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Clinical Trials/NCT07131644
NCT07131644RecruitingPhase 2

A Randomized Controlled Trial Comparing Three Sirolimus Discontinuation Strategies in Patients With Kaposiform Hemangioendothelioma

West China Hospital2 sites in 1 country120 target enrollmentStarted: September 13, 2025Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Recruiting
Enrollment
120
Locations
2
Primary Endpoint
Rebound growth rate of KHE
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

Sirolimus has demonstrated efficacy in the treatment of kaposiform hemangioendothelioma (KHE); however, a high rate of rebound growth following discontinuation has been reported, highlighting the clinical importance of establishing an appropriate withdrawal regimen.

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Single (Outcomes Assessor)

Eligibility Criteria

Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •KHE patients who have received sirolimus treatment and met the criteria for drug discontinuation.
  • •With evaluable clinical or imaging parameters to monitor disease activity or recurrence.
  • •Patients or their guardians are able to understand the study and provide written informed consent.
  • •Hematologic and hepatic/renal function meet the safety criteria for drug administration.

Exclusion Criteria

  • •Patients who are unable to comply with the follow-up or treatment schedule, potentially affecting the integrity of study data.

Arms & Interventions

Direct discontinuation group

Placebo Comparator

Sirolimus was gradually tapered and discontinued over a 2-month period.

Intervention: Sirolimus (Drug)

Intermittent treatment group

Experimental

Sirolimus was administered using a 3-days-on/4-days-off weekly schedule for 6 months, followed by gradual tapering and discontinuation over 2 months.

Intervention: Sirolimus (Drug)

Outcomes

Primary Outcomes

Rebound growth rate of KHE

Time Frame: 12 months

The primary endpoint is the rebound growth rate of KHE during the follow-up period.

Secondary Outcomes

  • Incidence of disease sequelae(12 months)
  • Incidence of disease sequelae(12 months)
  • Incidence of adverse events throughout the study.(12 months)

Investigators

Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Yi Ji

MD, PhD

West China Hospital

Study Sites (2)

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Sirolimus Discontinuation Strategies in... | Clinical Trial