Different Doses of Sirolimus for the Maintenance Treatment of Kaposiform Hemangioendothelioma

Launched by WEST CHINA HOSPITAL · Apr 5, 2022

Keywords

ClinConnect Summary

This clinical trial is studying how effective and safe different doses of a medication called sirolimus are for maintaining treatment in children with a rare condition called kaposiform hemangioendothelioma (KHE), a type of blood vessel tumor. The study is currently looking for participants who are between 0 and 14 years old and have been diagnosed with KHE through specific tests, such as a biopsy or MRI. To be eligible, children must have good liver, kidney, and bone marrow function, and their parents or guardians must give written consent for them to join the study.

During the trial, participants will receive different doses of sirolimus and will be monitored to see how well the treatment works and whether they experience any side effects. It’s important to note that children who have certain health issues, recent treatments, or infections may not be able to participate. This trial could help find the best way to manage KHE in young patients, potentially leading to better treatment options in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• Presenting a KHE with the following characteristics:
• • 1. Male and female;
• • 2. Between 0 and 14 years of age;
• 3. Diagnosis of KHE as determined by:
• • Biopsy;
• • Compatible MRI findings;
• • History and clinical features.
• • 4. Patients were required to have adequate liver, renal and bone marrow function, and absence of active infection
• • 5. Consent of parents (or the person with parental authority in families): signed and dated written informed consent.
• Exclusion Criteria:
• • 1. Patients contraindicated for the administration of sirolimus (e.g., those with an allergy to sirolimus or other rapamycin analog)
• • 2. Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;
• • 3. Patients had a history of a major surgery within 2 weeks before enrollment;
• • 4. Patients who have a history of treatment with sirolimus or other mTOR inhibitor;
• • 5. Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;
• • 6. 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).
• • 7. Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
• 8. Patients with inadequate liver function:
• • Total bilirubin higher than or equal to 1.5 × the upper limit of the normal (ULN) for age and alanine aminotransferase and aspartate aminotransferase higher than or equal to 2.5 × the ULN for age.
• 9. Patients with inadequate renal function:
• • 0-5 years of age maximum serum creatinine (mg/dL) of 0.8; 6-10 years of age maximum serum creatinine (mg/dL) of 1.0; 11-14 years of age maximum serum creatinine (mg/dL) of 1.2;
• 10. Adequate bone marrow function:
• • Absolute neutrophil count lower than 1 × 109/L;
• • 11. History of a malignancy within 5 years;
• • 12. HIV infection or known immunodeficiency;
• • 13. Indication for treatment with corticosteroids, vincristine, interferon-α, sirolimus, or tacrolimus for an indication other than IH;
• • 14. Patients with an inability to participate in or follow-up during the study treatment and assessment plan;
• • 15. Inability to give informed consent.