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Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP

NCT07656909 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 76

Last updated 2026-06-18

No results posted yet for this study

Summary

This randomized clinical trial evaluates if low-dose sirolimus (target trough 4-8 ng/mL) combined with prednisolone is noninferior in efficacy but superior in safety compared to standard high-dose sirolimus (target trough 10-15 ng/mL) combined with prednisolone in pediatric patients with kaposiform hemangioendothelioma and Kasabach-Merritt phenomenon (KHE with KMP), with participants randomized 1:1 to receive the assigned regimen, undergo routine blood and imaging monitoring, and be evaluated for clinical response and adverse events.

Conditions

  • Kaposiform Hemangioendothelioma (KHE)
  • Kasabach Merritt Phenomenon

Interventions

DRUG

Sirolimus (RAPAMUNE)

Participants will receive oral sirolimus in combination with prednisolone. Patients will be randomized in a 1:1 ratio to either a low-dose sirolimus group, with dose adjustments to maintain a target plasma trough concentration of 4-8 ng/mL, or a high-dose sirolimus group, with dose adjustments to maintain a target plasma trough concentration of 10-15 ng/mL. Prednisolone will be administered according to the study protocol and tapered based on clinical response. The total treatment duration will be 12 months.

Sponsors & Collaborators

  • Yi Ji

    lead OTHER

Principal Investigators

  • Yi Ji · West China Hospital

  • Jiangyuan Zhou, MD · West China Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
1 Day
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-16
Primary Completion
2027-12-31
Completion
2028-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07656909 on ClinicalTrials.gov