12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi

NCT05195762 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: 60276
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This study of NFX-179 is an open-label study, evaluating safety, tolerability, pharmacodynamic activity, and the clinical effect in subjects with Epidermal Nevus(ENS). NFX-179 is formulated as a gel for topical administration. NFX-179 has been shown in animal studies and in human extracts to suppress p-ERK with systemic absorption of NFX-179 following topical application to be extremely low, based on serum values observed in animal studies. Primary objectives:

• To determine the pharmacodynamic activity of NFX-179 Gel as defined by suppression of phospho-ERK (p-ERK) levels in Target EN in treatment group after 12 weeks of once-daily (QD) application
• To determine the safety and tolerability of treatment with NFX-179 Gel 1.50% applied QD for 12 weeks. Secondary objectives:
• Clinical effect of NFX-179 Gel 1.5% defined as the percent change in EN volume after 12 weeks of QD application

Conditions

Epidermal Nevi; Nevus Sebaceus

Outcome Measures

Primary Outcomes (2)

• The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, erythema, edema, scabbing/crusting, and vesiculation/erosion

  To determine the safety and tolerability of treatment with NFX-179 gel applied QD for 12 weeks of treatment.

  12 weeks

• The frequency of adverse events and serious adverse events as assessed by CTCAE v4.0

  To determine the safety and tolerability of treatment with NFX-179 gel applied QD for 12 weeks of treatment.

  12 weeks

Secondary Outcomes (2)

• Change in ILA (Investigator's Lesion Assessment)

  12 weeks

• Percent change in EN volume

  12 weeks

Study Arms (1)

Patients receiving NFX-179 Gel

EXPERIMENTAL

NFX-179 Gel 1.50% applied QD for 12 weeks

Drug: NFX-179 Gel 1.50%

Interventions

NFX-179 Gel 1.50% DRUG

Stanford University (Stanford) is studying a selective mitogen-activated protein kinase kinase (MEK) inhibitor (NFX-179) that has been chemically engineered for topical application to treat epidermal nevus syndromes (ENS). Preclinical studies evaluating topical application of NFX-179 to human nevus sebaceous explants demonstrated that NFX-179 can penetrate the tissue and suppress the p-ERK biomarker. It was also determined that NFX-179, when incubated with the tissues in medium, also suppressed ERK phosphorylation in human keratinocytic epidermal nevi. This clinical study is designed to evaluate the safety, tolerability, and clinical effect of NFX-179 Gel in Subjects with Epidermal Nevi.

Patients receiving NFX-179 Gel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• In order to be eligible to participate in this study, all of the following criteria must be met:
• Subject is at least 18 years of age
• Subject must provide written informed consent prior to any study procedures
• Subject has a Target Epidermal Nevi for treatment that:
• Is a documented, biopsy proven, keratinocytic epidermal nevi (KEN) or nevus sebaceous (NS) or has a 3mm punch biopsy taken for histologic examination to determine KEN or NS status
• Is a discrete lesion surrounded with at least 5mm of non-involved skin
• Is not irritated
• Does not have an active cutaneous infection
• Has a Length ≥15mm
• Has a surface area ≤100cm2
• Has an Investigator's Lesion Assessment grade ≥2
• Is located at an anatomical site that the subject can reach to apply the study medication
• Has never been surgically treated
• Subject is willing to have hair in the area surrounding the Target EN shaved, if necessary, to obtain photographs
• Subject is willing to minimize exposure of the Target EN to natural or artificial ultraviolet radiation

You may not qualify if:

• Subject has applied any of the following topical products in the previous 30 days on or in proximity to the Target EN that, in the investigator's opinion, impairs evaluation of the Target EN or which exposes the subject to an unacceptable risk by study participation:
• Topical glucocorticoid steroids
• Topical retinoids (e.g., tazarotene, tretinoin, adapalene)
• \> 5% of an alpha-hydroxy acid (e.g., glycolic acid, lactic acid)
• Fluorouracil
• Imiquimod
• The Target EN has ever been treated with a topical MEK inhibitor or a topical BRAF inhibitor
• The Subject has used any of the following systemic medications in the noted time period:
• Systemic retinoids (e.g., etretinate, isotretinoin) in the previous 90 days
• Systemic MEK inhibitors in the previous 180 days
• Systemic BRAF inhibitors in the previous 180 days
• Subject has a history of hypersensitivity to any of the ingredients in the study medications
• Subject has any know intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of the Target EN or which exposes the subject to an unacceptable risk by study participation
• Subject has, in the Investigator's opinion, a clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
• Subject has a history of metastatic disease, or active cancer (excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or Stage 0 chronic lymphocytic lymphoma) within the previous 5 years

Sponsors & Collaborators

• Albert Chiou: lead
• NFlection Therapeutics, Inc.: collaborator

Study Sites (1)

Stanford University

Redwood City, California, 94063, United States

Location

MeSH Terms

Conditions

Nevus, Sebaceous of Jadassohn

Condition Hierarchy (Ancestors)

Nevus; Nevi and Melanomas; Neoplasms by Histologic Type; Neoplasms; Neurocutaneous Syndromes; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Albert S Chiou, MD, MBA

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Associate Professor

Model Details: Open-label, all participants will apply NFX-179 gel for 12 weeks

Study Record Dates

• First Submitted: December 13, 2021
• First Posted: January 19, 2022
• Study Start: June 1, 2023
• Primary Completion: June 1, 2024
• Study Completion: December 1, 2024
• Last Updated: May 28, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)