: Mie, Tsu
country: Japan
facility: Mie University Hospital
city: Miyagi, Sendai
country: Japan
facility: Tohoku University Hospital
city: Tochigi, Shimotsuke
country: Japan
facility: Jichi Medical University Hospital
city: Tokyo, Itabashi-ku
country: Japan
facility: Teikyo University Hospital
city: Tokyo, Itabashi-ku
country: Japan
facility: Nihon University Itabashi Hospital
city: Ipoh
country: Malaysia
facility: Hospital Raja Permaisuri Bainun
city: Kuching
country: Malaysia
facility: Sarawak General Hospital
city: Pulau Pinang
country: Malaysia
facility: Hospital Pulau Pinang-Pulau Pinang-21953
city: Oslo
country: Norway
facility: Oslo Universitetssykehus HF, Rikshospitalet
city: Stavanger
country: Norway
facility: Helse Stavanger, Stavanger Universitetssykehus
city: Warsaw
country: Poland
facility: Military Medical Institute- National Research Institute
city: Wroclaw
country: Poland
facility: Cityclinic Medical and Psychological Clinic Matusiak Partnership
city: Coimbra
country: Portugal
facility: ULS de Coimbra, E.P.E.
city: Lisboa
country: Portugal
facility: ULS de São José, E.P.E. - Hospital Sto. António Capuchos
city: Lisboa
country: Portugal
facility: ULS de Santa Maria, E.P.E
city: Porto
country: Portugal
facility: ULS de Santo Antônio, E.P.E - Centro Hospitalar Universitário de Santo António
city: Madrid
country: Spain
facility: Hospital Ramón y Cajal
city: Malaga
country: Spain
facility: Hospital Virgen de la Victoria
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset Solna
city: Zürich
country: Switzerland
facility: University Hospital Zurich
Study officials:
References: