Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies

NCT04921722 | Recruiting Phase 4

• 1 other identifier: LK210106
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.

Conditions

Kaposiform Hemangioendothelioma; Tufted Angioma; Superficial Vascular Anomalies; Superficial Lymphatic Malformations

Outcome Measures

Primary Outcomes (1)

• Effective rate

  Achauer BM et al. made the four-level standard as follows. Grade I: tumor size and skin lesion color regression ≤ 25%; grade II: tumor size and skin lesion color regression 25%-50%; grade III: tumor size and skin lesion color regression 50-75%; grade IV: tumor size and skin lesion color regression ≥75%. After 6 months of treatment, the pzrticipant will be evaluated. Grade I will be viewed as invalid. Grade II and grade III will be viewed as effective, and grade IV will be viewed as very effective. Those in grade Ⅱ, Ⅲ or Ⅳ will be calculated in effectiveness rate.

  From admission to follow-up six months

Secondary Outcomes (4)

• Changes of resistance coefficient

  From admission to follow-up six months

• Incidence of adverse events

  From admission to follow-up six months

• Platelet count

  From admission to follow-up six months

• Changes of peak blood flow

  From admission to follow-up six months

Study Arms (2)

Topical use of sirolimus

EXPERIMENTAL

Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.

Drug: Percutaneous sirolimus

Oral use of sirolimus

ACTIVE COMPARATOR

Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.

Drug: Oral sirolimus

Interventions

Percutaneous sirolimus DRUG

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In experimental group, we administrate percutaneous sirolimus. Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.

Also known as: Rapamycin

Topical use of sirolimus

Oral sirolimus DRUG

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In active comparator group, we administrate oral sirolimus. Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.

Also known as: Rapamycin

Oral use of sirolimus

Eligibility Criteria

• Age: 0 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.
• The case is initial, with a relatively limited superficial lesion.
• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, 210012, China

RECRUITING

MeSH Terms

Conditions

Kaposiform Hemangioendothelioma; Tufted angioma

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2021
• First Posted: June 10, 2021
• Study Start: October 22, 2021
• Primary Completion: November 1, 2025
• Study Completion: November 1, 2025
• Last Updated: March 15, 2024

Record last verified: 2024-03

Locations

CN (1)