An Early Phase 1 interventional study of 5% simvastatin ointment in Hemangioma Skin, sponsored by Joyce Teng. Not yet recruiting at 1 site in United States. Open to participants aged 3 Months to 6 Months. Per ClinicalTrials.gov, last updated 2026-09-14.
Sponsored by Joyce Teng · Early Phase 1, Interventional, and Treatment
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.
Participants may be included in the study only if they meet all of the following criteria:
[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
Exclusion Criteria:
Participants meeting any of the following criteria will not be eligible to participate in the study:
Participants who have received any of the following treatments for their IH:
i) 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
Participants will be applied 5% simvastatin ointment on IH lesion
Drug: 5% simvastatin ointment
5% simvastatin ointment will be applied directly on IH lesion twice per day for 24 weeks
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).
Time frame: Baseline through week 48
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: 1\. 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
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
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: 2\. 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
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
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: 3\. 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).
Time frame: Baseline through week 48
Exploratory endpoint
Exploratory endpoint: • The relationship between SOX18-mevalonate pathway-axis activation, as assessed by immunofluorescent staining of available infantile hemangioma tissue, and clinical response through Week 24 and regrowth status at Week 48.
Time frame: Baseline through week 48
Plan to share: No
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Joyce Teng