Topical Simvastatin for Treating Infantile Hemangioma

NCT06273111 | Not Yet Recruiting Early Phase 1 FDA Drug

• 1 other identifier: 73840
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemangioma Skin

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Related Adverse Events

  Baseline through week 24

Secondary Outcomes (5)

• 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

Study Arms (1)

5% simvastatin ointment

EXPERIMENTAL

Participants will be applied 5% simvastatin ointment on IH lesion

Drug: 5% simvastatin ointment

Interventions

5% simvastatin ointment DRUG

5% simvastatin ointment will be applied directly on IH lesion twice per day for 24 weeks

5% simvastatin ointment

Eligibility Criteria

• Age: 3 Months - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Joyce Teng: lead
• Stanford University: collaborator

Study Sites (1)

Stanford University

Palo Alto, California, 94304, United States

Location

Study Officials

• Joyce Teng, MD, PhD

  Stanford University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Joyce Teng, MD, PhD

CONTACT

650-724-9627; jteng3@stanford.edu

Ramrada Lekwuttikarn, MD

CONTACT

650-313-8207; ramrada1@stanford.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Dermatology and Pediatric

Study Record Dates

• First Submitted: February 15, 2024
• First Posted: February 22, 2024
• Study Start: April 1, 2024
• Primary Completion: July 31, 2025
• Study Completion: December 31, 2025
• Last Updated: February 22, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)