Hemangioma SkinEarly Phase 1TSTIHNCT06273111
Topical Simvastatin for Treating Infantile Hemangioma
A Early Phase 1 study testing 5% simvastatin ointment in people with Hemangioma Skin, run by Joyce Teng at 1 site in United States.
Trial summary
This is a 48-week, open-label pilot study to evaluate the safety, including systemic exposure and skin tolerability, and preliminary efficacy of 5% simvastatin ointment in the treatment of 15 children with newly diagnosed superficial proliferating IH. Read more about this study
- Who is it for?
- Ages 3 to 6 months
- Conditions the study is for
- Hemangioma Skin
- What is being tested?
- 5% simvastatin ointment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2026to 30 Jun 2029
- 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 3 to 6 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants may be included in the study only if they meet all of the following criteria:
- Healthy children aged between 3-6 months without medical disorders that contradict topical statin treatment.
- Infants with newly diagnosed superficial IH. [3] Participants must possess at least one IH lesion with the longest diameter equal to or greater than 1 cm but less than 1% BSA, located on any part of the body except the lips.
- [4] Written informed consent from the parent(s)/guardian(s) must be obtained before any study procedure is performed.
- [5] Parent(s)/guardian(s) are willing to comply with the study protocol
Who cannot join
- Participants meeting any of the following criteria will not be eligible to participate in the study:
- IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.
- IH with active ulceration at screening visit.
- IH to be treated involving the lips mainly.
- IH with high-risk criteria that need systemic propranolol treatment to avoid the delay with standard treatment
- Participants with concurrent skin conditions that may impede accurate clinical assessment of the IH.
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- Participants with hereditary or metabolic disorders requiring systemic statin therapy.
- Participants who are allergic to statins, or other ingredients present in the topical medication.
- Participants who have received any of the following treatments for their IH:
- Topical medical therapy, i.e. imiquimod, sirolimus, timolol, intermediate or high strength steroids, etc. within the past 4 weeks ii) Systemic medical therapy, i.e. beta blockers, steroids, sirolimus for IH within the past 3 months iii) Surgical intervention including laser treatment within the past 6 weeks
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
Stanford University
Palo Alto, California, 94304
Ramrada Lekwuttikarn, MD, 650-313-8207, ramrada1@stanford.edu
Chaw-Ning Lee, MD, 650-497-7230, cnlee06@stanford.edu
About this study
Official title: A Pilot Study Using Topical Statins to Treat Children With Infantile Hemangioma
This is a 48-week, open-label pilot study to evaluate the safety, including systemic exposure and skin tolerability, and preliminary efficacy of 5% simvastatin ointment in the treatment of 15 children with newly diagnosed superficial proliferating IH.
The primary objective:
Read more
To evaluate the safety, including systemic exposure and skin tolerability of topical treatment with 5% simvastatin ointment for superficial proliferating IH over 48 weeks.
The secondary objective:
1.1 To evaluate the efficacy and durability of 5% simvastatin ointment when topical treatment is administered twice daily for 24 weeks, followed by a 24-week post-treatment follow-up period. Evaluation is performed at each clinic visit via investigator global assessment (IGA) based on standardized 3D digital photography and hemangioma activity score (HAS).
1.2 To evaluate the impact of 5% simvastatin ointment on quality of life using the IH-QoL questionnaire.
The exploratory objective:
To explore whether clinical response and regrowth following topical 5% simvastatin treatment are associated with SOX18-mevalonate pathway-axis activation in infantile hemangioma tissue.
- Conditions
- Hemangioma Skin
- Being tested
- 5% simvastatin ointment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5% simvastatin ointmentDrug
5% simvastatin ointment will be applied directly on IH lesion twice per day for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| 5% simvastatin ointment | Experimental | Participants will be applied 5% simvastatin ointment on IH lesion Receives: Drug: 5% simvastatin ointment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks
The primary endpoint is the evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components:
- Adverse Events: The incidence, severity, and proportion of participants experiencing adverse events (AEs) from Week 0 through Week 48.
- Systemic Exposure: The proportion of participants with detectable serum simvastatin levels, alongside descriptive statistics of measured concentrations, at Week 24 (End of Treatment).
- Skin Tolerability: The incidence and maximum severity score of local skin reactions at the treatment site at Week 24 (End of Treatment).
Measured: Baseline through week 48
Secondary outcomes (3)
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Key Efficacy Endpoints (Week 24)
The secondary endpoint is the evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components:
- Key Efficacy Endpoints (Week 24)
- Percentage of participants achieving 50% HAS reduction from baseline at week 24 Percentage of participants achieving a 75% HAS reduction from baseline at week 24.
- Percentage of participants achieving complete or nearly complete resolution of the targeted IH (IGA 0 or 1), defined as a minimal degree of telangiectasia, skin thickening, and no definitive palpable cutaneous texture changes, at week 24.
- Percentage of participants achieving targeted IH stabilization - (no noticeable change to baseline) at week 24.
- Percentage of patients whose have 50% improvement captured by 3D photographs and agreed by blinded assessor at week 24
- Percentage of participants with a significant improvement in quality-of-life, defined as > 50% reduction in the IH-QoL questionnaire score from base
Measured: Baseline through week 24
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Treatment Failure and Durability Endpoints
The secondary endpoint is the evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components:
- Treatment Failure and Durability Endpoints
- Disease Progression: Percentage of participants whose targeted IH progresses to the point of requiring systemic therapy, laser, or surgical interventions (resulting in Early Termination).
- Treatment Rebound (Durability): Percentage of participants experiencing targeted IH regrowth during the 24-week follow-up period (Weeks 24 to 48), alongside the median and range of time (in weeks) from Week 24 to the first notable rebound
Measured: Baseline through week 48
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Time-to-Event Assessments
The secondary endpoint is the evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components:
- Time-to-Event Assessments
- Time to Notable Response: The median and range of time (in weeks) from baseline to the first documented 25% reduction in HAS.
- Time to Progression: The median time to progression requiring rescue therapy (systemic, laser, or surgery), estimated using the Kaplan-Meier method. Participants without disease progression will be censored at Week 24 (or at their date of early discontinuation).
Measured: Baseline through week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2024
- Study start1 Nov 2026 (estimated)
- Primary completion31 Mar 2028 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemangioma SkinStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Joyce Teng
- Sponsor type
- University, hospital or other
- Collaborators
- Stanford University, National Institutes of Health (NIH)
- Responsible party
- Sponsor-investigator, Joyce Teng, Stanford University
- Investigators
- Joyce Teng, MD, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06273111
- Sponsor's protocol ID
- 73840
- Acronym
- TSTIH
- Status last verified
- Sep 2026
- First submitted
- 15 Feb 2024
- Last updated
- 14 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 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study Using Topical Statins to Treat Children With Infantile Hemangioma
- ClinicalTrials.gov ID
- NCT06273111
- Acronym / sponsor ID
- TSTIH / 73840
- Conditions
- Hemangioma Skin
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Joyce TengUniversity, hospital or other
- Collaborators
- Stanford University, National Institutes of Health (NIH)
- Study start
- 1 Nov 2026 (estimated)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 22 Feb 2024 (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.
This is a 48-week, open-label pilot study to evaluate the safety, including systemic exposure and skin tolerability, and preliminary efficacy of 5% simvastatin ointment in the treatment of 15 children with newly diagnosed superficial proliferating IH.
The primary objective:
To evaluate the safety, including systemic exposure and skin tolerability of topical treatment with 5% simvastatin ointment for superficial proliferating IH over 48 weeks.
The secondary objective:
1.1 To evaluate the efficacy and durability of 5% simvastatin ointment when topical treatment is administered twice daily for 24 weeks, followed by a 24-week post-treatment follow-up period. Evaluation is performed at each clinic visit via investigator global assessment (IGA) based on standardized 3D digital photography and hemangioma activity score (HAS).
1.2 To evaluate the impact of 5% simvastatin ointment on quality of life using the IH-QoL questionnaire.
The exploratory objective:
To explore whether clinical response and regrowth following topical 5% simvastatin treatment are associated with SOX18-mevalonate pathway-axis activation in infantile hemangioma tissue.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5% simvastatin ointment | Experimental | Drug: 5% simvastatin ointmentParticipants will be applied 5% simvastatin ointment on IH lesion |
Who may qualify
- Ages
- 3 to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Participants may be included in the study only if they meet all of the following criteria:
- Healthy children aged between 3-6 months without medical disorders that contradict topical statin treatment.
- Infants with newly diagnosed superficial IH. [3] Participants must possess at least one IH lesion with the longest diameter equal to or greater than 1 cm but less than 1% BSA, located on any part of the body except the lips.
- [4] Written informed consent from the parent(s)/guardian(s) must be obtained before any study procedure is performed.
- [5] Parent(s)/guardian(s) are willing to comply with the study protocol
Key exclusion criteria (10 listed)
- Participants meeting any of the following criteria will not be eligible to participate in the study:
- IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.
- IH with active ulceration at screening visit.
- IH to be treated involving the lips mainly.
- IH with high-risk criteria that need systemic propranolol treatment to avoid the delay with standard treatment
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 |
|---|---|---|
| 5% simvastatin ointment5% simvastatin ointment will be applied directly on IH lesion twice per day for 24 weeks | Drug | 5% simvastatin ointment |
Primary outcome
Evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks
Time frame: Baseline through week 48
The primary endpoint is the evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components: * Adverse Events: The incidence, severity, and proportion of participants experiencing adverse events (AEs) from Week 0 through Week 48. * Systemic Exposure: The proportion of participants with detectable serum simvastatin levels, alongside descriptive statistics of measured concentrations, at Week 24 (End of Treatment). * Skin Tolerability: The incidence and maximum severity score of local skin reactions at the treatment site at Week 24 (End of Treatment).
3 secondary outcomes
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Key Efficacy Endpoints (Week 24)
Time frame: Baseline through week 24
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Treatment Failure and Durability Endpoints
Time frame: Baseline through week 48
To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Time-to-Event Assessments
Time frame: Baseline through week 48
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 14 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.
Joyce Teng, MD, PhD principal investigator
Stanford 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 5% simvastatin ointment in people with Hemangioma Skin, run by Joyce Teng 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 5 inclusion and 10 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 14 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 Joyce Teng, MD, PhD (650-724-9627, jteng3@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks (time frame: Baseline through week 48). 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 14 Sep 2026.