Topical Simvastatin for Treating Infantile Hemangioma (TSTIH)

A Pilot Study Using Topical Statins to Treat Children With Infantile Hemangioma

This is a 24-week, open-label pilot study to evaluate the safety and preliminary efficacy of 5% simvastatin ointment in treating 12 children with superficial IH.

The primary objective:

To evaluate the safety and tolerability of topical treatment with 5% simvastatin ointment for superficial IH over 24 weeks.

The secondary objective:

1.1 To evaluate the efficacy of 5% simvastatin ointment when topical treatment is administered twice daily for 24 weeks. 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.

Study Overview

• Status: Not yet recruiting
• Conditions: Hemangioma Skin
• Intervention / Treatment: Drug: 5% simvastatin ointment

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Joyce Teng, MD, PhD; Phone Number: 650-724-9627; Email: jteng3@stanford.edu
• Study Contact Backup: Name: Ramrada Lekwuttikarn, MD; Phone Number: 650-313-8207; Email: ramrada1@stanford.edu

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
      • Contact: Thomas Buschbacher; Phone Number: 352-278-7603; Email: tbusch@stanford.edu
      • Contact: Ramrada Lekwuttikarn, MD; Phone Number: 650-313-8207; Email: ramrada1@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Healthy children aged between 3 months and 5 years.
• Newly diagnosed superficial IH or experiencing rebound growth with a superficial component.
• Participants must possess at least one IH lesion with the longest diameter equal to or greater than 2 cm, located on any part of the body except the lips.

• Participants must not have received any of the following treatments for their IH:

  • Topical medical therapy within the past 2 weeks.
  • Systemic medical therapy within the past 3 months.
  • Laser treatment within the past 6 weeks.
• Written informed consent from the parent(s)/guardian(s) of minor participants must be obtained before any study procedure is performed.

Exclusion Criteria:

• IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.
• IH with active ulceration.
• IH to be treated involving the lips mainly.
• Participants with concurrent skin conditions that may impede accurate clinical assessment of the IH.
• Participants with hereditary or metabolic disorders requiring systemic statin therapy.
• Participants who are allergic to statins, or other ingredients present in the topical medication.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 5% simvastatin ointment; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Treatment-Related Adverse Events	Baseline through week 24

Secondary Outcome Measures

Outcome Measure	Time Frame
Percentage of participants achieved a 50% HAS reduction at week 12 compared to baseline	Baseline through week 12
Percentage of participants achieved a 75% HAS reduction from baseline by the end of the study (week 24) compared to baseline.	Baseline through week 24
Percentage of targeted hemangiomas with complete or nearly complete resolution (IGA 0 or 1), defined as a minimal degree of telangiectasia, skin thickening, and no definitive palpable cutaneous texture changes.	Baseline through week 24
Percentage of targeted hemangiomas that are stabilized (no noticeable change to baseline) or continue to progress.	Baseline through week 24
Percentage of participants with a significant improvement in quality-of-life, defined as > 50% reduction in the IH-QoL questionnaire score from baseline to week 24.	Baseline through week 24

Collaborators and Investigators

• Sponsor: Joyce Teng
• Collaborators: Stanford University
• Investigators: Principal Investigator: Joyce Teng, MD, PhD, Stanford University

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): July 31, 2025
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: February 15, 2024
• First Submitted That Met QC Criteria: February 15, 2024
• First Posted (Actual): February 22, 2024

Study Record Updates

• Last Update Posted (Actual): February 22, 2024
• Last Update Submitted That Met QC Criteria: February 15, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Skin Abnormalities; Neoplasms, Vascular Tissue; Hemangioma, Capillary; Port-Wine Stain; Hemangioma; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites; Anticholesteremic Agents; Hypolipidemic Agents; Lipid Regulating Agents; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Simvastatin
• Other Study ID Numbers: 73840

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No