CompletedPhase 2

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

United States199 participantsStarted 2021-09-29

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. 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:
✓. Participant agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided
✓. Participant agrees not to use tanning beds
✓. Participant is willing to forego treatment of each Target cNF, except protocol specified therapy, during the study
✓. Female participants who are women of childbearing potential must have a negative urine pregnancy test result and be willing to use a protocol approved, contraceptive method for the duration of the study

Exclusion criteria

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: 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

Timeframe: Day 182

Trial details

NCT IDNCT05005845

SponsorNFlection Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-10-02

Results submitted2025-12-17

View on ClinicalTrials.gov More Cutaneous Neurofibroma trials