New gel could shrink neurofibroma tumors without surgery
NCT ID NCT05005845
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tested a gel called NFX-179 on skin tumors in 199 adults with neurofibromatosis type 1. Participants applied the gel daily for 26 weeks. The study measured safety and whether tumors shrank in size or height. Results suggest the gel may help reduce tumor volume, but more research is needed.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- NFX-179 gel
- What this could lead to
- If successful, this gel could offer a non-surgical way to shrink or control skin tumors in people with neurofibromatosis type 1.
- What could go wrong
- This is a phase 2 trial with a small number of participants. The gel may not work for everyone, and long-term effects are not yet known.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
199 people
The number who actually took part.
- Started
-
Sep 2021
- Finished
-
Oct 2023
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Participant is at least 18 years of age 2. Participant must provide written informed consent prior to any study procedures 3. Participant must have a clinical diagnosis of NF1 4. 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. 5. Participant agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided 6. Participant agrees not to use tanning beds 7. Participant is willing to forego treatment of each Target cNF, except protocol specified therapy, during the study 8. 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 9. Participant is willing and able to follow all study instructions and to attend all study visits. Exclusion Criteria: 1. 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. 2. 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 3. Participant has a history of hypersensitivity to any of the ingredients in the study medications 4. 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 5. Participant has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis 6. 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 7. 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 8. Participant has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Cutaneous neurofibroma are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Apex Clinical Research Center, LLC
Mayfield Heights, Ohio, 44124, United States
-
Center for Dermatology Clinical Research, Inc.
Fremont, California, 94538, United States
-
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
-
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
-
Dawes Fretzin Clinical Research Group
Indianapolis, Indiana, 46250, United States
-
Derm Dox Center for Dermatology
Sugarloaf, Pennsylvania, 18249, United States
-
Dermatology Treatment and Research Center
Dallas, Texas, 75230, United States
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112, United States
-
Northshore University HealthSystem
Evanston, Illinois, 60077, United States
-
Northwest Arkansas Clinical Trials Center, PLLC
Rogers, Arkansas, 72758, United States
-
Oregon Health & Science University
Portland, Oregon, 97239, United States
-
Sadick Research Group, LLC
New York, New York, 10075, United States
-
Skin Search of Rochester, Inc.
Rochester, New York, 14623, United States
-
Skin Specialists, P.C.
Omaha, Nebraska, 68144, United States
-
The Education & Research Foundation, Inc.
Lynchburg, Virginia, 24501, United States
-
The Johns Hopkins School of Medicine
Baltimore, Maryland, 21287, United States
-
UTHealth McGovern Medical School
Houston, Texas, 77030, United States
-
University Clinical Trials, Inc.
San Diego, California, 92123, United States
-
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205-7199, United States
-
University of Florida
Gainesville, Florida, 32610, United States
-
University of Utah
Salt Lake City, Utah, 84108, United States
-
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Light-Activated cream could slow growth of NF1 skin tumors
- Antioxidant drug shows promise for kids with NF1
- New drug combo shows promise for rare nerve tumors
- Could a common supplement help kids with NF1 learn and move better?
- New drug combo aims to tame rare childhood leukemia
- New drug shows promise for NF1 skin tumors in early trial