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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
199
NFX-179 topical gel is the active investigational product being studied
NFX-179 vehicle gel is the placebo comparator for this study
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Northwest Arkansas Clinical Trials Center, PLLC
Rogers, Arkansas, United States
Center for Dermatology Clinical Research, Inc.
Fremont, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University Clinical Trials, Inc.
San Diego, California, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
Northshore University HealthSystem
Evanston, Illinois, United States
Dawes Fretzin Clinical Research Group
Indianapolis, Indiana, United States
The Johns Hopkins School of Medicine
Baltimore, Maryland, United States
...and 13 more locations
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
Time frame: Baseline (Day 1) up to Day 211
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Volume Above the Surrounding Non-tumor Skin
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.
Time frame: Day 182
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin
Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF height after 6 months of treatment. Tumor dimensions were measured using a standardized ruler.
Time frame: Day 182
Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements
Mean percent change of tumor volume was calculated from ruler measurements throughout the course of treatment. Presented here are data analyzed at the participant level.
Time frame: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182
Effect of treatment with the PTA was the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The PTA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). Presented here are data analyzed at the participant level.
Time frame: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182
The SSA was the participant's assessment of the average overall severity of each Target cNF at a particular time point. The SSA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe).
Time frame: Baseline, Day 182
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