Efficacy and Safety of Sirolimus to Vascular Anomalies

• Status: Recruiting
• Trial end date: 2021-12-31
• Target enrollment: 0
• Participant gender: All

Summary

To evaluate the safety and efficacy of Sirolimus in complicated vascular anomalies in Chinese children

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: West China Hospital
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• All patients included in the present research must be diagnosed with one of the following vascular anomalies:

1. Kaposiform Hemangioendotheliomas without Kasabach-Merritt Phenomenon

2. Tufted Angioma without Kasabach-Merritt Phenomenon

3. Capillary Malformations

4. Lymphatic Malformations

5. Venous Malformations

6. Capillary-Venous Malformation (CVM)

7. Capillary-Lymphatic Malformation (CLM)

8. Lymphatic-Venous Malformation (LVM)

9. Capillary-Lymphatic-Venous Malformation (CLVM)

10. Multifocal Lymphangiomatosis and Thrombocytopenia (MLT)

• Patients must be 0 - 18 years of age at the time of study entry.

• Without functional impairment requiring treatment of corticosteroid.

• Organ function requirements:

• Adequate liver function Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and alanine transaminase (ALT) and aspartate aminotransferase (AST) less than or equal to 2.5 x upper limit normal (ULN) for age.

• Adequate 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 11-15 years of age maximum serum creatinine (mg/dL) of 1.2 16-18 years of age maximum serum creatinine (mg/dL) of 1.5

• Adequate bone marrow function:

Absolute Neutrophil Count (ANC) greater than or equal to 1 x 10 to the ninth/Liter

• Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.

Exclusion Criteria:

• Allergy to sirolimus or other rapamycin analogues.

• Allergy to sirolimus or other rapamycin analogues.

• Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization.

• Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected.

• Other concurrent severe and/or uncontrolled medical disease which 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 who have a history of malignancy.

• Patients with an inability to participate or to follow the study treatment and assessment plan.

• Patients who have a history of treatment with sirolimus or other mTOR inhibitor.