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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
43
Solution for infusion
Solution for infusion
Solution for injection
Solution for injection
Mission Dermatology Center
Rancho Santa Margarita, California, United States
Yale University School of Medicine
New Haven, Connecticut, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Virginia Clinical Research, Inc.
Norfolk, Virginia, United States
Westmead Hospital
Westmead, New South Wales, Australia
IASI response
Ichthyosis Area Severity Index (IASI) response is defined as a decrease of at least 50 % absolute change in IASI score from baseline at Week 16.
Time frame: At baseline and at Week 16.
Key secondary endpoint: IGA response
Investigator Global Assessment (IGA)
Time frame: at Week 16.
IGA score of 0 or 1
Investigator Global Assessment (IGA)
Time frame: up to Week 16.
IASI response
Ichthyosis Area Severity Index (IASI)
Time frame: At baseline and up to Week 12.
IASI-E subscore response
Ichthyosis Area Severity Index - Erythema (IASI-E)
Time frame: At baseline and up to Week 16.
IASI-S subscore response
Ichthyosis Area Severity Index - Scaling (IASI-S)
Time frame: At baseline and up to Week 16
Percent change from baseline in IASI score
Ichthyosis Area Severity Index (IASI)
Time frame: At baseline and up to Week 16.
Absolute change from baseline in NRS pain
The Numeric Pain Rating Scale (NRS)
Time frame: At baseline and up to Week 16.
Absolute change from baseline in NRS itch
The Numeric Rating Scale (NRS)
Time frame: At baseline and up to Week 16.
Absolute change from baseline in DLQI score
Dermatology Life Quality Index (DLQI)
Time frame: At baseline and up to Week 16.
Absolute change from baseline in CDLQI score
Children Dermatology Life Quality Index (CDLQI)
Time frame: At baseline and up to Week 16.
The occurrence of treatment emergent adverse events including serious and/or opportunistic infections
Time frame: up to 172 weeks.
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ASMC-IPSMC-skin and Veneral Diseases
Sofia, Bulgaria
Beijing Children's Hospital, Capital Medical University
Beijing, China
Southern Medical University Dermatology Hospital
Guangzhou, China
The Children's Hospital Zhejiang University School Of Medicine
Hangzhou, China
The First Affiliated Hospital, Zhejiang University
Hangzhou, China
...and 18 more locations