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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05856526




Registration number
NCT05856526
Ethics application status
Date submitted
26/04/2023
Date registered
12/05/2023

Titles & IDs
Public title
A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
Scientific title
EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome
Secondary ID [1] 0 0
2022-501104-10-00
Secondary ID [2] 0 0
1368-0104
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Netherton Syndrome 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Spesolimab - solution for infusion
Treatment: Drugs - Placebo matching to spesolimab - solution for infusion
Treatment: Drugs - Spesolimab - solution for injection
Treatment: Drugs - Placebo matching to spesolimab - solution for injection

Placebo comparator: Placebo -

Experimental: Spesolimab -


Treatment: Drugs: Spesolimab - solution for infusion
Solution for infusion

Treatment: Drugs: Placebo matching to spesolimab - solution for infusion
Solution for infusion

Treatment: Drugs: Spesolimab - solution for injection
Solution for injection

Treatment: Drugs: Placebo matching to spesolimab - solution for injection
Solution for injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
IASI response
Timepoint [1] 0 0
At baseline and at Week 16.
Secondary outcome [1] 0 0
Key secondary endpoint: IGA response
Timepoint [1] 0 0
at Week 16.
Secondary outcome [2] 0 0
IGA response
Timepoint [2] 0 0
up to Week 12.
Secondary outcome [3] 0 0
IASI response
Timepoint [3] 0 0
At baseline and up to Week 12.
Secondary outcome [4] 0 0
IASI-E subscore response
Timepoint [4] 0 0
At baseline and up to Week 16.
Secondary outcome [5] 0 0
IASI-S subscore response
Timepoint [5] 0 0
At baseline and up to Week 16
Secondary outcome [6] 0 0
Percent change from baseline in IASI score
Timepoint [6] 0 0
At baseline and up to Week 16.
Secondary outcome [7] 0 0
Absolute change from baseline in NRS pain
Timepoint [7] 0 0
At baseline and up to Week 16.
Secondary outcome [8] 0 0
Absolute change from baseline in NRS itch
Timepoint [8] 0 0
At baseline and up to Week 16.
Secondary outcome [9] 0 0
Absolute change from baseline in DLQI/CDLQI score
Timepoint [9] 0 0
At baseline and up to Week 16.
Secondary outcome [10] 0 0
The occurrence of treatment emergent adverse events including serious and/or opportunistic infections
Timepoint [10] 0 0
up to 172 weeks.

Eligibility
Key inclusion criteria
* Male or female patients, aged 12 years and older (weight minimum is 35kg).
* Confirmed diagnosis of Netherton syndrome (NS) (causative SPINK5 mutations) at baseline (Visit 2).
* At least moderate severity of erythema at baseline (visit 2) (Ichthyosis Area Severity Index (IASI) score = 16 and IASI-Erythema (E) score =8) and = 3 on Investigator Global Assessment (IGA) score.
* Signed and dated written informed consent and assent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission in the trial
* 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 is provided in the clinical trial protocol (CTP) as well as in the patient, parent(s) (or patient's legal guardian) information.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patients who have used topical corticosteroids (medium to high, US class I-V), topical retinoids, topical calcineurin inhibitors or keratolytics within 1 week prior to randomisation
* Patients who have used emollient on the area to be biopsied in the previous 24 hours
* Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
* Patients who have used systemic antibiotics within 2 weeks prior to randomisation
* Patients who have received live vaccines within 4 weeks prior to randomisation
* Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
* 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
* Patients who have any prior exposure to BI 655130 or another interleukin 36 receptor (IL-36R) inhibitor biologics
* 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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
3/01/2028
Actual
Sample size
Target
39
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Virginia
Country [4] 0 0
Belgium
State/province [4] 0 0
Leuven
Country [5] 0 0
Bulgaria
State/province [5] 0 0
Sofia
Country [6] 0 0
China
State/province [6] 0 0
Beijing
Country [7] 0 0
China
State/province [7] 0 0
Guangzhou
Country [8] 0 0
China
State/province [8] 0 0
Hangzhou
Country [9] 0 0
China
State/province [9] 0 0
Nanjing
Country [10] 0 0
China
State/province [10] 0 0
Shanghai
Country [11] 0 0
Finland
State/province [11] 0 0
Tampere
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
Germany
State/province [13] 0 0
Erlangen
Country [14] 0 0
Germany
State/province [14] 0 0
Heidelberg
Country [15] 0 0
Germany
State/province [15] 0 0
Kiel
Country [16] 0 0
Germany
State/province [16] 0 0
München
Country [17] 0 0
Israel
State/province [17] 0 0
Tel Aviv
Country [18] 0 0
Italy
State/province [18] 0 0
Roma
Country [19] 0 0
Italy
State/province [19] 0 0
Torino
Country [20] 0 0
Japan
State/province [20] 0 0
Aichi, Nagoya
Country [21] 0 0
Japan
State/province [21] 0 0
Chiba, Urayasu
Country [22] 0 0
Japan
State/province [22] 0 0
Okayama, Okayama
Country [23] 0 0
Malaysia
State/province [23] 0 0
Kuala Lumpur
Country [24] 0 0
Malaysia
State/province [24] 0 0
Selangor Darul Ehsan
Country [25] 0 0
Netherlands
State/province [25] 0 0
Rotterdam
Country [26] 0 0
Portugal
State/province [26] 0 0
Lisboa
Country [27] 0 0
Switzerland
State/province [27] 0 0
Zürich
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Glasgow

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

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
Boehringer Ingelheim
Address 0 0
Country 0 0
Phone 0 0
1-800-243-0127
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Once the criteria in section "Time Frame" are fulfilled, 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 in the website.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.mystudywindow.com/msw/datasharing


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05856526