Title

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2a Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
  • Phase

    Phase 2
  • Study Type

    Interventional
  • Status

    Completed No Results Posted
  • Study Participants

    48
This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days. Subjects will be randomly assigned to 1 of 4 treatment groups. 3 of the treatment groups will receive a specific dose NFX-179, and 1 group will receive placebo. The subject, study doctor, and NFlection Therapeutics will not know what treatment group each subject is assigned. Study participation requires at least 7 clinic visits, blood, urine, and tissue collection, images of the treated cutaneous neurofibromas, electrocardiograms, and information regarding the subject's medical and disease history.
Study Started
Aug 21
2020
Primary Completion
Apr 08
2021
Study Completion
Apr 14
2021
Last Update
Aug 13
2021

Drug NFX-179 Gel

gel for topical administration

Drug Vehicle Gel

vehicle gel for topical administration

NFX-179 Gel Low Experimental

NFX-179 Gel for topical administration, once daily for 28 days

NFX-179 Gel Mid Experimental

NFX-179 Gel for topical administration, once daily for 28 days

NFX-179 Gel High Experimental

NFX-179 Gel for topical administration, once daily for 28 days

Vehicle Arm Placebo Comparator

Vehicle Gel, for topical administration, once daily for 28 days

Criteria

Inclusion Criteria:

Subject is at least 18 years of age
Subject must provide written informed consent prior to any study procedures
Subject must have a clinical diagnosis of NF1

Subject has 6 Study cNF Tumors (5 Target cNF Tumors [1 on the face; 4 on the anterior trunk or upper extremities] that will be treated with the assigned study medication;1 Untreated cNF Tumor on the anterior trunk or upper extremities) that each meet the following criteria:

Has, in the investigator's opinion, a clinically typical appearance
Is dome shaped
Is not pedunculated
Is a discrete tumor
Is not irritated
Is not in an area subject to repeated trauma (e.g., area that is shaved, on the beltline, under a bra strap, etc.)
Does not have an active cutaneous infection
Has a diameter that is ≥5mm and ≤10mm
Has a height of ≥2mm
Is, when centered in the center of the provided template, the only cNF tumor visible
Is not within 5mm of the orbital rim.
Subject is willing to have the 5 Target cNF Tumors and the 1 Untreated cNF Tumor excised at the end of the treatment period
Subject is willing to have hair in the area surrounding the Target cNF Tumors shaved, if necessary, to obtain photographs
Subject is willing to minimize exposure of each Target cNF to natural and artificial ultraviolet radiation
Subject is willing to forego treatment of the Target cNF Tumors, except protocol specified therapy, during the study
Female subjects 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
Subject is willing and able to follow all study instructions and to attend all study visits.

Exclusion Criteria:

Subject has applied any of the following topical products in the previous 30 days on or in proximity to any Study cNF Tumor that, in the investigator's opinion, impairs evaluation of any the tumor or which exposes the subject to an unacceptable risk by study participation:

Corticosteroids
Retinoids (e.g., tazarotene, tretinoin, adapalene)
> 5% of an alpha-hydroxy acid (e.g., glycolic acid, lactic acid)
Fluorouracil
Imiquimod
Any Study cNF Tumor has ever been treated with an MEK inhibitor or a BRAF inhibitor

The subject has used any of the following systemic medications in the noted time period:

Retinoids (e.g., etretinate, isotretinoin) within the previous 90 days
MEK inhibitors within the previous 180 days
BRAF inhibitors within the previous 180 days
Subject has a history of hypersensitivity to any of the ingredients in the study medications
Subject has any known intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of a Target cNF Tumor or which exposes the subject to an unacceptable risk by study participation
Subject has, in the investigator's opinion, 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 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
Subject has any condition (e.g., other skin conditions or diseases, metabolic dysfunction, physical examination findings, clinical laboratory findings) or situation (e.g., vacation, scheduled surgery) that would, in the investigator's opinion, impair evaluation of a Target cNF Tumor or which exposes the subject to an unacceptable risk by study participation
Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
No Results Posted