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

NCT ID: NCT05005845

Last Updated: 2023-11-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 199 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-20
• Study Completion Date: 2023-10-04

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 subjects with cutaneous neurofibromas. At Visit 1, the investigator will identify 10 Target cNFs that fulfil the enrollment criteria. The Target cNFs must be located on the subject'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 QD to the Target cNFs for 182days (26 weeks). During the duration of the study subjects will be evaluated for safety and efficacy.

Conditions

Cutaneous Neurofibroma; Neurofibromatosis 1

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

0.5% NFX-179 gel

Topical gel applied once daily to target cNFs

Group Type: ACTIVE_COMPARATOR

NFX-179 gel

Intervention Type: DRUG

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

1.5% NFX-179 gel

Topical gel applied once daily to target cNFs

Group Type: ACTIVE_COMPARATOR

NFX-179 gel

Intervention Type: DRUG

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

Vehicle gel

Topical gel applied once daily to target cNFs

Group Type: PLACEBO_COMPARATOR

Vehicle gel

Intervention Type: DRUG

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

Interventions

NFX-179 gel

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

Intervention Type: DRUG

Vehicle gel

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

Intervention Type: DRUG

Other Intervention Names

NFX-179 topical gel; NFX-179 vehicle gel

Eligibility Criteria

Inclusion Criteria

1. Subject is at least 18 years of age

2. Subject must provide written informed consent prior to any study procedures

3. Subject must have a clinical diagnosis of NF1

4. Subject 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 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 (e.g., bleeding, inflamed)
• 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
• Target cNFs on the face must have the following tumor dimensions:
• Has a length that is ≥5mm and ≤14mm
• Has a width that is ≥5mm and ≤14mm
• Has a height that is ≥2mm.
• Target cNFs on the anterior trunk or upper extremities must have the following tumor dimensions:
• Has a length that is ≥7mm and ≤14mm
• Has a width that is ≥5mm and ≤14mm
• Has a height that is ≥2mm.

5. Subject agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided

6. Subject agrees NOT to use tanning beds

7. Subject is willing to forego treatment of each Target cNF, except protocol specified therapy, during the study

8. 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

9. Subject is willing and able to follow all study instructions and to attend all study visits.

Exclusion Criteria

1. Subject has used any of the following topical therapies within the specified period prior to Visit 1 on or in proximity to any Target cNF that, in the investigator's opinion, impairs evaluation of any the cNFs or which exposes the subject to an unacceptable risk by study participation:

• Corticosteroids; 30 days
• Prescription retinoids (e.g., tazarotene, tretinoin, adapalene); 30 days
• > 5% of an alpha-hydroxy acid (e.g., glycolic acid, lactic acid); 30 days
• Fluorouracil; 30 days
• Imiquimod; 30 days
• LASER, light (e.g., intense pulsed light [IPL], photo-dynamic therapy [PDT]) or other energy-based therapy; 180 days
• MEK inhibitor or BRAF inhibitor; ever.

2. The subject has used any of the following systemic medications therapies within the specified period prior to Visit 1:

• Retinoids (e.g., etretinate, isotretinoin); 90 days
• MEK inhibitors; 180 days
• BRAF inhibitors; 180 days

3. Subject has a history of hypersensitivity to any of the ingredients in the study medications

4. Subject 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 subject to an unacceptable risk by study participation

5. Subject has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis

6. 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

7. 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 or which exposes the subject to an unacceptable risk by study participation

8. Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NFlection Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Countries

United States

Other Identifiers

NFX-179-NF1-202

Identifier Type: -

Identifier Source: org_study_id