Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma

NCT07131644 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: 20250611
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

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.

Conditions

Kaposiform Hemangioendothelioma

Keywords

Kaposiform Hemangioendothelioma; Sirolimus; Discontinuation Strategies

Outcome Measures

Primary Outcomes (1)

• Rebound growth rate of KHE

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

  12 months

Secondary Outcomes (2)

• Incidence of disease sequelae

  12 months

• Incidence of adverse events throughout the study.

  12 months

Study Arms (3)

Direct discontinuation group

PLACEBO COMPARATOR

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

Drug: Sirolimus

Low-dose treatment group

EXPERIMENTAL

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.

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.

Drug: Sirolimus

Interventions

Sirolimus DRUG

Comparison of different sirolimus discontinuation strategies.

Direct discontinuation group; Intermittent treatment group; Low-dose treatment group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• West China Hospital: lead

Study Sites (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

Related Publications (5)

• 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.

  PMID: 40107509 BACKGROUND

• 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.

  PMID: 36916140 BACKGROUND

• 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.

  PMID: 35030255 BACKGROUND

• 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.

  PMID: 34273457 BACKGROUND

• 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.

  PMID: 39831682 BACKGROUND

MeSH Terms

Conditions

Kaposiform Hemangioendothelioma

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Yi Ji, PhD

  Sichuan University West China Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yi Ji, PhD

CONTACT

+862885423453; jijiyuanyuan@163.com

Jiangyuan Zhou, Doctor

CONTACT

+8613668491160; 13668491160@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: August 13, 2025
• First Posted: August 20, 2025
• Study Start: September 13, 2025
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): February 1, 2028
• Last Updated: September 8, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)