Cutaneous NeurofibromaPhase 2NCT05005845
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
A Phase 2 study testing NFX-179 gel in people with Cutaneous Neurofibroma and Neurofibromatosis 1, run by NFlection Therapeutics, Inc. at 23 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cutaneous Neurofibroma, Neurofibromatosis 1
- What is being tested?
- NFX-179 gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 199 participants
- Where
- 23 sitesUnited States
- Study dates
- 29 Sep 2021to 2 Oct 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- Has, in the investigator's opinion, a clinically typical appearance
- Is not within 1 centimeter (cm) of the orbital rim
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- 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.
- 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
- Participant is willing and able to follow all study instructions and to attend all study visits.
Who cannot join
- 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
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- 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.
- 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
- Participant has a history of hypersensitivity to any of the ingredients in the study medications
- 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
- Participant has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
- 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
- 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
- Participant has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
The Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dermatology Treatment and Research Center
Dallas, Texas, 75230
Northshore University HealthSystem
Evanston, Illinois, 60077
Center for Dermatology Clinical Research, Inc.
Fremont, California, 94538
University of Florida
Gainesville, Florida, 32610
UTHealth McGovern Medical School
Houston, Texas, 77030
Dawes Fretzin Clinical Research Group
Indianapolis, Indiana, 46250
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205-7199
Children's Hospital Los Angeles
Los Angeles, California, 90027
The Education & Research Foundation, Inc.
Lynchburg, Virginia, 24501
Apex Clinical Research Center, LLC
Mayfield Heights, Ohio, 44124
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112
Sadick Research Group, LLC
New York, New York, 10075
Skin Specialists, P.C.
Omaha, Nebraska, 68144
Oregon Health & Science University
Portland, Oregon, 97239
Skin Search of Rochester, Inc.
Rochester, New York, 14623
Northwest Arkansas Clinical Trials Center, PLLC
Rogers, Arkansas, 72758
University of Utah
Salt Lake City, Utah, 84108
University Clinical Trials, Inc.
San Diego, California, 92123
Derm Dox Center for Dermatology
Sugarloaf, Pennsylvania, 18249
Children's National Hospital
Washington D.C., District of Columbia, 20010
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157
About this study
Official title: A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2 Dose-Response Study to Determine Safety and Effectiveness of Two Concentrations of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
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.
- Conditions
- Cutaneous Neurofibroma, Neurofibromatosis 1
- Being tested
- NFX-179 gel, Vehicle gel
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NFX-179 gelDrug
Also called NFX-179 topical gel
NFX-179 topical gel is the active investigational product being studied
Vehicle gelDrug
Also called NFX-179 vehicle gel
NFX-179 vehicle gel is the placebo comparator for this study
| Study group | Role | What they receive |
|---|---|---|
| NFX-179 Topical Gel 0.5% | Experimental | NFX-179 topical gel 0.5% will be applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days. Receives: Drug: NFX-179 gel |
| NFX-179 Topical Gel 1.5% | Experimental | NFX-179 topical gel 1.5% will be applied once daily to the target cNFs for 182 days. Receives: Drug: NFX-179 gel |
| NFX-179 Topical Gel Vehicle | Placebo comparator | NFX-179 topical gel vehicle will be applied once daily to the target cNFs for 182 days. Receives: Drug: Vehicle gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an event that occurred or worsened on or after the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Measured: 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
Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF volume after 6 months of treatment. Tumor dimensions were measured using a standardized ruler.
Measured: Day 182
Secondary outcomes (4)
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin
Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF height after 6 months of treatment. Tumor dimensions were measured using a standardized ruler.
Measured: Day 182
Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements
Mean percent change of tumor volume was calculated from ruler measurements throughout the course of treatment. Presented here are data analyzed at the participant level.
Measured: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182
Effect of treatment with the PTA was the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The PTA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). Presented here are data analyzed at the participant level.
Measured: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182
The SSA was the participant's assessment of the average overall severity of each Target cNF at a particular time point. The SSA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe).
Measured: Baseline, Day 182
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2021
- Study start29 Sep 2021
- Primary completion2 Oct 2023
- Study completion2 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cutaneous NeurofibromaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 199 participants (actual)
Sponsor and investigators
- Lead sponsor
- NFlection Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05005845
- Sponsor's protocol ID
- NFX-179-NF1-202
- Status last verified
- Jan 2026
- First submitted
- 2 Aug 2021
- Last updated
- 17 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2 Dose-Response Study to Determine Safety and Effectiveness of Two Concentrations of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
- ClinicalTrials.gov ID
- NCT05005845
- Acronym / sponsor ID
- NFX-179-NF1-202
- Conditions
- Cutaneous Neurofibroma, Neurofibromatosis 1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 199 participants (actual)
- Lead sponsor
- NFlection Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Sep 2021 (actual)
- Primary completion
- 2 Oct 2023 (actual)
- Study completion
- 2 Oct 2023 (actual)
- First posted
- 16 Aug 2021 (actual)
- Results
- Posted 17 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| NFX-179 Topical Gel 0.5% | Experimental | Drug: NFX-179 gelNFX-179 topical gel 0.5% will be applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days. |
| NFX-179 Topical Gel 1.5% | Experimental | Drug: NFX-179 gelNFX-179 topical gel 1.5% will be applied once daily to the target cNFs for 182 days. |
| NFX-179 Topical Gel Vehicle | Placebo comparator | Drug: Vehicle gelNFX-179 topical gel vehicle will be applied once daily to the target cNFs for 182 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (29 listed)
- 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
Key exclusion criteria (18 listed)
- 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:
- The participant has used any of the following systemic medications therapies within the specified period prior to Visit 1 (screening visit):
- Participant has a history of hypersensitivity to any of the ingredients in the study medications
- 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
- Participant has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NFX-179 gelAlso: NFX-179 topical gelNFX-179 topical gel is the active investigational product being studied | Drug | NFX-179 Topical Gel 0.5%, NFX-179 Topical Gel 1.5% |
| Vehicle gelAlso: NFX-179 vehicle gelNFX-179 vehicle gel is the placebo comparator for this study | Drug | NFX-179 Topical Gel Vehicle |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline (Day 1) up to Day 211
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an event that occurred or worsened on or after the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Volume Above the Surrounding Non-tumor Skin
Time frame: Day 182
Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF volume after 6 months of treatment. Tumor dimensions were measured using a standardized ruler.
4 secondary outcomes
Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin
Time frame: Day 182
Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements
Time frame: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182
Time frame: Baseline, Day 182
Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182
Time frame: Baseline, Day 182
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NFX-179 gel in people with Cutaneous Neurofibroma and Neurofibromatosis 1, run by NFlection Therapeutics, Inc. at 23 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 29 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: Baseline (Day 1) up to Day 211). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2026.