Sirolimus Withdrawal Strategies for Kaposiform Hemangioendothelioma
Official title: A Randomized Controlled Trial Comparing Three Sirolimus Discontinuation Strategies in Patients With Kaposiform Hemangioendothelioma
Run by West China Hospital · NCT07131644 · Data updated 2026-11-12
This study tests how to safely stop sirolimus in people with Kaposiform Hemangioendothelioma to see if disease stays controlled after withdrawal.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Sex | All |
| Enrollment | 120 (estimated) |
| Sponsor | West China Hospital Academic / other |
| Locations | 1 site (Chengdu, Sichuan) |
| Status | Recruiting |
| Started | September 13, 2025 |
| Main results expected | September 1, 2027 |
| Study ends | February 1, 2028 |
| Registry record updated | September 25, 2026 |
| Trial ID | NCT07131644 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are KHE patients who have previously received sirolimus and are ready to discontinue treatment, with evaluable disease activity and the ability to adhere to follow-up.
✕Probably not a fit
Patients who are not candidates for discontinuation or who have ongoing active disease may not benefit from withdrawal strategies.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
In this phase 2 study, researchers will explore different approaches to discontinue sirolimus in patients with Kaposiform Hemangioendothelioma who are ready for withdrawal. Participants will be monitored with clinical exams and imaging to detect any rebound growth or recurrence after stopping therapy. The trial aims to identify a withdrawal plan that maintains disease control while reducing or avoiding ongoing medication. Safety monitoring includes regular checks of blood counts and organ function per protocol. Conducted at West China Hospital, the study seeks to provide guidance on discontinuation strategies for this condition.
Why it matters
Potential benefit: If successful, the withdrawal strategy could help patients maintain disease control with fewer months on sirolimus.
How similar studies have gone: Prior experiences with sirolimus in KHE show relief of disease but rebound after stopping, so withdrawal strategies are a logical next step though this exact approach is novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
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West China Hospital of Sichuan University
Chengdu, Sichuan, China Recruiting
Study contacts
- Principal investigator: Yi Ji, PhD — Sichuan University West China Hospital
- Study coordinator: Yi Ji, PhD
- Email: jijiyuanyuan@163.com
- Phone: +862885423453
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Low- vs High-Dose Sirolimus With Prednisolone for KHE/KMP in Children
This trial tests whether a lower dose of sirolimus with prednisolone works as well as a higher dose with prednisolone for kids with Kaposiform Hemangioendothelioma and Kasabach-Merritt phenomenon.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.