Clinical Trials List
Protocol Number1368-0013
NCT Number(ClinicalTrials.gov Identfier)NCT03782792
2019-01-22 - 2021-03-15
Phase II
Terminated4
ICD-10L40.1
Generalized pustular psoriasis
ICD-9694.3
Impetigo herpetiformis
Effisayil™ 1:Multi-center, Double-blind, Randomised, Placebo-controlled, Phase II Study to Evaluate Efficacy, Safety and Tolerability of a Single Intravenous Dose of BI 655130 in Patients With Generalized Pustular Psoriasis (GPP) Presenting With an Acute Flare of Moderate to Severe Intensity.
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Trial Applicant
Boehringer Ingelheim
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Sponsor
Boehringer Ingelheim
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
- Yu-Huei Huang Division of Dermatology
- 陳偉迪 Division of Dermatology
- Chung-Yao Hsu Division of Dermatology
- Chun-Bing Chen Division of Dermatology
- 吳吉妮 Division of Dermatology
- 王芳穎 Division of Dermatology
- 紀景琪 Division of Dermatology
- Chun-Wei Lu Division of Dermatology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- 吳貞宜 Division of Dermatology
- Chih-Chiang Chen Division of Dermatology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Chih-Chieh Chan Division of Dermatology
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Audit
None
Condition/Disease
Generalized Pustular Psoriasis (GPP)
Objectives
To evaluate efficacy, safety, and tolerability of BI 655130 compared to placebo in patients with
Generalized Pustular Psoriasis (GPP) presenting with an acute flare of moderate to severe intensity.
Test Drug
Spesolimab (BI 655130)
Active Ingredient
BI 655130
Dosage Form
solution for infusion
Dosage
450 mg/vial (60 mg/mL)
Endpoints
Primary endpoints
The co-primary endpoints of the study are:
A Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week
1.
A GPPGA pustulation sub-score of 0 indicating no visible pustules at Week 1.
For the estimand concept on the above-defined co-primary binary endpoint definition(s), death or any
use of escape medication (see Section 3.1) prior to Week 1 will be considered to represent a nonresponse at the Week 1 time-point.
2.1.3 Secondary endpoints
Secondary Endpoints at Week 4:
Secondary Endpoints of the study at Week 4 which are included in the statistical testing strategy in a
hierarchical manner subsequent to performance of the tests on the co-primary endpoints (see Section
7.2) are:
A Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4.
Change from baseline in Pain Visual Analog Scale (VAS) score at Week 4.
Change from baseline in Psoriasis Symptom Scale (PSS) score at Week 4.
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score
at Week 4.
Other secondary endpoints of the study at Week 4 are:
A Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week
4.
A GPPGA pustulation sub-score of 0 indicating no visible pustules at Week 4.
A GPPASI 50 at Week 4.
The percent reduction in GPPASI from baseline at Week 4.
A GPPASI 50 at Week 1.
The percent reduction in GPPASI from baseline at Week 1.
The occurrence of Treatment Emergent Adverse Events (TEAEs)
The co-primary endpoints of the study are:
A Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week
1.
A GPPGA pustulation sub-score of 0 indicating no visible pustules at Week 1.
For the estimand concept on the above-defined co-primary binary endpoint definition(s), death or any
use of escape medication (see Section 3.1) prior to Week 1 will be considered to represent a nonresponse at the Week 1 time-point.
2.1.3 Secondary endpoints
Secondary Endpoints at Week 4:
Secondary Endpoints of the study at Week 4 which are included in the statistical testing strategy in a
hierarchical manner subsequent to performance of the tests on the co-primary endpoints (see Section
7.2) are:
A Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4.
Change from baseline in Pain Visual Analog Scale (VAS) score at Week 4.
Change from baseline in Psoriasis Symptom Scale (PSS) score at Week 4.
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score
at Week 4.
Other secondary endpoints of the study at Week 4 are:
A Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week
4.
A GPPGA pustulation sub-score of 0 indicating no visible pustules at Week 4.
A GPPASI 50 at Week 4.
The percent reduction in GPPASI from baseline at Week 4.
A GPPASI 50 at Week 1.
The percent reduction in GPPASI from baseline at Week 1.
The occurrence of Treatment Emergent Adverse Events (TEAEs)
Inclution Criteria
1. a. Patients with GPPGA score of 0 or 1 and a known and documented history of GPP (per
ERASPEN criteria) regardless of IL36RN mutation status, and in addition with previous evidence
of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis
with peripheral blood neutrophilia (above ULN)
OR
b. Patients with an acute flare of moderate to severe intensity meeting the ERASPEN criteria of
GPP with a known and documented history of GPP (per ERASPEN criteria) regardless of IL36RN
mutation status, and in addition with previous evidence of fever, and/or asthenia, and/or myalgia,
and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above
ULN).
OR
c. Patients with first episode of an acute GPP flare of moderate to severe intensity with evidence of
fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with
peripheral blood neutrophilia (above ULN). For these patients the diagnosis will be confirmed
retrospectively by a central external expert/committee.
2. Patients may or may not be receiving background treatment with retinoids and/or methotrexate
and/or cyclosporine. Patients must discontinue retinoids/methotrexate/cyclosporine prior to
receiving the first dose of BI 655130 or placebo.
3. Male or female patients, aged 18 to 75 years at screening.
4. Signed and dated written informed consent prior to admission to the study in accordance with ICHGCP and local legislation prior to start of any screening procedures.
5. Women of childbearing potential 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
Section 4.2.2.3 as well as in the patient information. Note: A woman is considered of childbearing
potential (WOCBP), i.e. fertile, following menarche and until becoming postmenopausal unless
permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy
and bilateral oophorectomy. Tubal ligation is not a method of permanent sterilization. A
postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
ERASPEN criteria) regardless of IL36RN mutation status, and in addition with previous evidence
of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis
with peripheral blood neutrophilia (above ULN)
OR
b. Patients with an acute flare of moderate to severe intensity meeting the ERASPEN criteria of
GPP with a known and documented history of GPP (per ERASPEN criteria) regardless of IL36RN
mutation status, and in addition with previous evidence of fever, and/or asthenia, and/or myalgia,
and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above
ULN).
OR
c. Patients with first episode of an acute GPP flare of moderate to severe intensity with evidence of
fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with
peripheral blood neutrophilia (above ULN). For these patients the diagnosis will be confirmed
retrospectively by a central external expert/committee.
2. Patients may or may not be receiving background treatment with retinoids and/or methotrexate
and/or cyclosporine. Patients must discontinue retinoids/methotrexate/cyclosporine prior to
receiving the first dose of BI 655130 or placebo.
3. Male or female patients, aged 18 to 75 years at screening.
4. Signed and dated written informed consent prior to admission to the study in accordance with ICHGCP and local legislation prior to start of any screening procedures.
5. Women of childbearing potential 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
Section 4.2.2.3 as well as in the patient information. Note: A woman is considered of childbearing
potential (WOCBP), i.e. fertile, following menarche and until becoming postmenopausal unless
permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy
and bilateral oophorectomy. Tubal ligation is not a method of permanent sterilization. A
postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
Exclusion Criteria
1. Patients with SAPHO (Synovitis–acne–pustulosis–hyperostosis–osteitis) syndrome.
2. Patients with primary erythrodermic psoriasis vulgaris.
3. Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that
are restricted to psoriatic plaques.
4. Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP).
5. Immediate life-threatening flare of GPP or requiring intensive care treatment, according to the
investigator’s judgement. Life-threatening complications mainly include, but are not limited to,
cardiovascular/cytokine driven shock, pulmonary distress syndrome, or renal failure.
6. 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.
7. Treatment with:
a. Any restricted medication as specified in Table 4.2.2.1: 1, 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
8. Patients with dose escalation of their maintenance therapy with cyclosporine and/or methotrexate
and/or retinoids within the 2 weeks prior to receiving the first dose of BI 655130/ placebo.
9. The initiation of systemic agents such as cyclosporine and/or retinoids and/or methotrexate 2
weeks prior to receiving the first dose of BI 655130/ placebo.
10. Patients with congestive heart disease, as assessed by the investigator.
11. Active systemic infections (Fungal and bacterial disease) during the last 2 weeks prior to receiving
first drug administration, as assessed by the investigator.
12. 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.
13. Relevant chronic or acute infections including HIV or viral hepatitis. For patients screened while
having a flare (inclusion criteria 1b or 1c), if Visit 1 HIV or viral hepatitis results are not available
in time for randomization, these patients may receive randomized treatment as long as the
investigator has ruled out active disease based on available documented history (i.e. negative HIV
and viral hepatitis test results) within 3 months prior to Visit 2. A patient can be re-screened if the
patient was treated and is cured from acute infection.
14. Active or Latent TB:
QuantiFERON® TB test will be performed at screening. If the result is positive, the patient may
participate in the study if further work up (according to local practice/guidelines) establishes
conclusively that the patient has no evidence of active tuberculosis. Active TB patients must be
excluded. If presence of latent tuberculosis is established, then treatment should have been initiated
and maintained according to local country guidelines. For patients screened while having a flare
(inclusion criteria 1b or 1c), if the TB test results are not available in time for randomization, these
patients may receive randomized treatment (provided they meet all other inclusion/exclusion
criteria) as long as the investigator has ruled out active disease based on available documented
history (i.e. negative for active TB) within 3 months prior to Visit 2.
15. History of allergy/hypersensitivity to a systemically administered trial medication agent or its
excipients.
16. Patients who have previously undergone allergy immunotherapy for prevention of anaphylactic
reaction.
17. 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.
18. Currently enrolled in another investigational device or drug study, or less than 30 days since
ending another investigational device or drug study(s), or receiving other investigational
treatment(s).
19. 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 up to 16 weeks after the study drug administration (see
Section 4.2.2).
20. 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.
21. Evidence of a current or previous disease, medical condition (including chronic alcohol or drug
abuse 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 and would make the study participant unreliable to adhere to the protocol, comply with
all study visits/procedures or to complete the trial, compromise the safety of the patient or
compromise the quality of the data.
2. Patients with primary erythrodermic psoriasis vulgaris.
3. Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that
are restricted to psoriatic plaques.
4. Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP).
5. Immediate life-threatening flare of GPP or requiring intensive care treatment, according to the
investigator’s judgement. Life-threatening complications mainly include, but are not limited to,
cardiovascular/cytokine driven shock, pulmonary distress syndrome, or renal failure.
6. 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.
7. Treatment with:
a. Any restricted medication as specified in Table 4.2.2.1: 1, 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
8. Patients with dose escalation of their maintenance therapy with cyclosporine and/or methotrexate
and/or retinoids within the 2 weeks prior to receiving the first dose of BI 655130/ placebo.
9. The initiation of systemic agents such as cyclosporine and/or retinoids and/or methotrexate 2
weeks prior to receiving the first dose of BI 655130/ placebo.
10. Patients with congestive heart disease, as assessed by the investigator.
11. Active systemic infections (Fungal and bacterial disease) during the last 2 weeks prior to receiving
first drug administration, as assessed by the investigator.
12. 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.
13. Relevant chronic or acute infections including HIV or viral hepatitis. For patients screened while
having a flare (inclusion criteria 1b or 1c), if Visit 1 HIV or viral hepatitis results are not available
in time for randomization, these patients may receive randomized treatment as long as the
investigator has ruled out active disease based on available documented history (i.e. negative HIV
and viral hepatitis test results) within 3 months prior to Visit 2. A patient can be re-screened if the
patient was treated and is cured from acute infection.
14. Active or Latent TB:
QuantiFERON® TB test will be performed at screening. If the result is positive, the patient may
participate in the study if further work up (according to local practice/guidelines) establishes
conclusively that the patient has no evidence of active tuberculosis. Active TB patients must be
excluded. If presence of latent tuberculosis is established, then treatment should have been initiated
and maintained according to local country guidelines. For patients screened while having a flare
(inclusion criteria 1b or 1c), if the TB test results are not available in time for randomization, these
patients may receive randomized treatment (provided they meet all other inclusion/exclusion
criteria) as long as the investigator has ruled out active disease based on available documented
history (i.e. negative for active TB) within 3 months prior to Visit 2.
15. History of allergy/hypersensitivity to a systemically administered trial medication agent or its
excipients.
16. Patients who have previously undergone allergy immunotherapy for prevention of anaphylactic
reaction.
17. 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.
18. Currently enrolled in another investigational device or drug study, or less than 30 days since
ending another investigational device or drug study(s), or receiving other investigational
treatment(s).
19. 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 up to 16 weeks after the study drug administration (see
Section 4.2.2).
20. 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.
21. Evidence of a current or previous disease, medical condition (including chronic alcohol or drug
abuse 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 and would make the study participant unreliable to adhere to the protocol, comply with
all study visits/procedures or to complete the trial, compromise the safety of the patient or
compromise the quality of the data.
The Estimated Number of Participants
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Taiwan
10 participants
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Global
90 participants
