NFX-179 Gel for Neurofibromatosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new topical gel, NFX-179, for safety and effectiveness in treating cutaneous neurofibromas (cNFs), which are skin growths associated with Neurofibromatosis 1 (NF1). The study compares two concentrations of the gel to a placebo (vehicle gel), assessing how well each reduces these growths with daily application. It suits adults with NF1 who have at least 10 specific cNFs on areas like the face or upper body that impact daily life. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of participants.
Do I need to stop my current medications for the trial?
The trial requires you to stop certain topical and systemic medications before starting. For example, you must stop using corticosteroids and prescription retinoids 30 days before, and systemic retinoids 90 days before. Check with the study team for specific guidance on your current medications.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that NFX-179 gel was safe and well-tolerated in earlier studies. Participants who used it daily for 28 days on cutaneous neurofibromas (cNF) experienced no treatment-related side effects on their skin or in their bodies. Additionally, researchers monitored the drug levels in the bloodstream and found no harmful concentrations. This suggests that the gel is unlikely to cause serious side effects when used as directed. These findings indicate that NFX-179 gel is safe for further testing in humans.12345
Why are researchers excited about this trial's treatments?
Researchers are excited about NFX-179 gel for treating cutaneous neurofibromas in Neurofibromatosis 1 because it offers a novel topical approach. Unlike many existing treatments that involve surgical removal or laser therapy of the tumors, NFX-179 gel is non-invasive, providing an easier and potentially safer option. The gel contains a unique active ingredient that targets specific pathways in the skin to reduce tumor growth, which could make it a game-changer for patients who prefer a less aggressive treatment. Additionally, having both 0.5% and 1.5% concentrations allows for personalized treatment, potentially optimizing effectiveness and minimizing side effects.
What evidence suggests that this trial's treatments could be effective for cutaneous neurofibromas?
Research shows that NFX-179 Gel holds promise for treating skin growths known as cutaneous neurofibromas (cNFs) in individuals with Neurofibromatosis 1 (NF1). In this trial, participants will receive either a 1.5% concentration of NFX-179 Gel, a 0.5% concentration, or a vehicle gel as a placebo comparator. One study found that the 1.5% concentration significantly shrinks these growths with few side effects. The gel penetrates the skin and blocks a protein pathway called MEK, which helps slow tumor growth. Another study found that using a 0.5% concentration for 28 days led to a 47% decrease in a marker called p-ERK, linked to tumor activity. These findings suggest that NFX-179 Gel could effectively reduce cNFs in people with NF1.13467
Are You a Good Fit for This Trial?
Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive NFX-179 Gel or vehicle gel applied topically once daily to target cNFs for 182 days
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- NFX-179 gel
- Vehicle gel
Find a Clinic Near You
Who Is Running the Clinical Trial?
NFlection Therapeutics, Inc.
Lead Sponsor