A study to test whether spesolimab helps people with skin disease called Netherton syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A., Boehringer Ingelheim RCV GmbH & Co. KG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

6 Jun 2023 → 1 Aug 2025

Decision date (initial)

2023-05-02

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2022-501104-10-00

WHO UTN

U1111-1289-6825

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

Evaluate the treatment response of spesolimab

Secondary objectives 4

1. Reduction in other skin severity assessments
2. Improvement in quality-of-life assessments
3. Improvement in sleep, itch and scalp hair assessments
4. Describe safety descriptively, with incidence of AEs

Conditions and MedDRA coding

Netherton syndrome (NS)

Study design 6 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-002475-PIP03-22

Plan to share IPD

Yes

IPD plan description

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 on the website. Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program. Access Criteria: 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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Male or female patients, aged 12 years and older (weight minimum is 35kg).
2. Confirmed diagnosis of NS (SPINK5 causative mutations) at baseline
3. At least moderate severity of erythema at baseline (IASI score ≥ 16 and IASI-E score ≥8) and ≥ 3 on IGA score
4. Signed and dated written informed consent and assent in accordance with ICH-GCP and local legislation prior to admission in the trial
5. 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 information.

Exclusion criteria 11

1. 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
2. Patients who have used emollient on the area to be biopsied in the previous 24 hours
3. Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
4. Patients who have used systemic antibiotics within 2 weeks prior to randomisation
5. Patients who have received live vaccines within 4 weeks prior to randomisation
6. Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
7. Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in AST or ALT or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin.
8. Patients who have any prior exposure to BI 655130 or another IL-36R inhibitor biologics.
9. Increased risk of infectious complications (e.g., recent pyogenic infection, any congenital or acquired immunodeficiency (e.g., HIV), past organ or stem cell transplantation), as assessed by the investigator.
10. Relevant chronic or acute infections including active tuberculosis, human immunodeficiency virus (HIV) infection or viral hepatitis at the time of randomisation. A patient can be re-screened if the patient was treated and is cured from the acute infection.
11. Further criteria apply

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. IASI response, defined as a decrease of at least 50 % absolute change in IASI score from baseline (Yes/No).

Secondary endpoints 11

1. Key secondary endpoint: IGA response, defined as a decrease of at least 1-grade absolute change in IGA score from baseline
2. IGA score of 0 or 1
3. IASI response
4. IASI-E subscore response
5. IASI-S subscore response
6. Percent change from baseline in IASI score
7. Absolute change from baseline in NRS pain
8. Absolute change from baseline in NRS itch
9. Absolute change from baseline in DLQI score
10. Absolute change from baseline in CDLQI score
11. The occurrence of treatment emergent adverse events including serious and/or opportunistic infections

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50

City

Sant Cugat Del Valles

Postcode

08174

Country

Spain

Boehringer Ingelheim RCV GmbH & Co. KG

Sponsor organisation

Boehringer Ingelheim RCV GmbH & Co. KG

Address

Dr.-Boehringer-Gasse 5-11, Meidling Meidling

City

Vienna

Postcode

1121

Country

Austria

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

11 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 251 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

16 submissions — initial application plus substantial / non-substantial modifications