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New gel could shrink neurofibroma tumors without surgery

NCT ID NCT05005845

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

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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

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