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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04399837




Registration number
NCT04399837
Ethics application status
Date submitted
20/05/2020
Date registered
22/05/2020
Date last updated
14/12/2023

Titles & IDs
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.
Secondary ID [1] 0 0
2018-003081-14
Secondary ID [2] 0 0
1368-0027
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Generalized Pustular Psoriasis 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Spesolimab
Treatment: Drugs - Placebo

Experimental: Spesolimab SC low dose -

Experimental: Spesolimab SC medium dose -

Experimental: Spesolimab SC high dose -

Placebo comparator: Placebo -


Treatment: Drugs: Spesolimab
Solution for injection

Treatment: Drugs: Placebo
Solution for injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to First Generalized Pustular Psoriasis (GPP) Flare
Timepoint [1] 0 0
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Secondary outcome [1] 0 0
Key Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 48
Timepoint [1] 0 0
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Secondary outcome [2] 0 0
Time to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 48
Timepoint [2] 0 0
PSS assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Visit window was ±7 days.
Secondary outcome [3] 0 0
Time to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 48
Timepoint [3] 0 0
DLQI assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 24, 36 and 48). Visit window was ±7 days. Time window for Week 48 was from Week 46 to Week 50.
Secondary outcome [4] 0 0
Sustained Remission
Timepoint [4] 0 0
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Secondary outcome [5] 0 0
The Occurrence of Treatment Emergent Adverse Events (TEAEs)
Timepoint [5] 0 0
Up to 62 weeks (for detailed timeframe see description).

Eligibility
Key inclusion criteria
* Patients with a known and documented history of GPP per ERASPEN criteria (see Section 3.3.1) regardless of IL36RN mutation status, with at least 2 presentations of moderate to severe GPP flares with fresh pustulation (new appearance or worsening) in the past.
* Patients with a GPPGA score of 0 or 1 at screening and randomization.
* Patients who are not on concomitant GPP treatment at time of randomization (V2) must have had at least two presentations of moderate to severe GPP flare in the past year, at least one of which had evidence of either fever and/or elevated CRP and/or elevated WBC, and/or asthenia and/or myalgia.
* Patients who are not on concomitant GPP treatment at time of randomization (V2) but who were on concomitant GPP treatment until shortly before randomization (V2) (= 12 weeks before randomization), these patients must have a history of flaring while on concomitant treatment for GPP or in case of dose reduction or discontinuation of their concomitant medication.
* Patients who are on concomitant treatment regimen with retinoids and/or methotrexate and/or cyclosporine must stop at the day of randomization (V2). These patients must have a history of flaring while on concomitant treatment for GPP or in case of dose reduction or discontinuation of these concomitant medications.
* Male or female patients, aged 12 to 75 years at screening. For all patients, a minimum weight of 40 kg is required.
* Signed and dated written informed consent and assent in accordance with ICH-GCP and local legislation prior to admission in the trial.
* 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, parent(s) (or patient's legal guardian) information.
Minimum age
12 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin.
4. Treatment with:

1. 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.
2. 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 Tuberculosis (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)
* Patients with suspected false positive or indeterminate QuantiFERON® (or if applicable, T-Spot®) TB result may be re-tested once
* If QuantiFERON® (or if applicable, T-Spot®) TB testing is not available or provides indeterminate results after repeat testing, a tuberculin skin test (TST) can be performed: A TST reaction of =10mm (=5mm if receiving =15mg/d prednisone or its equivalent) is considered positive.
8. History of allergy/hypersensitivity to the systemically administered trial medication agent or its excipients.

Further exclusion criteria apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/11/2022
Sample size
Target
Accrual to date
Final
123
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Michigan
Country [2] 0 0
United States of America
State/province [2] 0 0
Missouri
Country [3] 0 0
Argentina
State/province [3] 0 0
Caba
Country [4] 0 0
Belgium
State/province [4] 0 0
Bruxelles
Country [5] 0 0
Chile
State/province [5] 0 0
Vitacura
Country [6] 0 0
China
State/province [6] 0 0
Guangzhou
Country [7] 0 0
China
State/province [7] 0 0
Hangzhou
Country [8] 0 0
China
State/province [8] 0 0
Shanghai
Country [9] 0 0
China
State/province [9] 0 0
Shenyang
Country [10] 0 0
China
State/province [10] 0 0
Tianjin
Country [11] 0 0
China
State/province [11] 0 0
Xi'an
Country [12] 0 0
France
State/province [12] 0 0
Nice
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Germany
State/province [14] 0 0
Bad Bentheim
Country [15] 0 0
Germany
State/province [15] 0 0
Bonn
Country [16] 0 0
Germany
State/province [16] 0 0
Frankfurt am Main
Country [17] 0 0
Germany
State/province [17] 0 0
München
Country [18] 0 0
Germany
State/province [18] 0 0
Münster
Country [19] 0 0
Germany
State/province [19] 0 0
Oldenburg
Country [20] 0 0
Germany
State/province [20] 0 0
Würzburg
Country [21] 0 0
Greece
State/province [21] 0 0
Thessaloniki
Country [22] 0 0
Italy
State/province [22] 0 0
Rozzano (MI)
Country [23] 0 0
Japan
State/province [23] 0 0
Aichi, Nagoya
Country [24] 0 0
Japan
State/province [24] 0 0
Fukuoka, Kitakyushu
Country [25] 0 0
Japan
State/province [25] 0 0
Ibaraki, Inashiki-gun
Country [26] 0 0
Japan
State/province [26] 0 0
Saitama, Iruma-gun
Country [27] 0 0
Japan
State/province [27] 0 0
Tokyo, Hachioji
Country [28] 0 0
Japan
State/province [28] 0 0
Tokyo, Shinjuku-ku
Country [29] 0 0
Korea, Republic of
State/province [29] 0 0
Busan
Country [30] 0 0
Korea, Republic of
State/province [30] 0 0
Seoul
Country [31] 0 0
Malaysia
State/province [31] 0 0
Ipoh
Country [32] 0 0
Malaysia
State/province [32] 0 0
Johor Bahru
Country [33] 0 0
Malaysia
State/province [33] 0 0
Kota Kinabalu
Country [34] 0 0
Malaysia
State/province [34] 0 0
Kuala Lumpur
Country [35] 0 0
Malaysia
State/province [35] 0 0
Kuching, Sarawak
Country [36] 0 0
Malaysia
State/province [36] 0 0
Muar
Country [37] 0 0
Malaysia
State/province [37] 0 0
Pulau Pinang
Country [38] 0 0
Mexico
State/province [38] 0 0
Guadalajara
Country [39] 0 0
Mexico
State/province [39] 0 0
Monterrey
Country [40] 0 0
Netherlands
State/province [40] 0 0
Rotterdam
Country [41] 0 0
Philippines
State/province [41] 0 0
Davao City
Country [42] 0 0
Philippines
State/province [42] 0 0
Iloilo City, Iloilo
Country [43] 0 0
Philippines
State/province [43] 0 0
Makati City
Country [44] 0 0
Russian Federation
State/province [44] 0 0
Chelyabinsk
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Kazan
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Kirov
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Saint-Petersburg
Country [48] 0 0
Russian Federation
State/province [48] 0 0
Saratov
Country [49] 0 0
Russian Federation
State/province [49] 0 0
St. Petersburg

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Boehringer Ingelheim
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a study in adolescents and adults with Generalized Pustular Psoriasis (GPP). People between 12 and 75 years old can take part in the study. The study is open to people who had GPP flare-ups in the past but whose skin is clear or almost clear when they join the study. The purpose of the study is to test 3 different doses of a medicine called spesolimab and to see whether it helps to prevent GPP flare-ups.

Participants are put into 4 groups by chance. Three groups get different doses of spesolimab. The fourth group gets a placebo. Placebo looks like spesolimab but does not contain any medicine.

Spesolimab and placebo are given as an injection under the skin. Participants are in the study for about 1 year and 4 months. During this time, they visit the study site about 15 times. For the first 11 months, participants get spesolimab or placebo injections every month. At the study visits, the doctors check participants' skin for signs of a new GPP flare-up. The doctors also check the general health of the participants.

If a participant has a GPP flare-up during the study, more visits may be necessary. In case of a flare-up, participants get a dose of spesolimab as an infusion into a vein.
Trial website
https://clinicaltrials.gov/study/NCT04399837
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04399837