NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Completed · Phase 2 · Has a placebo group

Conditions studied: Cutaneous Neurofibroma, Neurofibromatosis 1

In brief

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.

Key facts

Study ID

NCT05005845

Run by

NFlection Therapeutics, Inc.

People needed

199

Starts

2021-09-29

Expected to finish

2023-10-02

Last updated by the study team

2026-02-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Is a discrete cNF surrounded by sufficient non-affected skin that, in the investigator's opinion:
• The dimensions can be measured
• The perimeter can be outlined in the study photographs
• Is not irritated (or example, bleeding, inflamed)
• Is not in an area participant to repeated trauma (or example, area that is shaved, on the beltline, under a bra strap, etc.)
• Does not have an active cutaneous infection
• Target cNFs on the face must have the following tumor dimensions:
• Has a length that is ≥5 millimeters (mm) and ≤14 mm
• Has a width that is ≥5 mm and ≤14 mm
• Has a height that is ≥2 mm.
• Target cNFs on the anterior trunk or upper extremities must have the following tumor dimensions:
• Has a length that is ≥ 7mm and ≤14 mm
• Has a width that is ≥5 mm and ≤14 mm
• Has a height that is ≥2 mm.
• Participant agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided

You may not qualify if…

• 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
• MEK inhibitors; 180 days
• BRAF inhibitors; 180 days
• Participant has a history of hypersensitivity to any of the ingredients in the study medications
• Participant has any known intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of a Target cNF or which exposes the participant to an unacceptable risk by study participation
• Participant has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
• Participant has a history of metastatic disease, or active cancer (excluding nonmelanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or Stage 0 chronic lymphocytic lymphoma) within the previous 5 years
• Participant has any condition (for example, other skin conditions or diseases, metabolic dysfunction, physical examination findings, clinical laboratory findings) or situation (for example, vacation, scheduled surgery) that would, in the investigator's opinion, impair evaluation of a Target cNF or which exposes the participant to an unacceptable risk by study participation
• Participant has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days

Where it is running

• 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
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Minnesota Clinical Study Center — New Brighton, Minnesota, United States
• Skin Specialists, P.C. — Omaha, Nebraska, United States
• Sadick Research Group, LLC — New York, New York, United States
• Skin Search of Rochester, Inc. — Rochester, New York, United States
• Wake Forest School of Medicine — Winston-Salem, North Carolina, United States
• Apex Clinical Research Center, LLC — Mayfield Heights, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Derm Dox Center for Dermatology — Sugarloaf, Pennsylvania, United States
• Dermatology Treatment and Research Center — Dallas, Texas, United States
• UTHealth McGovern Medical School — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• The Education & Research Foundation, Inc. — Lynchburg, Virginia, United States

Full record on ClinicalTrials.gov

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