Spesolimab for Netherton Syndrome

Palo Alto (17 mi)

Age: Any Age

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Boehringer Ingelheim

Prior Safety Data

Approved in 2 jurisdictions

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 and 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 about 1 year. During this time, they visit the study site 16 times. Where possible, 4 of 16 visits can be done at the participant's home instead of the study site. 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.

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 have been diagnosed with Netherton syndrome.

Exclusion Criteria

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

My liver tests are not severely abnormal.

Treatment Details

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.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: SpesolimabExperimental Treatment3 Interventions

Group II: PlaceboPlacebo Group4 Interventions

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

🇺🇸 Approved in United States as Spevigo for: