A study to test whether BI 655130 (Spesolimab) prevents flare-ups in patients with Generalized Pustular Psoriasis

• Register: EUCTR
• Last refreshed on: 30 October 2023
• Main ID: EUCTR2018-003081-14-BG
• Date of registration: 14/04/2021
• Prospective Registration: Yes
• Primary sponsor: Boehringer Ingelheim RCV GmbH & Co KG
• Public title: A study to test whether BI 655130 (Spesolimab) prevents flare-ups in patients with Generalized Pustular Psoriasis
• Scientific title: Effisayil™ 2: Multi-center, randomized, parallel group, double blind, placebo controlled, Phase IIb dose-finding study to evaluate efficacy and safety of BI 655130 (Spesolimab) compared to placebo in preventing generalized pustular psoriasis (GPP) flares in patients with history of GPP
• Date of first enrolment: 17/05/2021
• Target sample size: 98
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-003081-14
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 4
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, China, Colombia, Croatia, Czech Republic, Czechia, Egypt, France, Georgia, Germany, Greece, Israel, Italy, Japan, Korea, Republic of, Latvia, Malaysia, Mexico, Netherlands, Philippines, Poland, Russian Federation, Singapore, South Africa, Spain, Taiwan, Thailand, Tunisia, Turkey, Ukraine, United States, Vietnam

Contacts

• Name: CT Disclosure & Data Transparency
• Affiliation: Boehringer Ingelheim Pharma GmbH & Co. KG

• Name: CT Disclosure & Data Transparency
• Affiliation: Boehringer Ingelheim Pharma GmbH & Co. KG

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patients with a known and documented history of GPP per ERASPEN criteria (see Section 3.3.1) regardless of IL36RN mutation status.

2. Patients with a GPPGA score of 0 or 1 at screening and randomization.

3. Male or female patients, aged 12 to 75 years at screening. For all patients, a minimum weight of 40 kg is required.

4. Signed and dated written informed consent and assent in accordance with ICH-GCP and local legislation prior to admission in the trial.

5. Women of childbearing potential (WOCBP)1 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 is provided in the CTP as well as in the patient (or patient’s legal guardian) information.

Additional inclusion criteria will apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 80
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
1. Patients with SAPHO (Synovitis–acne–pustulosis–hyperostosis–osteitis) syndrome.

2. Patients with primary erythrodermic psoriasis vulgaris.

3. Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in AST or ALT or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin.

4. Treatment with:
a. Any restricted medication as specified in the CTP, or any drug considered likely to interfere with the safe conduct of the study, as assessed by the investigator.
b. Any prior exposure to BI 655130 or another IL36R inhibitor biologic.

5. Increased risk of infectious complications (e.g. recent pyogenic infection, any congenital or acquired immunodeficiency (e.g. HIV), past organ or stem cell transplantation), as assessed by the investigator.

6. Relevant chronic or acute infections including active tuberculosis, human immunodeficiency virus (HIV) infection or viral hepatitis at the time of randomization. A patient can be re-screened if the patient was treated and is cured from the acute infection.

7. Active or Latent TB:
• Patients with active tuberculosis should be excluded
• Patients with a positive QuantiFERON® (or if applicable, T-Spot®) TB test during screening are excluded, unless the patient had previous diagnosis of active or latent TB and has completed appropriate treatment per the discretion of the local investigator within the last 3 years and at the latest at the time of screening (i.e. 2 to 4 weeks before study drug administration); patients may be re-screened once to meet this criterion)

8. History of allergy/hypersensitivity to the systemically administered trial medication agent or its excipients.

9. Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal or squamous cell carcinoma of the skin or in situ carcinoma of uterine cervix.

10. Women who are pregnant, nursing, or who plan to become pregnant while in the trial. Women who stop nursing before the study drug administration do not need to be excluded from participating; they should refrain from breastfeeding for 16 weeks after the last study drug administration.

11. Major surgery (major according to the investigator’s assessment) performed within 12 weeks prior to receiving first dose of study drug or planned during the study, e.g. hip replacement, aneurynsm removal, stomach ligation, as assessed by the investigator.

12. Evidence of a current or previous disease, medical condition (including chronic alcohol or drug abuse or congestive heart disease or any condition) other than GPP, surgical procedure, psychiatric or social problems, medical examination finding (including vital signs and electrocardiogram (ECG)), or laboratory value at the screening outside the reference range that in the opinion of the investigator is clinically significant.

Additional exclusion criteria will apply.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Generalized Pustular Psoriasis
MedDRA version: 21.0 Level: LLT Classification code 10037159 Term: Psoriasis pustular System Organ Class: 100000004858

Intervention(s)

Product Code: BI 655130
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Spesolimab
Current Sponsor code: BI 655130
Other descriptive name: MONOCLONAL ANTIBODY ANTI-IGG1
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 60-

Product Code: BI 655130
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Spesolimab
Current Sponsor code: BI 655130
Other descriptive name: MONOCLONAL ANTIBODY ANTI-IGG1
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Primary end point(s): 1) Time to first Generalized Pustular Psoriasis (GPP) flare (defined by increase in Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score by = 2 from baseline and the pustular component of GPPGA = 2) up to week 48.

Timepoint(s) of evaluation of this end point: 1) up to week 48

Main Objective: The primary objective of the trial is to demonstrate a non-flat curve and evaluate the dose-response relationship for 3 subcutaneous dosing regimens of BI 655130 (with each regimen consisting of a single loading dose and a separate maintenance subcutaneous dosing regimen) versus placebo, on the primary endpoint, the time to the first GPP flare onset up to week 48.

Secondary Objective: The secondary objective is to demonstrate superiority versus placebo for each of BI 655130 regimen on the primary endpoint, the time to the first GPP flare onset up to week 48, as well as the key secondary endpoint, the occurrence of at least one GPP flare up to 48 weeks.

Secondary Outcome(s)

Secondary end point(s): 1) The occurrence of at least one GPP flare (defined by increase in GPPGA score by = 2 from baseline and the pustular component of GPPGA = 2) up to week 48.
2) Time to first worsening of Psoriasis Symptom Scale (PSS) up to week 48 defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC, will be considered as onset of a worsening.
3) Time to first worsening of Dermatology Quality of Life Index (DLQI) up to week 48 defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC, will be considered as onset of a worsening.
4) Sustained remission, defined as a patient with a GPPGA score of 0 or 1 (clear or almost clear) at all visits up to week 48, without intake of rescue medication, or investigator-prescribed SoC.
5) The occurrence of treatment emergent adverse events (TEAEs)

Timepoint(s) of evaluation of this end point: 1) up to week 48
2) up to week 48
3) up to week 48
4) up to week 48
5) up to week 48

Secondary ID(s)

2018-003081-14-DE

1368-0027

Source(s) of Monetary Support

Boehringer Ingelheim RCV GmbH & Co KG,

Ethics review

Status: Approved
Approval date: 17/05/2021
Contact:

Results

• Results available: Yes
• Date Posted: 07/06/2023
• Date Completed: 23/11/2022
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-003081-14/results