A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 5
- 主要终点
- Efficacy Endpoints-2-point IGA
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Double-blind
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
QRX003-4% BID
Subjects will apply test article twice daily BID for 12 weeks
干预措施: QRX003-4% Lotion BID (Drug)
Vehicle Lotion
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
干预措施: Vehicle (Drug)
QRX003-2% QAM
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
干预措施: QRX003-2% Lotion (Drug)
QRX003-4% QAM
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
干预措施: QRX003-4% Lotion QAM (Drug)
结局指标
主要结局
Efficacy Endpoints-2-point IGA
时间窗: Up to week 16
Proportion of subjects with 2-point reduction in IGA from Baseline.
Efficacy Endpoints-1-point IGA
时间窗: 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.
Safety Endpoints-LSR
时间窗: 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.
Efficacy Endpoints-WI-NRS score
时间窗: 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".
Safety Endpoints-AEs
时间窗: Up to week 16
Any local and systemic AEs (Adverse Events)/serious AEs
Efficacy Endpoints-NS Sign
时间窗: 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-TSQM
时间窗: 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-BSA
时间窗: Up to week 16
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Efficacy Endpoints-Rescue Therapy
时间窗: Up to week 16
Proportion of subjects requiring rescue therapy
次要结局
未报告次要终点