영아 혈관종 치료를 위한 국소 심바스타틴 (TSTIH)

실험적: 5% 심바스타틴 연고

참가자는 IH 병변에 5% 심바스타틴 연고를 바르게 됩니다.

의약품: 5% 심바스타틴 연고

5% 심바스타틴 연고를 24주 동안 하루 2회 IH 병변에 직접 도포합니다.

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).

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

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

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).

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.