Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma

NCT ID: NCT07131644

Last Updated: 2025-09-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-13

Study Completion Date

2028-02-01

Brief Summary

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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.

Detailed Description

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Conditions

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Kaposiform Hemangioendothelioma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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Direct discontinuation group

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

Group Type PLACEBO_COMPARATOR

Sirolimus

Intervention Type DRUG

Comparison of different sirolimus discontinuation strategies.

Low-dose treatment group

Low-dose sirolimus treatment was administered for 6 months (with blood levels maintained at 2-5 ng/mL), followed by gradual tapering and discontinuation over 2 months.

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Comparison of different sirolimus discontinuation strategies.

Intermittent treatment group

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.

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Comparison of different sirolimus discontinuation strategies.

Interventions

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Sirolimus

Comparison of different sirolimus discontinuation strategies.

Intervention Type DRUG

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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West China Hospital

OTHER

Sponsor Role lead

Responsible Party

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Yi Ji

MD, PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Yi Ji, PhD

Role: PRINCIPAL_INVESTIGATOR

Sichuan University West China Hospital

Locations

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West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status

Countries

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China

Central Contacts

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Yi Ji, PhD

Role: CONTACT

+862885423453

Jiangyuan Zhou, Doctor

Role: CONTACT

+8613668491160

Facility Contacts

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Yi Ji, PhD

Role: primary

+862885423453

Jiangyuan Zhou, Doctor

Role: backup

References

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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.

Reference Type BACKGROUND
PMID: 40107509 (View on PubMed)

Zhou 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.

Reference Type BACKGROUND
PMID: 36916140 (View on PubMed)

Ji 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.

Reference Type BACKGROUND
PMID: 35030255 (View on PubMed)

Zhou 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.

Reference Type BACKGROUND
PMID: 34273457 (View on PubMed)

Zhou 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.

Reference Type BACKGROUND
PMID: 39831682 (View on PubMed)

Other Identifiers

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20250611

Identifier Type: -

Identifier Source: org_study_id

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