Spesolimab for Netherton Syndrome

No longer recruiting at 35 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if spesolimab can help individuals with Netherton syndrome (NS), a skin condition causing redness and scaling. Participants divide into two groups: one receives spesolimab, and the other receives a placebo (a look-alike with no medicine). After four months, all participants switch to spesolimab. Those with a confirmed diagnosis of NS and moderate skin redness might be suitable for this study. Participants will attend regular check-ups over three years to monitor symptoms and general health. As a Phase 2 trial, this research measures how well spesolimab works in an initial, smaller group, offering a chance to contribute to important findings.

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.

Is there any evidence suggesting that spesolimab is likely to be safe for humans?

Research has shown that spesolimab has been tested for its effects on skin diseases similar to Netherton syndrome. In some studies, patients experienced quick and lasting improvements in their skin, suggesting that the treatment can be effective and safe for some individuals.

However, limited information exists on its safety specifically for Netherton syndrome. Other sources indicate that more evidence is needed to fully confirm the safety of spesolimab for this condition. Since this trial is in later stages, earlier studies have demonstrated that it is somewhat safe for humans. This is a positive sign, as it indicates the treatment has already passed important safety tests.

Participants in the study will undergo regular check-ups to monitor their health and any side effects, helping to catch any issues early. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Netherton syndrome?

Researchers are excited about Spesolimab for Netherton Syndrome because it offers a novel approach compared to traditional treatments, which mainly focus on managing symptoms with emollients and anti-inflammatory drugs. Unlike these standard options, Spesolimab targets the interleukin-36 receptor, potentially addressing one of the underlying causes of inflammation in Netherton Syndrome. This unique mechanism of action may provide more effective relief and improve the quality of life for patients who struggle with this challenging skin condition.

What evidence suggests that spesolimab might be an effective treatment for Netherton syndrome?

Research shows that spesolimab, which participants in this trial may receive, effectively treats skin conditions similar to Netherton syndrome. In studies on generalized pustular psoriasis, patients who received spesolimab experienced far fewer flare-ups compared to those who received a placebo (a harmless pill with no active medicine). One study found an 84% reduction in flare-ups over 48 weeks for those treated with spesolimab. Another study demonstrated that spesolimab effectively relieved the signs and symptoms of skin flares. While these results are specific to psoriasis, they suggest that spesolimab might also help manage symptoms of Netherton syndrome. ^{4 5 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11298804/

Spesolimab for generalized pustular psoriasis: a review of two ... Infections at week 1 were reported in 6/35 (17%) patients receiving spesolimab and in 1/18 (6%) patients receiving placebo. These data ...

2. clinicaltrials.eu clinicaltrials.eu/trial/study-to-evaluate-the-effectiveness-of-spesolimab-in-treating-patients-with-netherton-syndrome/

Study to Evaluate the Effectiveness of Spesolimab in ... The purpose of this study is to evaluate how well spesolimab works in treating patients with Netherton syndrome. Participants in the study will receive either ...

3. io.nihr.ac.uk io.nihr.ac.uk/wp-content/uploads/2024/07/30965-TSID_11789-Spesolimab-for-Generalised-Pustular-Psoriasis-V1.0-JUL2024-NON-CONF.pdf

Health Technology Briefing July 2024 Newly published results showed spesolimab significantly improved signs and symptoms of flares in rare, life-threatening skin disease, generalized pustular ...

4. boehringer-ingelheim.com boehringer-ingelheim.com/human-health/boehringer-leo-pharma-partnership-commercialize-spevigo

Boehringer LEO Pharma partnership commercialize ... Study protocol of the global Effisayil 1 Phase II, multicentre, randomised, double-blind, placebo-controlled trial of spesolimab in patients ...

5. clinicaltrialsarena.com clinicaltrialsarena.com/news/spesolimab-trial-psoriasis-flare/

GPP patients suffer no flare-ups after four weeks of ... Data from the phase II trial shows an 84% reduction in generalised pustular psoriasis flare-ups over 48 weeks compared to placebo.

6. hra.nhs.uk hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/1368-0104-efficacy-and-safety-of-spesolimab-in-patients-with-netherton-syndrome/

1368-0104: Efficacy and safety of spesolimab in patients ... A placebo-controlled trial to evaluate the efficacy and safety of spesolimab in the treatment of patients with Netherton syndrome.

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39373152/

Spesolimab, the first-in-class anti-IL-36R antibody Spesolimab treatment of GPP flares resulted in rapid and sustained improvements in pustular and skin clearance, and clinically significant ...

8. eocco.com eocco.com/-/media/EOCCO/PDFs/Formulary/EOCCO-spesolimab-SC-Spevigo-Policy.pdf

spesolimab SC (Spevigo®) There is insufficient evidence to support the safety and efficacy of spesolimab SC (Spevigo) in this disease space. Requests for this indication are ...