Using sirolimus to treat cystic lymphatic malformations in children
Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations
This study is testing different doses of sirolimus to see if it can safely help children with cystic lymphatic malformations.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | 1 Year to 18 Years |
| Sex | All |
| Sponsor | West China Hospital Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Chengdu, Sichuan) |
| Trial ID | NCT06673290 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the efficacy and safety of different doses of sirolimus, an immunosuppressant, in treating cystic lymphatic malformations in children aged 0 to 18. The condition is characterized by abnormal lymphatic vessel development, leading to fluid accumulation and swelling. By comparing various concentration gradients of sirolimus, the researchers hope to find a dosage that maximizes therapeutic benefits while minimizing side effects. The study is being conducted at the West China Hospital of Sichuan University.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 0 to 18 with a confirmed diagnosis of cystic lymphatic malformation.
Not a fit: Patients with contraindications to sirolimus or those who have recently undergone certain treatments or surgeries may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a more effective and safer treatment option for children suffering from cystic lymphatic malformations.
How similar studies have performed: Previous studies have indicated potential benefits of sirolimus in treating lymphatic malformations, suggesting this approach may be promising.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* Presenting a LM with the following characteristics:
1. Male and female;
2. Between 0 and 18 years of age;
3. LM 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:
Biopsy; Compatible MRI findings; History and clinical features.
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)
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.