Topical Simvastatin for Hemangioma

(TSTIH Trial)

Overseen By Joyce Teng, MD, PhD

Age: < 18

Sex: Any

Trial Phase: Phase < 1

Sponsor: Joyce Teng

Must not be taking: Statins

Disqualifiers: Subcutaneous IH, Ulceration, Skin conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

12 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must not have used topical treatments in the past 2 weeks or systemic treatments in the past 3 months for your hemangioma.

What data supports the effectiveness of the drug Simvastatin for treating hemangioma?

Research shows that simvastatin, when applied topically, can help with wound healing by reducing inflammation and promoting tissue regeneration. This suggests it might also be effective in treating hemangiomas, which are growths of blood vessels. ^{1 2 3 4 5}

Is topical simvastatin safe for use in humans?

Simvastatin, when used for lowering cholesterol, has been associated with skin reactions like rashes and eczema in some people. However, in a study of its use for wound healing, it was found to be effective with few adverse events reported. ^{2 3 6 7 8}

How is the drug simvastatin unique in treating hemangioma?

Simvastatin is unique for treating hemangioma because it is used topically, which means it is applied directly to the skin, unlike many other treatments that are taken orally. This method may help target the affected area more directly and reduce potential side effects associated with oral medications. ^{2 3 9 10 11}

What is the purpose of this trial?

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.

Research Team

Joyce Teng, MD, PhD

Principal Investigator

Stanford University

Eligibility Criteria

This trial is for children with superficial infantile hemangioma (IH), which are birthmarks that may look like rubbery bumps and can be red or purple. Participants should not have other skin conditions affecting the same area, nor should they be on medications that could interfere with the study.

Inclusion Criteria

Written informed consent from the parent(s)/guardian(s) of minor participants must be obtained before any study procedure is performed

I haven't had any treatments for IH in the specified time frames.

My child is healthy and between 3 months and 5 years old.

Exclusion Criteria

I have an active ulcer.

My IH is mostly under the skin with little surface skin involvement.

I have skin conditions that could affect skin cancer assessments.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 5% simvastatin ointment applied topically twice daily for 24 weeks

24 weeks

Regular clinic visits for assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Simvastatin

Trial Overview The trial tests a cream containing 5% simvastatin applied twice daily to see if it's safe and can help reduce the size or appearance of IH over a period of 24 weeks. Progress will be checked using photos, an activity score, and quality of life surveys.

Participant Groups

Group I: 5% simvastatin ointment Experimental Treatment 1 Intervention

Participants will be applied 5% simvastatin ointment on IH lesion

Simvastatin is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Zocor for:

• Hypercholesterolemia
• Cardiovascular disease

Approved in United States as Zocor for:

• Hypercholesterolemia
• Cardiovascular disease

Approved in Canada as Zocor for:

• Hypercholesterolemia
• Cardiovascular disease

Approved in Japan as Zocor for:

• Hypercholesterolemia
• Cardiovascular disease

Who Is Running the Clinical Trial?

Joyce Teng

Lead Sponsor

Trials

2

Recruited

70+

Stanford University

Collaborator

Trials

2,527

Recruited

17,430,000+

Findings from Research

In a study involving 438 patients and 100 healthcare providers in South Africa, simvastatin demonstrated significant effectiveness in lowering total cholesterol levels, with a 20.5% reduction for those on 10 mg daily and a 27.6% reduction for those who increased to 20 mg daily.

The safety profile of simvastatin was consistent with premarketing data, indicating it is a safe option for patients, and the study's methodology proved effective for monitoring drug safety and efficacy in the postmarketing phase.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Zocor, the postmarketing experience. Smart, AJ., Walters, L., Marais, AD., et al.[2014]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Topical simvastatin enhances tissue regeneration in full-thickness skin wounds in rat models. [2021]

2. Iran pubmed.ncbi.nlm.nih.gov

Simvastatin-Loaded Nanostructured Lipid Carriers as Topical Drug Delivery System for Wound Healing Purposes: Preparation, Characterization, and In Vivo Histopathological Studies. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Cheilitis due to treatment with simvastatin. [2013]

4. France pubmed.ncbi.nlm.nih.gov

[Simvastatin-induced lichen planus pemphigoides]. [2013]

5. United States pubmed.ncbi.nlm.nih.gov

SACRED: Effect of simvastatin on hepatic decompensation and death in subjects with high-risk compensated cirrhosis: Statins and Cirrhosis: Reducing Events of Decompensation. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

Statins and "chameleon-like" cutaneous eruptions: simvastatin-induced acral cutaneous vesiculobullous and pustular eruption in a 70-year-old man. [2017]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Generalized eczematous skin rash possibly due to HMG-CoA reductase inhibitors. [2018]

8. South Africa pubmed.ncbi.nlm.nih.gov

Zocor, the postmarketing experience. [2014]

9. Iran pubmed.ncbi.nlm.nih.gov

Preparation and Safety Evaluation of Topical Simvastatin Loaded NLCs for Vitiligo. [2021]

10. India pubmed.ncbi.nlm.nih.gov

A Comparison of Betamethasone Valerate 0.1% Cream Twice Daily Plus Oral Simvastatin Versus Betamethasone Valerate 0.1% Cream Alone in the Treatment of Vitiligo Patients. [2022]

11. England pubmed.ncbi.nlm.nih.gov

LDL-cholesterol lowering effect of a generic product of simvastatin compared to simvastatin (Zocor) in Thai hypercholesterolemic subjects -- a randomized crossover study, the first report from Thailand. [2019]