Netherton Syndrome
Conditions
Brief summary
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
Interventions
DRUGQRX003-2% Lotion
QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
DRUGQRX003-4% Lotion QAM
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
DRUGVehicle
Vehicle Lotion
DRUGQRX003-4% Lotion BID
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Sponsors
Quoin Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Double-blind
Eligibility
Sex/Gender
ALL
Age
14 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject is a male or non-pregnant female at least 14 years of age. * Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. * Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation. * Subject has NS lesions in the Treatment Area (i.e., arms or lower legs). * Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
Exclusion criteria
* Subject is pregnant, lactating, or is planning to become pregnant during the study. * Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy. * Subject has Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females * Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area. * Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled. * Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline. * Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis. * Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline. * Subject has used systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline. * Subject has used systemic biologic therapy for NS. * Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline. * Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline. * Subject is currently enrolled in an investigational drug, biologic, or device study. * Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2/Baseline.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy Endpoints-1-point IGA | Up to week 16 | 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. |
| Efficacy Endpoints-2-point IGA | Up to week 16 | Proportion of subjects with 2-point reduction in IGA from Baseline. |
| Efficacy Endpoints-NS Sign | Up to week 16 | Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale. |
| Efficacy Endpoints-BSA | Up to week 16 | Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area |
| Efficacy Endpoints-WI-NRS score | Up to week 16 | 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". |
| Efficacy Endpoints-TSQM | Up to week 16 | 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. |
| Efficacy Endpoints-Rescue Therapy | Up to week 16 | Proportion of subjects requiring rescue therapy |
| Safety Endpoints-AEs | Up to week 16 | Any local and systemic AEs (Adverse Events)/serious AEs |
| Safety Endpoints-LSR | Up to week 16 | Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit. |
Countries
United States
Contacts
STUDY_DIRECTORTony Andrasfay
Therapeutics, Inc.
Outcome results
None listed