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NCT03188068

Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma

Completed Phase 2 Last updated 21 April 2022
What this trial tests

Phase 2 trial testing Sirolimus in Kaposiform Hemangioendothelioma in 30 participants. Completed in 31 December 2021.

Timeline
1 June 2017
Primary endpoint
31 December 2021
31 December 2021

Quick facts

Lead sponsorWest China Hospital
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment30
Start date1 June 2017
Primary completion31 December 2021
Estimated completion31 December 2021
Sites1 location across China

Drugs / interventions tested

Conditions studied

Sponsor

West China Hospital

Who can join

Adults 1 Day to 18, any sex, with Kaposiform Hemangioendothelioma or Kasabach Merritt Phenomenon. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm that occurs predominantly in infancy or early childhood. KHE has a nearly equal sex ratio. The annual incidence of KHE has been estimated at 0.071 per 100,000 children. KHE presents with intermediate-malignant and locally aggressive characteristics but without distant metastases. This pilot trial studies sirolimus versus sirolimus plus pednisolone in treating patients diagnosed with kaposiform hemangioendothelioma (KHE) and Kasabach-Merritt phenomemon (KMP) that cannot be removed by surgery. The purpose of this study is to compare the efficacy and safety of orally administered sirolimus versus sirolimus plus pednisolone in the treatment of KHE associated with KMP.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Kaposiform hemangioendothelioma: current knowledge and future perspectives.
    Ji Y, Chen S, Yang K, Xia C, et al · · 2020 · cited 103× · PMID 32014025 · DOI 10.1186/s13023-020-1320-1
  2. Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial.
    Ji Y, Chen S, Zhou J, Yang K, et al · · 2022 · cited 64× · PMID 35030255 · DOI 10.1182/blood.2021014027
  3. The role of metabolic ecosystem in cancer progression - metabolic plasticity and mTOR hyperactivity in tumor tissues.
    Sebestyén A, Dankó T, Sztankovics D, Moldvai D, et al · · 2021 · cited 35× · PMID 35029792 · DOI 10.1007/s10555-021-10006-2
  4. Hypoxia-driven angiogenesis and metabolic reprogramming in vascular tumors.
    Liu L, Yu J, Liu Y, Xie L, et al · · 2025 · cited 6× · PMID 40443737 · DOI 10.3389/fcell.2025.1572909
  5. mTOR hyperactivity and <i>RICTOR</i> amplification as targets for personalized treatments in malignancies.
    Sztankovics D, Moldvai D, Petővári G, Dankó T, et al · · 2024 · cited 4× · PMID 38515456 · DOI 10.3389/pore.2024.1611643

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