Kaposiform HemangioendotheliomaPhase 2NCT07131644
Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma
A Phase 2 study testing Sirolimus in people with Kaposiform Hemangioendothelioma, run by West China Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Kaposiform Hemangioendothelioma
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 1 siteChina
- Study dates
- 13 Sep 2025to 1 Feb 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Patients who are unable to comply with the follow-up or treatment schedule, potentially affecting the integrity of study data.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041
Yi Ji, PhD, +862885423453, jijiyuanyuan@163.com
Jiangyuan Zhou, Doctor, 13668491160@163.com
About this study
Official title: A Randomized Controlled Trial Comparing Three Sirolimus Discontinuation Strategies in Patients With Kaposiform Hemangioendothelioma
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.
- Conditions
- Kaposiform Hemangioendothelioma
- Being tested
- Sirolimus
- Keywords
- Kaposiform Hemangioendothelioma, Sirolimus, Discontinuation Strategies
- Medical subject headings
- Kaposiform Hemangioendothelioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Comparison of different sirolimus discontinuation strategies.
| Study group | Role | What they receive |
|---|---|---|
| Direct discontinuation group | Placebo comparator | Sirolimus was gradually tapered and discontinued over a 2-month period. Receives: Drug: Sirolimus |
| 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. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rebound growth rate of KHE
The primary endpoint is the rebound growth rate of KHE during the follow-up period.
Measured: 12 months
Secondary outcomes (2)
Incidence of disease sequelae
Incidence of disease sequelae, assessed by the number of participants who develop clinically confirmed sequelae during the follow-up period, as determined through questionnaires and clinical plus imaging evaluations.
Measured: 12 months
Incidence of adverse events throughout the study.
Incidence of adverse events during the follow-up period.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2025
- Study start13 Sep 2025
- Primary completion1 Sep 2027 (estimated)
- Study completion1 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Kaposiform HemangioendotheliomaStudies of the same condition, open and under way.
- More trials run by West China HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- West China Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yi Ji, West China Hospital
- Investigators
- Yi Ji, PhD, Principal investigator, Sichuan University West China Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07131644
- Sponsor's protocol ID
- 20250611
- Status last verified
- Sep 2026
- First submitted
- 13 Aug 2025
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zhou J, Lan Y, Qiu T, Zhang Z, Gong X, Zhang X, Yang C, Zhou Z, Zhang Y, Yang M, Fu J, He C, Peng Q, Hu F, Xia C, Kong F, Chen S, Ji Y. Efficacy and safety of high-vs low-dose sirolimus in patients with kaposiform hemangioendothelioma: A randomized clinical trial. J Am Acad Dermatol. 2025 Jul;93(1):124-131. doi: 10.1016/j.jaad.2025.03.023. Epub 2025 Mar 17.
PubMed 40107509Zhou J, Li Y, Qiu T, Gong X, Yang K, Zhang X, Zhang Z, Lan Y, Hu F, Peng Q, Zhang Y, Kong F, Chen S, Ji Y. Long-term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges. Int J Cancer. 2023 Aug 1;153(3):600-608. doi: 10.1002/ijc.34509. Epub 2023 Mar 22.
PubMed 36916140Ji Y, Chen S, Zhou J, Yang K, Zhang X, Xiang B, Qiu T, Gong X, Zhang Z, Lan Y, Hu F, Kong F, Qiu Q, Zhang Y. Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial. Blood. 2022 Mar 17;139(11):1619-1630. doi: 10.1182/blood.2021014027.
PubMed 35030255Zhou J, Yang K, Dai S, Qiu T, Zhang X, Gong X, Chen S, Ji Y. Clinical features and management of kaposiform hemangioendothelioma and tufted angioma: Similarities and differences. J Am Acad Dermatol. 2022 Jul;87(1):172-174. doi: 10.1016/j.jaad.2021.07.012. Epub 2021 Jul 14. No abstract available.
PubMed 34273457Zhou J, Qiu T, Zhang Z, Lan Y, Huo R, Xiang B, Chen S, Qiu L, Xia C, Xu X, Li J, Ma Y, Yao W, Wang Z, Dong C, Qin Z, Tai M, Guo L, He X, Gu S, Li L, Hou F, Cai Y, Wang H, Wang J, Jiang X, Zheng J, Li K, Ji Y. Consensus statement for the diagnosis, treatment, and prognosis of kaposiform hemangioendothelioma. Int J Cancer. 2025 May 15;156(10):1986-1994. doi: 10.1002/ijc.35344. Epub 2025 Jan 20.
PubMed 39831682
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Trial Comparing Three Sirolimus Discontinuation Strategies in Patients With Kaposiform Hemangioendothelioma
- ClinicalTrials.gov ID
- NCT07131644
- Acronym / sponsor ID
- 20250611
- Conditions
- Kaposiform Hemangioendothelioma
- Keywords
- Kaposiform Hemangioendothelioma, Sirolimus, Discontinuation Strategies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- West China HospitalUniversity, hospital or other
- Study start
- 13 Sep 2025 (actual)
- Primary completion
- 1 Sep 2027 (estimated)
- Study completion
- 1 Feb 2028 (estimated)
- First posted
- 20 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Direct discontinuation group | Placebo comparator | Drug: SirolimusSirolimus was gradually tapered and discontinued over a 2-month period. |
| Intermittent treatment group | Experimental | Drug: SirolimusSirolimus was administered using a 3-days-on/4-days-off weekly schedule for 6 months, followed by gradual tapering and discontinuation over 2 months. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- Patients who are unable to comply with the follow-up or treatment schedule, potentially affecting the integrity of study data.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusComparison of different sirolimus discontinuation strategies. | Drug | Direct discontinuation group, Intermittent treatment group |
Primary outcome
Rebound growth rate of KHE
Time frame: 12 months
The primary endpoint is the rebound growth rate of KHE during the follow-up period.
2 secondary outcomes
Incidence of disease sequelae
Time frame: 12 months
Incidence of adverse events throughout the study.
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
- Results publication: PubMed 40107509Zhou J, Lan Y, Qiu T, Zhang Z, Gong X, Zhang X, Yang C, Zhou Z, Zhang Y, Yang M, Fu J, He C, Peng Q, Hu F, Xia C, Kong F, Chen S, Ji Y. Efficacy and safety of high-vs low-dose sirolimus in patients with kaposiform hemangioendothelioma: A randomized clinical trial. J Am Acad Dermatol. 2025 Jul;93(1):124-131. doi: 10.1016/j.jaad.2025.03.023. Epub 2025 Mar 17.
- Results publication: PubMed 36916140Zhou J, Li Y, Qiu T, Gong X, Yang K, Zhang X, Zhang Z, Lan Y, Hu F, Peng Q, Zhang Y, Kong F, Chen S, Ji Y. Long-term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges. Int J Cancer. 2023 Aug 1;153(3):600-608. doi: 10.1002/ijc.34509. Epub 2023 Mar 22.
- Results publication: PubMed 35030255Ji Y, Chen S, Zhou J, Yang K, Zhang X, Xiang B, Qiu T, Gong X, Zhang Z, Lan Y, Hu F, Kong F, Qiu Q, Zhang Y. Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial. Blood. 2022 Mar 17;139(11):1619-1630. doi: 10.1182/blood.2021014027.
- Results publication: PubMed 34273457Zhou J, Yang K, Dai S, Qiu T, Zhang X, Gong X, Chen S, Ji Y. Clinical features and management of kaposiform hemangioendothelioma and tufted angioma: Similarities and differences. J Am Acad Dermatol. 2022 Jul;87(1):172-174. doi: 10.1016/j.jaad.2021.07.012. Epub 2021 Jul 14. No abstract available.
- Results publication: PubMed 39831682Zhou J, Qiu T, Zhang Z, Lan Y, Huo R, Xiang B, Chen S, Qiu L, Xia C, Xu X, Li J, Ma Y, Yao W, Wang Z, Dong C, Qin Z, Tai M, Guo L, He X, Gu S, Li L, Hou F, Cai Y, Wang H, Wang J, Jiang X, Zheng J, Li K, Ji Y. Consensus statement for the diagnosis, treatment, and prognosis of kaposiform hemangioendothelioma. Int J Cancer. 2025 May 15;156(10):1986-1994. doi: 10.1002/ijc.35344. Epub 2025 Jan 20.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yi Ji, PhD principal investigator
Sichuan University West China Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Kaposiform Hemangioendothelioma, run by West China Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yi Ji, PhD (+862885423453, jijiyuanyuan@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rebound growth rate of KHE (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07131644, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026.