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NCT Number: NCT04077515

Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma

to evaluate the safety and efficacy of Low-dose sirolimus in Kaposiform Hemangioendothelioma in Chinese children by a prospective, randomized open trial.

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Key information

Conditions

Hemangioma Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Kaposiform Hemangioendothelioma Kasabach-Merritt Syndrome Neoplasms Neoplasms by Histologic Type Neoplasms, Vascular Tissue Thrombocytopenia

Age range

Up to 12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Children's Hospital of Fudan University

Shanghai, 210012, China

About this study

The clinically commonly used dose of sirolimus for Kaposiform Hemangioendothelioma is 0.8 mg/m2 administered twice daily, and the blood concentration can be maintained at 10-15 ng/ml according to the pharmacokinetic formula.Related research reports that maintaining low blood concentration of sirolimus is effective in the treatment of certain vascular malformations and hemangioma, and complications are less. In the clinical practice, we found that the blood concentration was maintained at 7-10 ng/ml, and the patients still achieved good results and the chance of infections decreased. Therefore, this clinical trial was designed.In this trial, two different dosing regimens with corresponding blood concentration were designed to compare the safety and efficacy.In the high concentration group, the sirolimus dosage was adjusted monthly to achieve trough levels between 10 and 15 ng/mL(excluding 10 ng/ml), and it is still used at 0.8 mg/m2 administered twice daily.The low concentration group is 7-10 ng/ml (including 10 ng/ml), and the initial use of sirolimus is 0.7mg/m2 administered twice daily.The dose was adjusted according to the formula after two weeks.The follow-up and evaluation were performed according to a strictly established follow-up schedule after taking the drug.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Kaposiform Hemangioendotheliomas with or without Kasabach-Merritt Phenomenon.
  • 0 - 12 years of age at the time of study entry.
  • Male or female.
  • Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.

Exclusion criteria

  • with hematological diseases.
  • with other solid tumors.
  • with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
  • with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment.

Treatment and study plan

Sirolimus(0.8mg/m2)

Drug

The initial use of sirolimus is 0.8mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.

Other names: Rapamycin

Sirolimus(0.7mg/m2)

Drug

The initial use of sirolimus is 0.7mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.

Other names: Rapamycin

Primary outcomes

  1. response to treatment

    Time frame: 1 year after taking the drug

    it is a variable outcome, including complete responses, partial response and no response.

    The definitions are :

    Complete Response：

    • platelets counts is greater than 100×10^9/L.
    • significant volume reduction is greater than 80%.
    • Fibrinogen levels at 2-4g/L.
    • The surface skin of the tumor is lighter or the tumor is softer significantly.

    Partial Response:

    • platelets counts is greater than 40×10^9/L.
    • significant volume reduction is greater than 50%.
    • Fibrinogen levels at less than 50% reduction from baseline.
    • The surface skin of the tumor and palpation of the tumor have no change or less change.

    No Response:

    • platelets counts is less than 40×10^9/L.
    • significant volume reduction is less than 50% or the tumor is bigger.
    • Fibrinogen levels at grater then 50% reduction from baseline.
    • The surface skin of the tumor is darker or the tumor is harder.

Secondary outcomes

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Time frame: 1 year after taking the drug

    Monitoring patient's clinical biochemical indicators and symptoms

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma:A Prospective, Randomized Open Trial

Important dates

Study start
2019
Primary completion
2022
Study completion
2022
First posted
Sep 4, 2019
Registry last updated
Jan 30, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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