CompletedPhase 2

Efficacy and Safety of Different Concentrations of Sirolimus in the Treatment of Kaposiform Hemangioendothelioma.

China79 participantsStarted 2021-02-17

Plain-language summary

The purpose of this study is to compare the efficacy and safety of different concentration gradients of sirolimus in the treatment of Kaposiform hemangioendothelioma.

Who can participate

Age range1 Day – 14 Years

SexALL

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Inclusion criteria

✓. Male and female;
✓. Between 0 and 14 years of age;
✓. KHE diagnosis was confirmed by local investigators and by consensus of our multidisciplinary vascular anomaly group at the West China Hospital of Sichuan University based on:
✓. Without KMP, which was defined as a platelet count of less than 100×10\^9/L, with consumptive coagulopathy and hypofibrinogenemia.
✓. Patients were required to have adequate liver, renal and bone marrow function, and absence of active infection
✓. Consent of parents (or the person with parental authority in families): signed and dated written informed consent.

Exclusion criteria

✕. Patients contraindicated for the administration of sirolimus (e.g., those with an allergy to sirolimus or other rapamycin analog)
✕. Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;
✕. Patients had a history of a major surgery within 2 weeks before enrollment;
✕. Patients who have a history of treatment with sirolimus or other mTOR inhibitor;
✕. Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;
✕. Concurrent severe and/or uncontrolled medical diseases that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
✕. Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
✕. Patients with inadequate liver function:

What they're measuring

The proportion of patients achieving an objective response at month 12

Timeframe: 12 months

Trial details

NCT IDNCT04775173

SponsorWest China Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-08-10

View on ClinicalTrials.gov More Kaposiform Hemangioendothelioma trials