Topical Simvastatin for Treating Infantile Hemangioma (TSTIH)

Joyce Teng

Status and phase: Begins enrollment this month; Early Phase 1

Conditions: Hemangioma Skin

Treatments: Drug: 5% simvastatin ointment

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT06273111

73840

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 3 to 6 months old

Volunteers: No Healthy Volunteers

Inclusion criteria

Participants may be included in the study only if they meet all of the following criteria:

1. Healthy children aged between 3-6 months without medical disorders that contradict topical statin treatment.
2. 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

Exclusion criteria

Participants meeting any of the following criteria will not be eligible to participate in the study:

1. IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.

2. IH with active ulceration at screening visit.

3. IH to be treated involving the lips mainly.

4. IH with high-risk criteria that need systemic propranolol treatment to avoid the delay with standard treatment

5. Participants with concurrent skin conditions that may impede accurate clinical assessment of the IH.

6. Participants with hereditary or metabolic disorders requiring systemic statin therapy.

7. Participants who are allergic to statins, or other ingredients present in the topical medication.

8. 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

15 participants in 1 patient group

5% simvastatin ointment

Experimental group

Description:

Participants will be applied 5% simvastatin ointment on IH lesion

Treatment:

Drug: 5% simvastatin ointment