The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).
Study Type
OBSERVATIONAL
Enrollment
12
Mission Dermatology Center
Rancho Santa Margarita, California, United States
Northwestern University
Chicago, Illinois, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Istituto Dermopatico Dell'Immacolata - IDI - IRCCS
Roma, Italy
Severity of Netherton Syndrome (NS) assessed by the Ichthyosis Area Severity Index (IASI)
For patients participating in Part 2. IASI is a composite score that evaluates severity of erythema (subscale IASI-E) and scaling (subscale IASI-S) in different body regions as a function of their respective body surface areas. Severity of erythema and scaling is rated on a 5-point Likert scale of 0-4 in each of 4 body regions: head and neck (including scalp), arms (including palms), legs (including soles) and trunk, prorated based on body surface area in these body regions and the percentage of involvement in each of these body regions. The total IASI score ranges between 0-48 (i.e., sum of a maximum score of 24 for erythema and maximum score of 24 for scaling). Higher score denotes worse clinical severity.
Time frame: up to 1 year
Severity Netherton Syndrome (NS) assessed by the Investigator Global Assessment (IGA)
For patients participating in Part 2. IGA for NS will assess the global severity of erythema and scaling in NS using 5-point Likert scale ranging from 0=clear, to 4=severe.
Time frame: up to 1 year
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