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

NCT ID: NCT05856526

Last Updated: 2025-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-12

Study Completion Date

2025-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Netherton Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Group Type PLACEBO_COMPARATOR

Spesolimab - solution for infusion

Intervention Type DRUG

Solution for infusion

Placebo matching to spesolimab - solution for infusion

Intervention Type DRUG

Solution for infusion

Spesolimab - solution for injection

Intervention Type DRUG

Solution for injection

Placebo matching to spesolimab - solution for injection

Intervention Type DRUG

Solution for injection

Spesolimab

Group Type EXPERIMENTAL

Spesolimab - solution for infusion

Intervention Type DRUG

Solution for infusion

Placebo matching to spesolimab - solution for infusion

Intervention Type DRUG

Solution for infusion

Spesolimab - solution for injection

Intervention Type DRUG

Solution for injection

Interventions

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Spesolimab - solution for infusion

Solution for infusion

Intervention Type DRUG

Placebo matching to spesolimab - solution for infusion

Solution for infusion

Intervention Type DRUG

Spesolimab - solution for injection

Solution for injection

Intervention Type DRUG

Placebo matching to spesolimab - solution for injection

Solution for injection

Intervention Type DRUG

Other Intervention Names

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BI 655130 BI 655130

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boehringer Ingelheim

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mission Dermatology Center

Rancho Santa Margarita, California, United States

Site Status

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Virginia Clinical Research, Inc.

Norfolk, Virginia, United States

Site Status

Westmead Hospital

Westmead, New South Wales, Australia

Site Status

ASMC-IPSMC-skin and Veneral Diseases

Sofia, , Bulgaria

Site Status

Beijing Children's Hospital, Capital Medical University

Beijing, , China

Site Status

Southern Medical University Dermatology Hospital

Guangzhou, , China

Site Status

The Children's Hospital Zhejiang University School Of Medicine

Hangzhou, , China

Site Status

The First Affiliated Hospital, Zhejiang University

Hangzhou, , China

Site Status

Dermatology Hospital, Chinese Academy of Medical Sciences

Nanjing, , China

Site Status

Shanghai Skin Disease Hospital

Shanghai, , China

Site Status

Xinhua Hospital Affiliated to Shanghai Jiaotong University

Shanghai, , China

Site Status

HOP Saint-Louis

Paris, , France

Site Status

Universitätsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Universitätsklinikum Schleswig-Holstein, Campus Kiel

Kiel, , Germany

Site Status

Klinikum der Universität München AÖR

München, , Germany

Site Status

Sourasky Medical Center

Tel Aviv, , Israel

Site Status

Istituto Dermopatico Dell'Immacolata - IDI - IRCCS

Roma, , Italy

Site Status

AO Città della Salute e Scienza

Torino, , Italy

Site Status

Nagoya University Hospital

Aichi, Nagoya, , Japan

Site Status

Juntendo University Urayasu Hospital

Chiba, Urayasu, , Japan

Site Status

Okayama University Hospital

Okayama, Okayama, , Japan

Site Status

Hospital Tunku Azizah

Kuala Lumpur, , Malaysia

Site Status

Erasmus MC - Sophia Kinderziekenhuis

Rotterdam, , Netherlands

Site Status

ULS de São José, E.P.E. - Hospital Sto. António Capuchos

Lisbon, , Portugal

Site Status

University Children Hospital Zürich

Zurich, , Switzerland

Site Status

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status

Countries

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

Related Links

Other Identifiers

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2022-501104-10-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

U1111-1289-6825

Identifier Type: REGISTRY

Identifier Source: secondary_id

1368-0104

Identifier Type: -

Identifier Source: org_study_id

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