Clinical trial NCT05521438: Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
Official title: A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Netherton Syndrome
Lead sponsor: Quoin Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2022-08-30
Enrollment: 30
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
measure: Efficacy Endpoints-1-point IGA
timeFrame: Up to week 16
measure: Efficacy Endpoints-2-point IGA
timeFrame: Up to week 16
measure: Efficacy Endpoints-NS Sign
timeFrame: Up to week 16
measure: Efficacy Endpoints-BSA
timeFrame: Up to week 16
measure: Efficacy Endpoints-WI-NRS score
timeFrame: Up to week 16
measure: Efficacy Endpoints-TSQM
timeFrame: Up to week 16
measure: Efficacy Endpoints-Rescue Therapy
timeFrame: Up to week 16
measure: Safety Endpoints-AEs
timeFrame: Up to week 16
measure: Safety Endpoints-LSR
timeFrame: Up to week 16
Secondary outcomes:
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Site #1
state: California
city: Indianapolis
country: United States
facility: Site #4
state: Indiana
city: Quincy
country: United States
facility: Site #5
state: Massachusetts
city: Charleston
country: United States
facility: Site #3
state: South Carolina
city: San Antonio
country: United States
facility: Site #2
state: Texas
Study officials:
affiliation: Therapeutics, Inc.
name: Tony Andrasfay
role: STUDY_DIRECTOR
References: