Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome
ConditionNetherton Syndrome
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age14+
SponsorQuoin Pharmaceuticals
About this trial
To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
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 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.
Trial design
Design model
Single group
Treatments tested in this trial
QRX003, 4% Lotion
DrugQRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: QRX003, 4% QAMExperimental treatment 1 intervention
Group B: QRX003, 4% BIDExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Proportion of subjects with 1-point reduction on 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 162
Proportion of subjects with 2-point reduction on IGA
Proportion of subjects with 2-point reduction in IGA from Baseline.
Time frame
Up to week 163
NS surface area change
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Time frame
Up to week 164
WI-NRS score change
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score ≥4. 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 16Sponsors and contacts
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Source ClinicalTrials.gov