Status:

COMPLETED

Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma

Lead Sponsor:

Children's Hospital of Fudan University

Conditions:

Kaposiform Hemangioendothelioma

Kasabach-Merritt Syndrome

Eligibility:

All Genders

Up to 12 years

Phase:

PHASE4

Brief Summary

This randomized controlled trial aims to compare guided discontinuation with maintenance treatment of sirolimus in pediatric patients with KHE.

Detailed Description

Kaposiform Hemangioendothelioma (KHE) is a rare vascular tumor that occurs in infants and children. KHE is characterized by sheets of spindle cells with an infiltrative pattern in the dermis, subcutan...

Eligibility Criteria

Inclusion

  • Participant diagnosed with KHE with or without KMP
  • Participant age 0-12 years
  • Participant with detailed medical records of the disease at the time of screening
  • Participant with at least two years of remission of KHE and no previous toxicity or adverse events
  • Participant with normal liver and kidney function
  • Participant with signed and dated informed consent from the guardian(s)

Exclusion

  • Participant with other hematological diseases
  • Participant with other solid tumor
  • Participant with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
  • Participant with infectious diseases
  • Unwilling participant

Key Trial Info

Start Date :

July 1 2020

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 1 2023

Estimated Enrollment :

30 Patients enrolled

Trial Details

Trial ID

NCT04448873

Start Date

July 1 2020

End Date

July 1 2023

Last Update

September 26 2023

Active Locations (1)

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Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China, 210012