Recruiting NCT05789056
Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: QRX003, 4% Lotion.
- Who it may be relevant to
- Registry conditions: Netherton Syndrome. Basic parameters: from 14 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multicenter, Open Label Study of QRX003 Lotion in Subjects With Netherton Syndrome
Overview
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
Interventions
- Drug QRX003, 4% Lotion
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Primary outcome measures
- Proportion of subjects with 1-point reduction on IGA [Time frame: Up to week 16]
- Proportion of subjects with 2-point reduction on IGA [Time frame: Up to week 16]
- NS surface area change [Time frame: Up to week 16]
- WI-NRS score change [Time frame: Up to week 16]
- Assessment of subject satisfaction with treatment based on TSQM [Time frame: Up to week 16]
- Proportion of subjects requiring rescue therapy [Time frame: Up to week 16]
- Safety Assessment-AEs [Time frame: Up to week 16]
- Safety Assessment-LSRs [Time frame: Up to week 16]
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.
- Subject is on a stable treatment regimen including systemic therapy for NS prior to baseline that is expected to remain stable for the duration of the study
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 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 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 4 centers
- Site #1 — San Diego
- Site #4 — Indianapolis
- Site #5 — Quincy
- Site #2 — San Antonio
Identifiers
NCT: NCT05789056 · CL-QRX003-002