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

NCT05856526 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: 1368-01042022-501104-10-00U1111-1289-6825
• Study Type: interventional
• Target: 43
• Locations: 14 countries
• Sites: 28

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.

Conditions

Netherton Syndrome

Outcome Measures

Primary Outcomes (1)

• IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No)

  Proportion of participants with an Ichthyosis Area Severity Index (IASI) response at Week 16 is reported. IASI response was defined as a decrease of at least 50% absolute change in IASI score from baseline at Week 16. Proportions were rounded to 3 decimal places. IASI is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema and scaling. The score ranges from 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity. 95% confidence intervals (CI) are calculated using the method of Wilson.

  At baseline and at Week 16.

Secondary Outcomes (11)

• Key Secondary Endpoint: IGA Response, Defined as a Decrease of at Least 1-grade Absolute Change in IGA Score From Baseline at Week 16 (Yes/No)

  Baseline and at Week 16 after start of study treatment administration.

• IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No)

  At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.

• IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Weeks 4, 8, and 12 (Yes/No)

  At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration.

• IASI-E Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-E Subscore at Weeks 4, 8, 12, and 16 (Yes/No)

  At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.

• IASI-S Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-S Subscore From Baseline at Weeks 4, 8, 12, and 16 (Yes/No)

  At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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

Spesolimab

EXPERIMENTAL

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

Interventions

Spesolimab - solution for infusion DRUG

Solution for infusion

Also known as: BI 655130

Placebo; Spesolimab

Placebo matching to spesolimab - solution for infusion DRUG

Solution for infusion

Placebo; Spesolimab

Spesolimab - solution for injection DRUG

Solution for injection

Also known as: BI 655130

Placebo; Spesolimab

Placebo matching to spesolimab - solution for injection DRUG

Solution for injection

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (28)

Mission Dermatology Center

Rancho Santa Margarita, California, 92688, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06519, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Virginia Clinical Research, Inc.

Norfolk, Virginia, 23502, United States

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

ASMC-IPSMC-skin and Veneral Diseases

Sofia, 1407, Bulgaria

Location

Beijing Children's Hospital, Capital Medical University

Beijing, 100045, China

Location

Southern Medical University Dermatology Hospital

Guangzhou, 510091, China

Location

The Children's Hospital Zhejiang University School Of Medicine

Hangzhou, 310000, China

Location

The First Affiliated Hospital, Zhejiang University

Hangzhou, 310003, China

Location

Dermatology Hospital, Chinese Academy of Medical Sciences

Nanjing, 210000, China

Location

Shanghai Skin Disease Hospital

Shanghai, 200000, China

Location

Xinhua Hospital Affiliated to Shanghai Jiaotong University

Shanghai, 200092, China

Location

HOP Saint-Louis

Paris, 75010, France

Location

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

Universitätsklinikum Schleswig-Holstein, Campus Kiel

Kiel, 24105, Germany

Location

Klinikum der Universität München AÖR

München, 80337, Germany

Location

Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Istituto Dermopatico Dell'Immacolata - IDI - IRCCS

Roma, 00167, Italy

Location

Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino

Torino, 10126, Italy

Location

Nagoya University Hospital

Aichi, Nagoya, 466-8560, Japan

Location

Juntendo University Urayasu Hospital

Chiba, Urayasu, 279-0021, Japan

Location

Okayama University Hospital

Okayama, Okayama, 700-8558, Japan

Location

Hospital Tunku Azizah

Kuala Lumpur, 50300, Malaysia

Location

Erasmus MC Sophia Kinderziekenhuis

Rotterdam, 3015 GD, Netherlands

Location

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

Lisbon, 1169-050, Portugal

Location

University Children Hospital Zürich

Zurich, 8032, Switzerland

Location

Queen Elizabeth University Hospital

Glasgow, G51 4TF, United Kingdom

Location

Related Links

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MeSH Terms

Conditions

Netherton Syndrome

Interventions

spesolimab; Injections

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Boehringer Ingelheim, Call Center
• Organization: Boehringer Ingelheim

clintriage.rdg@boehringer-ingelheim.com

1-800-243-0127

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2023
• First Posted: May 12, 2023
• Study Start: June 12, 2023
• Primary Completion: January 13, 2025
• Study Completion: July 31, 2025
• Last Updated: March 12, 2026
• Results First Posted: March 12, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3); GB (1); BU (1); FR (1); AU (1); JP (3); IL (1); IT (2); MY (1); NL (1); PT (1); CH (1); US (4); CN (7)