BI 1368-0121
Research type
Research Study
Full title
Non-interventional study of patients with Netherton Syndrome to characterize natural history of the disease.
IRAS ID
340841
Contact name
Jemima Mellerio
Contact email
Sponsor organisation
Boehringer Ingleheim Pharmaceuticals, IC.
Clinicaltrials.gov Identifier
EUPAS103733, EU PAS register number
Duration of Study in the UK
2 years, 1 months, 0 days
Research summary
This study will collect real-world information to describe the natural history of Netherton Syndrome (NS). Data will be collected on demographics, severity of NS, NS clinical characteristics, NS flares, NS treatment patterns and quality of life, in a “real-world” setting. “Real-world” setting means that the data comes from patients who are being treated for NS in a normal medical practice situation.
At present, there is no approved therapy for NS. It is hoped that the information to be collected will give valuable information on the NS disease course and of the current treatment practices, which will help doctors to better understand and treat NS in the future.
This is a “non-interventional study” which means it is an observational study where no study medication is provided, or tests involved. Treatment and medical care for participants will not change because of participating in this study. Participants’ regular doctors and other healthcare providers will continue to make all decisions regarding their treatment and care.
Only patients diagnosed with Netherton Syndrome (NS) will be eligible to participate. It is estimated that 100 people of all ages will participate in real-world clinical practice settings worldwide.This study consists of 2 parts. Patients may choose to take part in part I only or in both parts I and II. Participation in this study will last approximately 1 year if patients agree to participate in both parts I and II. For part I, the data will be obtained from historical medical records. Data for part II will be collected from participants’ current medical charts. In addition, there may be visits for part II that are outside routine clinic visit schedule for clinician assessments and surveys/questionnaires for patients to complete. The surveys/questionnaires will ask participants about symptoms, how they are feeling/functioning and their quality of life with NS.
REC name
London - Hampstead Research Ethics Committee
REC reference
24/PR/0884
Date of REC Opinion
5 Sep 2024
REC opinion
Further Information Favourable Opinion