A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

Netherton Syndrome Clinical Trial

Official title:

EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05856526
• Other study ID #: 1368-0104
• Secondary ID: 2022-501104-10-0
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: May 15, 2023
• Est. completion date: January 3, 2028

Study information

• Verified date: April 2024
• Source: Boehringer Ingelheim
• Contact: Boehringer Ingelheim
• Phone: 1-800-243-0127
• Email: clintriage.rdg[@]boehringer-ingelheim.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years and older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS. Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month. After 4 months, participants in the placebo group switch to spesolimab treatment. Participants are in the study for about 1 year. During this time, they visit the study site 16 times. Where possible, 4 of 16 visits can be done at the participant's home instead of the study site. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 39
• Est. completion date: January 3, 2028
• Est. primary completion date: December 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

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

Related Conditions & MeSH terms

• Netherton Syndrome
• Syndrome

Intervention

Drug:
• Spesolimab - solution for infusion
Solution for infusion
• Placebo matching to spesolimab - solution for infusion
Solution for infusion
• Spesolimab - solution for injection
Solution for injection
• Placebo matching to spesolimab - solution for injection
Solution for injection

Locations

Country	Name	City	State
Australia	Westmead Hospital	Westmead	New South Wales
Austria	Medical University of Salzburg (SALK) Paracelsus Medical Privatuniversity Salzburg (PMU)	Salzburg
Belgium	UZ Leuven	Leuven
Bulgaria	ASMC-IPSMC-skin and Veneral Diseases	Sofia
China	Beijing Children's Hospital, Capital Medical University	Beijing
China	Southern Medical University Dermatology Hospital	Guangzhou
China	The Children's Hospital Zhejiang University School Of Medicine	Hangzhou
China	The First Affiliated Hospital, Zhejiang University	Hangzhou
China	Dermatology Hospital, Chinese Academy of Medical Sciences	Nanjing
China	Shanghai Skin Disease Hospital	Shanghai
China	Xinhua Hospital Affiliated to Shanghai Jiaotong University	Shanghai
France	HOP Saint-Louis	Paris
Germany	Universitätsklinikum Erlangen	Erlangen
Germany	Universitätsklinikum Heidelberg	Heidelberg
Germany	Universitätsklinikum Schleswig-Holstein, Campus Kiel	Kiel
Germany	Klinikum der Universität München AÖR	München
Israel	Sourasky Medical Center	Tel Aviv
Italy	Istituto Dermopatico Dell'Immacolata - IDI - IRCCS	Roma
Italy	AO Città della Salute e Scienza	Torino
Japan	Nagoya University Hospital	Aichi, Nagoya
Japan	Juntendo University Urayasu Hospital	Chiba, Urayasu
Japan	Okayama University Hospital	Okayama, Okayama
Malaysia	Hospital Tunku Azizah	Kuala Lumpur
Malaysia	Sunway Medical Centre	Selangor Darul Ehsan
Netherlands	Erasmus MC - Sophia Kinderziekenhuis	Rotterdam
Portugal	ULS de São José, E.P.E. - Hospital Sto. António Capuchos	Lisboa
Switzerland	University Children Hospital Zürich	Zürich
United Kingdom	Queen Elizabeth University Hospital	Glasgow
United States	Yale University School of Medicine	New Haven	Connecticut
United States	Virginia Clinical Research, Inc.	Norfolk	Virginia
United States	Mission Dermatology Center	Rancho Santa Margarita	California

Sponsors (1)

Lead Sponsor	Collaborator
Boehringer Ingelheim

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Belgium,  Bulgaria,  China,  France,  Germany,  Israel,  Italy,  Japan,  Malaysia,  Netherlands,  Portugal,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	IASI response	Ichthyosis Area Severity Index (IASI) response is defined as a decrease of at least 50 % absolute change in IASI score from baseline at Week 16.	At baseline and at Week 16.
Secondary	Key secondary endpoint: IGA response	Investigator Global Assessment (IGA)	at Week 16.
Secondary	IGA response	Investigator Global Assessment (IGA)	up to Week 12.
Secondary	IASI response	Ichthyosis Area Severity Index (IASI)	At baseline and up to Week 12.
Secondary	IASI-E subscore response	Ichthyosis Area Severity Index - Erythema (IASI-E)	At baseline and up to Week 16.
Secondary	IASI-S subscore response	Ichthyosis Area Severity Index - Scaling (IASI-S)	At baseline and up to Week 16
Secondary	Percent change from baseline in IASI score	Ichthyosis Area Severity Index (IASI)	At baseline and up to Week 16.
Secondary	Absolute change from baseline in NRS pain	The Numeric Pain Rating Scale (NRS)	At baseline and up to Week 16.
Secondary	Absolute change from baseline in NRS itch	The Numeric Rating Scale (NRS)	At baseline and up to Week 16.
Secondary	Absolute change from baseline in DLQI/CDLQI score	Dermatology Life Quality Index (DLQI)/Children Dermatology Life Quality Index (CDLQI)	At baseline and up to Week 16.
Secondary	The occurrence of treatment emergent adverse events including serious and/or opportunistic infections		up to 172 weeks.

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