This study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% QAM or 4% BID in subjects with Netherton Syndrome (NS) in comparison to vehicle
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
30
QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Vehicle Lotion
Site #1
San Diego, California, United States
Site #4
Indianapolis, Indiana, United States
Site #5
Quincy, Massachusetts, United States
Site #3
Charleston, South Carolina, United States
Site #2
Efficacy Endpoints-1-point IGA
Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Time frame: Up to week 16
Efficacy Endpoints-2-point IGA
Proportion of subjects with 2-point reduction in IGA from Baseline.
Time frame: Up to week 16
Efficacy Endpoints-NS Sign
Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.
Time frame: Up to week 16
Efficacy Endpoints-BSA
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Time frame: Up to week 16
Efficacy Endpoints-WI-NRS score
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Time frame: Up to week 16
Efficacy Endpoints-TSQM
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Time frame: Up to week 16
Efficacy Endpoints-Rescue Therapy
Proportion of subjects requiring rescue therapy
Time frame: Up to week 16
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QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
San Antonio, Texas, United States
Safety Endpoints-AEs
Any local and systemic AEs (Adverse Events)/serious AEs
Time frame: Up to week 16
Safety Endpoints-LSR
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
Time frame: Up to week 16