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

NCT05005845 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NFX-179-NF1-202
• Study Type: interventional
• Target: 199
• Locations: 1 country
• Sites: 23

Brief Summary

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.

Conditions

Cutaneous Neurofibroma; Neurofibromatosis 1

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Day 182

Secondary Outcomes (4)

• Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin

  Day 182

• Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements

  Baseline, Day 182

• Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182

  Baseline, Day 182

• Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182

  Baseline, Day 182

Study Arms (3)

NFX-179 Topical Gel 0.5%

EXPERIMENTAL

NFX-179 topical gel 0.5% will be applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days.

Drug: NFX-179 gel

NFX-179 Topical Gel 1.5%

EXPERIMENTAL

NFX-179 topical gel 1.5% will be applied once daily to the target cNFs for 182 days.

Drug: NFX-179 gel

NFX-179 Topical Gel Vehicle

PLACEBO COMPARATOR

NFX-179 topical gel vehicle will be applied once daily to the target cNFs for 182 days.

Drug: Vehicle gel

Interventions

NFX-179 gel DRUG

NFX-179 topical gel is the active investigational product being studied

Also known as: NFX-179 topical gel

NFX-179 Topical Gel 0.5%; NFX-179 Topical Gel 1.5%

Vehicle gel DRUG

NFX-179 vehicle gel is the placebo comparator for this study

Also known as: NFX-179 vehicle gel

NFX-179 Topical Gel Vehicle

Eligibility Criteria

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

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.)

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

Sponsors & Collaborators

• NFlection Therapeutics, Inc.: lead

Study Sites (23)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205-7199, United States

Location

Northwest Arkansas Clinical Trials Center, PLLC

Rogers, Arkansas, 72758, United States

Location

Center for Dermatology Clinical Research, Inc.

Fremont, California, 94538, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University Clinical Trials, Inc.

San Diego, California, 92123, United States

Location

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Northshore University HealthSystem

Evanston, Illinois, 60077, United States

Location

Dawes Fretzin Clinical Research Group

Indianapolis, Indiana, 46250, United States

Location

The Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location

Skin Specialists, P.C.

Omaha, Nebraska, 68144, United States

Location

Sadick Research Group, LLC

New York, New York, 10075, United States

Location

Skin Search of Rochester, Inc.

Rochester, New York, 14623, United States

Location

Wake Forest School of Medicine

Winston-Salem, North Carolina, 27157, United States

Location

Apex Clinical Research Center, LLC

Mayfield Heights, Ohio, 44124, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Derm Dox Center for Dermatology

Sugarloaf, Pennsylvania, 18249, United States

Location

Dermatology Treatment and Research Center

Dallas, Texas, 75230, United States

Location

UTHealth McGovern Medical School

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

The Education & Research Foundation, Inc.

Lynchburg, Virginia, 24501, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Vice President, Clinical Operations
• Organization: NFlection

phorwath@nflection.com

6513439370

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 2, 2021
• First Posted: August 16, 2021
• Study Start: September 29, 2021
• Primary Completion: October 2, 2023
• Study Completion: October 2, 2023
• Last Updated: February 17, 2026
• Results First Posted: February 17, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (23)