Epidermal NeviEarly Phase 1NCT07813533
A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.
A Early Phase 1 study testing sirolimus topical gel 0.2% in people with Epidermal Nevi, run by Northwestern University at 1 site in United States.
Trial summary
We hypothesize that topical sirolimus could suppress the mTOR activation of linear verrucous epidermal nevi, particularly when the variants are involving genes upstream of mTOR and perhaps even those of the RAS pathway through crosstalk and downstream signaling. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Epidermal Nevi
- What is being tested?
- sirolimus topical gel 0.2%
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2026to Dec 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients 6 years of age or above with a non-inflammatory epidermal nevi
- Lesion(s) that have been present for at least 3 years and are relatively stable
- At least 2 cm in length (ideally continuous, but can be cumulatively)
- The lesions is raised, hyperpigmented compared to the surrounding skin, and is noninflammatory with a physician global assessment (PGA) of at least 3 (moderate).
- Agree to avoid emollient use at the nevus site throughout the study
- Agree to not bath or shower 2 hours post-application, knowing that the gel can be applied after bathing or showering.
Who cannot join
- Patients currently or recently (within past 4 weeks) using a topical keratolytic or topical anti-inflammatory medication at the lesional site (i.e. topical steroid, topical retinoid, alpha-OH acid)
- Pregnant or planning to become pregnant during the course of the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Northwestern University Feinberg School of Medicine, Department of Dermatology
Chicago, Illinois, 60611
About this study
We hypothesize that topical sirolimus could suppress the mTOR activation of linear verrucous epidermal nevi, particularly when the variants are involving genes upstream of mTOR and perhaps even those of the RAS pathway through crosstalk and downstream signaling. Indeed, a recent study noted a few cases of epidermal nevi that were improved within 3-4 months by the use of topical sirolimus. We propose to perform a pilot study of epidermal nevi to observe the impact of treatment twice daily of epidermal nevi with serial photography and scoring to capture alterations during the 3 months of the study. We will also grade local tolerance to the topical gel.
Read the detailed description
Epidermal nevi are skin growths that develop because of a genetic change that occurs after conception, meaning only some skin cells are affected. These growths appear as thickened, often darker areas of skin that form lines or swirls. On the arms and legs, they usually appear as straight streaks, while on the trunk and face they follow curved patterns called the lines of Blaschko, which reflect how skin cells develop before birth.
These skin changes are caused by specific genetic changes that turn on certain cell growth pathways inside skin cells. Most linear verrucous epidermal nevi involve one of two major pathways that control cell growth: the PI3K/AKT/mTOR pathway or the RAS/MAPK pathway. In more than 40% of cases, changes in the FGFR3 or PIK3CA genes cause constant activation of the PI3K/AKT/mTOR pathway, leading to excess skin growth.
Sirolimus (also called rapamycin) is a medication that blocks the mTOR pathway, slowing down cell growth and protein production. Medications like this are already used to treat certain cancers caused by overactive mTOR signaling.
Topical sirolimus is FDA-approved as a 0.2% gel (Hyftor) to treat facial angiofibromas in people with tuberous sclerosis complex (TSC). In TSC, genetic changes cause the mTOR pathway to be overly active, leading to skin growths called angiofibromas. Studies have shown that applying sirolimus to the skin can reduce the size and visibility of these lesions.
- Conditions
- Epidermal Nevi
- Being tested
- sirolimus topical gel 0.2%
- Keywords
- epidermal nevi, topical sirolimus, Hyftor, mTOR
- Medical subject headings
- Nevus, Sebaceous of Jadassohn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sirolimus topical gel 0.2%Drug
Also called Hyftor
Topical sirolimus will be applied twice daily to the nevi with a maximum daily dose of 600 mg for patients 6-11 years of age and 800 mg for patients 12 years and older.
| Study group | Role | What they receive |
|---|---|---|
| Patients 6 years of age or older with a non-inflammatory epidermal nevi | Experimental | Not described Receives: Drug: sirolimus topical gel 0.2% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement of nevi flattening and normalization of skin color
The extent of nevi improvement will be scored at baseline and week 12 from standardized photographs by two independent asessors with an adjudicator (if needed).
The score will be defined as follows:
0 = 100% improvement
- = 90-99% improvement
- = 69-89% improvement
- = 50-69% improvement
- = 30-49% improvement
- = 10-29% improvement
- = 0-9% improvement
- = worsening
Measured: 12 weeks
Secondary outcomes (3)
Satisfaction scores of treatment
Patients (and parents/LAR) will be asked for their satisfaction of the treatment (not satisfied, satisfied, very satisfied) at week 12
Measured: 12 weeks
Patient-reported improvement of lesion
Patients (and parents/LAR) will be asked their self-evaluation of lesion improvment from 0-5 (0= cleared, 1= almost cleared, 2= good improvement, 3= slight improvement, 4= no change, 5= worse) at baseline and week 12
Measured: 12 weeks
Reduction in peak itch score
At least 2-point reduction in peak itch NRS score (on a scale of 0-10, 0=no itch and 10=worse itch) from baseline to week 12
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2026
- Study startOct 2026 (estimated)
- Primary completionSep 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermal NeviStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amy Paller, Northwestern University
- Investigators
- Amy Paller, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07813533
- Sponsor's protocol ID
- STU00224904
- Status last verified
- Sep 2026
- First submitted
- 4 Sep 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.
- ClinicalTrials.gov ID
- NCT07813533
- Acronym / sponsor ID
- STU00224904
- Conditions
- Epidermal Nevi
- Keywords
- epidermal nevi, topical sirolimus, Hyftor, mTOR
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Study start
- Oct 2026 (estimated)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 10 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
We hypothesize that topical sirolimus could suppress the mTOR activation of linear verrucous epidermal nevi, particularly when the variants are involving genes upstream of mTOR and perhaps even those of the RAS pathway through crosstalk and downstream signaling. Indeed, a recent study noted a few cases of epidermal nevi that were improved within 3-4 months by the use of topical sirolimus. We propose to perform a pilot study of epidermal nevi to observe the impact of treatment twice daily of epidermal nevi with serial photography and scoring to capture alterations during the 3 months of the study. We will also grade local tolerance to the topical gel.
Detailed description
Epidermal nevi are skin growths that develop because of a genetic change that occurs after conception, meaning only some skin cells are affected. These growths appear as thickened, often darker areas of skin that form lines or swirls. On the arms and legs, they usually appear as straight streaks, while on the trunk and face they follow curved patterns called the lines of Blaschko, which reflect how skin cells develop before birth.
These skin changes are caused by specific genetic changes that turn on certain cell growth pathways inside skin cells. Most linear verrucous epidermal nevi involve one of two major pathways that control cell growth: the PI3K/AKT/mTOR pathway or the RAS/MAPK pathway. In more than 40% of cases, changes in the FGFR3 or PIK3CA genes cause constant activation of the PI3K/AKT/mTOR pathway, leading to excess skin growth.
Sirolimus (also called rapamycin) is a medication that blocks the mTOR pathway, slowing down cell growth and protein production. Medications like this are already used to treat certain cancers caused by overactive mTOR signaling.
Topical sirolimus is FDA-approved as a 0.2% gel (Hyftor) to treat facial angiofibromas in people with tuberous sclerosis complex (TSC). In TSC, genetic changes cause the mTOR pathway to be overly active, leading to skin growths called angiofibromas. Studies have shown that applying sirolimus to the skin can reduce the size and visibility of these lesions.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients 6 years of age or older with a non-inflammatory epidermal nevi | Experimental | Drug: sirolimus topical gel 0.2% |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patients 6 years of age or above with a non-inflammatory epidermal nevi
- Lesion(s) that have been present for at least 3 years and are relatively stable
- At least 2 cm in length (ideally continuous, but can be cumulatively)
- The lesions is raised, hyperpigmented compared to the surrounding skin, and is noninflammatory with a physician global assessment (PGA) of at least 3 (moderate).
- Agree to avoid emollient use at the nevus site throughout the study
Key exclusion criteria (2 listed)
- Patients currently or recently (within past 4 weeks) using a topical keratolytic or topical anti-inflammatory medication at the lesional site (i.e. topical steroid, topical retinoid, alpha-OH acid)
- Pregnant or planning to become pregnant during the course of the study
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 |
|---|---|---|
| sirolimus topical gel 0.2%Also: HyftorTopical sirolimus will be applied twice daily to the nevi with a maximum daily dose of 600 mg for patients 6-11 years of age and 800 mg for patients 12 years and older. | Drug | Patients 6 years of age or older with a non-inflammatory epidermal nevi |
Primary outcome
Improvement of nevi flattening and normalization of skin color
Time frame: 12 weeks
The extent of nevi improvement will be scored at baseline and week 12 from standardized photographs by two independent asessors with an adjudicator (if needed). The score will be defined as follows: 0 = 100% improvement 1. = 90-99% improvement 2. = 69-89% improvement 3. = 50-69% improvement 4. = 30-49% improvement 5. = 10-29% improvement 6. = 0-9% improvement 7. = worsening
3 secondary outcomes
Satisfaction scores of treatment
Time frame: 12 weeks
Patient-reported improvement of lesion
Time frame: 12 weeks
Reduction in peak itch score
Time frame: 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- 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.
Amy Paller, MD principal investigator
Northwestern University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing sirolimus topical gel 0.2% in people with Epidermal Nevi, run by Northwestern University at 1 site 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 6 inclusion and 2 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: opening soon, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dermatology CTU (312-503-5944, NUderm-research@northwestern.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement of nevi flattening and normalization of skin color (time frame: 12 weeks). 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 10 Sep 2026.