Natural History of Netherton Syndrome
- Conditions
- Netherton Syndrome
- Registration Number
- NCT05902663
- Lead Sponsor
- Boehringer Ingelheim
- Brief Summary
The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 12
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Severity of Netherton Syndrome (NS) assessed by the Ichthyosis Area Severity Index (IASI) up to 1 year 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.
- Secondary Outcome Measures
Name Time Method Severity Netherton Syndrome (NS) assessed by the Investigator Global Assessment (IGA) up to 1 year 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.
Trial Locations
- Locations (4)
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