Clinical trial NCT05856526: A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
Official title: EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Netherton Syndrome
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2023-05-12
Enrollment: 43
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 or 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 up to 3 years. During this time, they visit the study site up to 42 times. 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.


Eligibility criteria: 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

Primary outcomes:
measure: IASI response
timeFrame: At baseline and at Week 16.
Secondary outcomes:
measure: Key secondary endpoint: IGA response
timeFrame: at Week 16.
measure: IGA score of 0 or 1
timeFrame: up to Week 16.
measure: IASI response
timeFrame: At baseline and up to Week 12.
measure: IASI-E subscore response
timeFrame: At baseline and up to Week 16.
measure: IASI-S subscore response
timeFrame: At baseline and up to Week 16
measure: Percent change from baseline in IASI score
timeFrame: At baseline and up to Week 16.
measure: Absolute change from baseline in NRS pain
timeFrame: At baseline and up to Week 16.
measure: Absolute change from baseline in NRS itch
timeFrame: At baseline and up to Week 16.
measure: Absolute change from baseline in DLQI score
timeFrame: At baseline and up to Week 16.
measure: Absolute change from baseline in CDLQI score
timeFrame: At baseline and up to Week 16.
measure: The occurrence of treatment emergent adverse events including serious and/or opportunistic infections
timeFrame: up to 172 weeks.
Other outcomes:
Locations:
city: Rancho Santa Margarita
country: United States
facility: Mission Dermatology Center
state: California
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Norfolk
country: United States
facility: Virginia Clinical Research, Inc.
state: Virginia
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Sofia
country: Bulgaria
facility: ASMC-IPSMC-skin and Veneral Diseases
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Guangzhou
country: China
facility: Southern Medical University Dermatology Hospital
city: Hangzhou
country: China
facility: The Children's Hospital Zhejiang University School Of Medicine
city: Hangzhou
country: China
facility: The First Affiliated Hospital, Zhejiang University
city: Nanjing
country: China
facility: Dermatology Hospital, Chinese Academy of Medical Sciences
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shanghai
country: China
facility: Xinhua Hospital Affiliated to Shanghai Jiaotong University
city: Paris
country: France
facility: HOP Saint-Louis
city: Heidelberg
country: Germany
facility: Universitätsklinikum Heidelberg
city: Kiel
country: Germany
facility: Universitätsklinikum Schleswig-Holstein, Campus Kiel
city: München
country: Germany
facility: Klinikum der Universität München AÖR
city: Tel Aviv
country: Israel
facility: Sourasky Medical Center
city: Roma
country: Italy
facility: Istituto Dermopatico Dell'Immacolata - IDI - IRCCS
city: Torino
country: Italy
facility: AO Città della Salute e Scienza
city: Aichi, Nagoya
country: Japan
facility: Nagoya University Hospital
city: Chiba, Urayasu
country: Japan
facility: Juntendo University Urayasu Hospital
city: Okayama, Okayama
country: Japan
facility: Okayama University Hospital
city: Kuala Lumpur
country: Malaysia
facility: Hospital Tunku Azizah
city: Rotterdam
country: Netherlands
facility: Erasmus MC - Sophia Kinderziekenhuis
city: Lisboa
country: Portugal
facility: ULS de São José, E.P.E. - Hospital Sto. António Capuchos
city: Zürich
country: Switzerland
facility: University Children Hospital Zürich
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
Study officials:
References: