A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2 Dose-Response Study to Determine Safety and Effectiveness of Two Concentrations of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
概览
- 阶段
- 2 期
- 干预措施
- NFX-179 gel
- 疾病 / 适应症
- Neurofibromatosis 1
- 发起方
- NFlection Therapeutics, Inc.
- 入组人数
- 199
- 试验地点
- 23
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
- 状态
- 已完成
- 最后更新
- 2个月前
概览
简要总结
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF. At Visit 1 (Screening visit), the investigator will identify 10 Target cNFs that fulfil the enrollment criteria. The Target cNFs must be located on the participant's face, anterior trunk, or upper extremities. Two Target cNFs must be on the face and 8 must be on the anterior trunk or upper extremities. The study medication will be applied topically once daily to the Target cNFs for 182 days (26 weeks). During the duration of the study participants will be evaluated for safety and efficacy.
研究者
入排标准
入选标准
- •Participant is at least 18 years of age
- •Participant must provide written informed consent prior to any study procedures
- •Participant must have a clinical diagnosis of NF1
- •Participant has 10 clinically diagnosed Target cNFs with preferably 2 Target cNFs located on the face and 8 Target cNFs located on the anterior trunk or upper extremities. Alternatively, at least 1 Target cNF is located on the face, in which case 9 Target cNFs must be located on the anterior trunk or upper extremities. Each Target cNF must meet the following criteria:
- •Has, in the investigator's opinion, a clinically typical appearance
- •Is not within 1 centimeter (cm) of the orbital rim
- •Is not covered with hair that might, in the investigator's opinion, interfere with obtaining photographs or impair evaluation of the cNF
- •Has a Physician's Tumor Assessment grade ≥2
- •Is dome shaped
- •Is not pedunculated
排除标准
- •Participant has used any of the following topical therapies within the specified period prior to Visit 1 (screening visit) on or in proximity to any Target cNF that, in the investigator's opinion, impairs evaluation of any the cNFs or which exposes the participant to an unacceptable risk by study participation:
- •Corticosteroids; 30 days
- •Prescription retinoids (for example, tazarotene, tretinoin, adapalene); 30 days
- •\> 5% of an alpha-hydroxy acid (for example, glycolic acid, lactic acid); 30 days
- •Fluorouracil; 30 days
- •Imiquimod; 30 days
- •LASER, light (for example, intense pulsed light \[IPL\], photo-dynamic therapy \[PDT\]) or other energy-based therapy; 180 days
- •Mitogen-activated protein kinase (MEK) inhibitor or V-Raf Murine Sarcoma Viral Oncogene Homolog B (BRAF) inhibitor; ever.
- •The participant has used any of the following systemic medications therapies within the specified period prior to Visit 1 (screening visit):
- •Retinoids (for example, etretinate, isotretinoin); 90 days
研究组 & 干预措施
NFX-179 Topical Gel 0.5%
NFX-179 topical gel 0.5% will be applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days.
干预措施: NFX-179 gel
NFX-179 Topical Gel 1.5%
NFX-179 topical gel 1.5% will be applied once daily to the target cNFs for 182 days.
干预措施: NFX-179 gel
NFX-179 Topical Gel Vehicle
NFX-179 topical gel vehicle will be applied once daily to the target cNFs for 182 days.
干预措施: Vehicle gel
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: Baseline (Day 1) up to Day 211
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an event that occurred or worsened on or after the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Volume Above the Surrounding Non-tumor Skin
时间窗: Day 182
Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF volume after 6 months of treatment. Tumor dimensions were measured using a standardized ruler.
次要结局
- Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin(Day 182)
- Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements(Baseline, Day 182)
- Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182(Baseline, Day 182)
- Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182(Baseline, Day 182)