Comparing different doses of sirolimus for treating kaposiform hemangioendothelioma
Different Doses of Sirolimus for the Maintenance Treatment of Kaposiform Hemangioendothelioma: a Randomized Controlled Trial
This study is testing different doses of sirolimus to see which one works best and is safest for treating kaposiform hemangioendothelioma in infants and young children.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | N/A to 14 Years |
| Sex | All |
| Sponsor | West China Hospital Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Chengdu, Sichuan) |
| Trial ID | NCT05324384 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the efficacy and safety of varying doses of sirolimus in the maintenance treatment of kaposiform hemangioendothelioma (KHE), a rare and aggressive vascular tumor primarily affecting infants and young children. The research will focus on optimizing sirolimus blood concentrations to enhance treatment effectiveness while minimizing adverse effects, such as oral mucositis. By comparing lower and higher doses, the study seeks to establish a more effective treatment protocol for KHE, which currently lacks high-level evidence for low-dose maintenance therapy.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 0 to 14 years diagnosed with kaposiform hemangioendothelioma.
Not a fit: Patients with contraindications to sirolimus or those who have recently undergone other treatments may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved treatment protocols for children with kaposiform hemangioendothelioma, enhancing their quality of life and treatment outcomes.
How similar studies have performed: Previous studies have indicated good efficacy of low-dose sirolimus for KHE, but this specific approach is novel and lacks high-level evidence.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Chengdu, Sichuan
- West China Hospital of Sichuan University — Chengdu, Sichuan, China (Recruiting)
Study contacts
- Principal investigator: Yi Ji, MD, PhD — West China Hospital
- Study coordinator: Yi Ji, MD, PhD
- Email: jijiyuanyuan@163.com
- Phone: 86 28 85423453
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.