This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days. Subjects will be randomly assigned to 1 of 4 treatment groups. 3 of the treatment groups will receive a specific dose NFX-179, and 1 group will receive placebo. The subject, study doctor, and NFlection Therapeutics will not know what treatment group each subject is assigned. Study participation requires at least 7 clinic visits, blood, urine, and tissue collection, images of the treated cutaneous neurofibromas, electrocardiograms, and information regarding the subject's medical and disease history.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
48
gel for topical administration
vehicle gel for topical administration
Center for Dermatology Clinical Research, Inc.
Fremont, California, United States
Minnesota Clinical Study Center
New Brighton, Minnesota, United States
Skin Search of Rochester, Inc.
Rochester, New York, United States
DermResearch
Austin, Texas, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
The Education & Research Foundation, Inc.
Lynchburg, Virginia, United States
Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application
Pharmacodynamic activity (biochemical and physiologic effects of drugs) of NFX-179 Gel as defined by suppression of phospho-ERK (p-ERK) levels in Target cNF Tumors in each NFX-179 Gel group compared with the Vehicle Gel group after 28 days of once-daily (QD) application will be measured at Week 4
Time frame: Baseline through Week 4
Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment
Safety and tolerability will be measured via a local tolerability assessment. The investigator will assess erythema, edema, scabbing/crusting, vesiculation, and erosion. The subject will assess stinging, burning, and pruritus. All assessments are performed using a 4-point scale (0 none, 1 mild, 2 moderate, 3 severe).
Time frame: Baseline through Week 4
Assessment of Adverse Events
Assessment of adverse events (AEs)
Time frame: Baseline through Week 8
Percent Change in cNF Tumor Volume (Cubic Millimeters)
Percent change in cNF tumor volume after 28 days of QD applications of NFX-179 gel based on tumor volume derived from ruler measurements.
Time frame: Baseline through Week 4
Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration
Systemic exposure of NFX-179 will be measured during the 28 days of QD applications at select investigative sites. Pharmacokinetic samples will be drawn at the baseline visit and week 4 visit. 5 time point samples will be collected at the week 4 visit.
Time frame: Baseline through Week 4
Change in Physician Assessment of Tumor Severity Score
Effect of treatment with The Physician Tumor Assessment is the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The Physician Tumor Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit.
Time frame: Baseline through Week 4
Change in Subject Self-Assessment of Tumor Severity Score
The Subject Self-Assessment is the subject's assessment of the average overall severity of each Target cNF at a particular time point and is not a comparison with any other time point. The Subject Self-Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit.
Time frame: Baseline through Week 4
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.