Spesolimab for Netherton Syndrome

Recruiting at 33 trial locations

Overseen By Boehringer Ingelheim

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Boehringer Ingelheim

Must not be taking: Corticosteroids, Retinoids, Immunosuppressants, others

Disqualifiers: Hepatic disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 58

Duration: 16 weeks

43 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before joining. You must not have used specific topical treatments within 1 week, systemic treatments like retinoids or corticosteroids within 4 weeks, or systemic antibiotics within 2 weeks before starting the trial.

How is the drug Spesolimab different from other treatments for Netherton Syndrome?

Spesolimab is unique because it targets the immune system by blocking a specific protein involved in inflammation, which is different from other treatments that mainly provide supportive care. This approach is novel for Netherton Syndrome, as there are no standard effective therapies available for this condition. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for individuals aged 12 and older with Netherton syndrome, a skin condition. Participants must have moderate to severe symptoms, be willing to use effective birth control if applicable, and weigh at least 35kg. They cannot have used certain medications or treatments recently or received live vaccines within the last month.

Inclusion Criteria

My skin condition is moderately severe with a high redness and overall severity score.

I am 12 years or older and weigh at least 35kg.

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

I haven't taken any experimental drugs or biologics in the last month.

I have never used BI 655130 or any IL-36R inhibitor treatments.

I have not received any live vaccines in the last 4 weeks.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive spesolimab or placebo injections. Initially, injections are given intravenously, followed by monthly subcutaneous injections.

16 weeks

Up to 16 visits (in-person)

Crossover

Participants in the placebo group switch to spesolimab treatment after 4 months.

4 months

Long-term Follow-up

Participants continue to receive treatment and are monitored for safety and effectiveness. Regular health checks and symptom assessments are conducted.

Up to 3 years

Up to 42 visits (in-person)

Treatment Details

Interventions

• Placebo matching to Spesolimab
• Spesolimab

Trial Overview The study tests spesolimab's effectiveness in treating Netherton syndrome compared to a placebo. Initially given as an infusion, then monthly injections under the skin, participants are randomly assigned to either treatment with a higher chance of receiving spesolimab.

Participant Groups

Group I: Spesolimab Experimental Treatment 3 Interventions

Group II: Placebo Placebo Group 4 Interventions

Spesolimab is already approved in United States, European Union for the following indications:

Approved in United States as Spevigo for:

• Generalized pustular psoriasis (GPP) in adults and pediatric patients 12 years of age and older and weighing at least 40 kg

Approved in European Union as Spevigo for:

• Generalized pustular psoriasis (GPP) flares in adults

Who Is Running the Clinical Trial?

Boehringer Ingelheim

Lead Sponsor

Trials

2,566

Recruited

16,150,000+

Findings from Research

A case of Netherton syndrome (NS), a rare genetic disorder caused by mutations in the SPINK5 gene, was confirmed through whole exome sequencing, identifying two new mutations from the patient's parents.

The patient showed significant clinical improvement after treatment with dupilumab, a medication typically used for other conditions, with no reported adverse effects, suggesting potential for dupilumab as a safe therapeutic option for NS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful treatment of Netherton syndrome with dupilumab: A case report and review of the literature. Wang, J., Yu, L., Zhang, S., et al.[2022]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Treatment Experiences with Intravenous Immunoglobulins, Ixekizumab, Dupilumab, and Anakinra in Netherton Syndrome: A Case Series. [2023]

2. France pubmed.ncbi.nlm.nih.gov

Establishment of a mouse model of Netherton syndrome based on CRISPR/Cas9 technology [2022]

3. England pubmed.ncbi.nlm.nih.gov

Successful treatment of Netherton syndrome with dupilumab: A case report and review of the literature. [2022]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Combined novel homozygous variants in both SGPL1 and STAT1 presenting with severe combined immune deficiency: case report and literature review. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Netherton Syndrome in a Mother and Her Two Children. [2023]