Topical sirolimus for treating superficial vascular anomalies
Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial
This study is testing a new skin treatment using sirolimus to see if it works better than taking it by mouth for people with certain complicated vascular growths.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 75 (estimated) |
| Ages | 0 Years to 18 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai) |
| Trial ID | NCT04921722 on ClinicalTrials.gov |
What this trial studies
This study investigates the safety and efficacy of percutaneous administration of sirolimus for treating superficial complicated vascular anomalies, including Kaposiform Hemangioendothelioma and Tufted Angioma. Sirolimus, an mTOR inhibitor, is known for its anti-proliferative and anti-angiogenic properties, making it a promising treatment option. The study aims to assess how well this topical treatment works compared to oral sirolimus, particularly in patients with residual skin manifestations after previous treatments. Participants will be closely monitored for both efficacy and safety throughout the trial.
Who should consider this trial
Good fit: Ideal candidates include children diagnosed with Kaposiform Hemangioendothelioma, Tufted Angioma, or other complicated superficial vascular anomalies who have limited superficial lesions.
Not a fit: Patients with severe complications such as Kasabach-Merritt Phenomenon or those with significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the appearance and psychological well-being of patients with superficial vascular anomalies.
How similar studies have performed: Previous studies have shown that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma, indicating a promising approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components. 1. The case is initial, with a relatively limited superficial lesion. 2. The participant has residual surface lesions after oral medication. * Participant with no use of other medication or surgical treatment * Participant with detailed medical records of the disease at the time of screening * Participant with signed and dated informed consent from the guardian(s) Exclusion Criteria: * Participants with Kasabach-Merritt Phenomenon, with platelets \<50×10 9 /L. * Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency. * Participants with other hematological diseases or solid tumor. * Participants allergic to sirolimus or dressing.
Where this trial is running
Shanghai
- Children's Hospital of Fudan University — Shanghai, China (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.