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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of a 5% simvastatin ointment for treating children with superficial infantile hemangiomas (IH), which are red birthmarks caused by extra blood vessels. Researchers seek to determine if applying this ointment twice a day for 24 weeks reduces the size and activity of these birthmarks and improves the child’s quality of life. Healthy children between 3 months and 5 years old with a superficial IH lesion at least 2 cm in diameter may qualify. The trial excludes children who have received recent treatments for IH or have other skin conditions that could interfere with results.

As an Early Phase 1 trial, this research focuses on understanding how the treatment works in children, offering participants the chance to be among the first to receive this potentially beneficial therapy.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Earlier studies have found simvastatin ointment to be safe when applied to the skin. One specific study showed that both 2.5% and 5% simvastatin ointments were safe for treating skin conditions. The 5% ointment, used in this trial, proved more effective on thicker skin areas.

While these results are encouraging, this is an early-stage study, indicating the need for more research to confirm the treatment's safety in a larger group. However, since simvastatin is already used in other forms for different conditions, it suggests general safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for hemangiomas, which typically include beta-blockers like propranolol or corticosteroids, simvastatin is a cholesterol-lowering medication that is being explored for its potential effects on blood vessel growth. Researchers are excited about simvastatin ointment because it offers a novel topical approach, potentially reducing systemic side effects associated with oral medications. By applying simvastatin directly to the hemangioma, it targets the condition locally, which could lead to fewer side effects and easier application, especially for infants. This new method of delivery could revolutionize treatment by combining effectiveness with greater safety and convenience.

What evidence suggests that this treatment might be an effective treatment for superficial IH?

Research has shown that statins, drugs often used to lower cholesterol, might help treat infantile hemangiomas (IH), which are non-cancerous growths in children. Studies have found that simvastatin can slow the growth of the cells that form these tumors in lab tests. This trial will evaluate the effectiveness of a 5% simvastatin ointment applied to IH lesions. Early results suggest that simvastatin could be a useful cream treatment for these tumors. Although more research is needed to confirm these findings in people, the initial evidence appears promising. ^{1 2 6 7 8}

Who Is on the Research Team?

Joyce Teng, MD, PhD

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06273111

Topical Simvastatin for Treating 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 ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11957685/

Unveiling the cholesterol-hemangioma axis: a path to new ... The findings suggest that statins could potentially be repurposed to treat children with infantile hemangioma alone or in combination with propranolol.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11957709/

An endothelial SOX18–mevalonate pathway axis enables ... In summary, our data show that simvastatin and atorvastatin inhibit endothelial differentiation of HemSCs in vitro and HemSC vasculogenesis in a preclinical IH ...

4. ctv.veeva.com ctv.veeva.com/study/topical-simvastatin-for-treating-infantile-hemangioma

Topical Simvastatin for Treating Infantile Hemangioma (TSTIH) This is a 24-week, open-label pilot study to evaluate the safety and preliminary efficacy of 5% simvastatin ointment in treating 12 children

5. phacesyndromecommunity.org phacesyndromecommunity.org/post/cholesterol-connection-statins-show-promise-in-treating-infantile-hemangiomas

Statins Show Promise in Treating Infantile Hemangiomas This brand-new study, which found that statins, drugs commonly used to lower cholesterol in adults, might be a new treatment for infantile hemangiomas (IH).

6. withpower.com withpower.com/trial/early-phase-1-hemangioma-3-2024-89b3c

Topical Simvastatin for Hemangioma (TSTIH 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 ...

7. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT06273111/

Trial | NCT06273111 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 ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11484598/

5% Simvastatin Ointment as Treatment for Congenital ... Their case series concluded that 2.5 and 5% simvastatin ointment is safe and effective, with 5% formulation showing better improvement at thicker areas.