Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) Clinical Trial
— SCMC-KK2020Official title:
A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance
a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)
Status | Not yet recruiting |
Enrollment | 20 |
Est. completion date | June 30, 2023 |
Est. primary completion date | January 31, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 12 Years |
Eligibility |
Inclusion Criteria: - Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon - 0 - 12 years of age at the time of study entry - Male or female - Consent of parents (or the person having parental authority in families) - Signed and dated written informed consent Exclusion Criteria: - with hematological diseases - with other solid tumors - with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney - dysfunction, and cardiopulmonary insufficiency - with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment |
Country | Name | City | State |
---|---|---|---|
China | Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine | Shanghai | Shanghai |
Lead Sponsor | Collaborator |
---|---|
Shanghai Children's Medical Center |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | response to treatment | Complete Response: platelets counts is greater than 100×10^9/L. significant volume reduction is greater than 80%. Fibrinogen levels at 2-4g/L. The surface skin of the tumor is lighter or the tumor is softer significantly. Partial Response: platelets counts is greater than 40×10^9/L. significant volume reduction is greater than 50%. Fibrinogen levels at less than 50% reduction from baseline. The surface skin of the tumor and palpation of the tumor have no change or less change. No Response: platelets counts is less than 40×10^9/L. significant volume reduction is less than 50% or the tumor is bigger. Fibrinogen levels at grater then 50% reduction from baseline. The surface skin of the tumor is darker or the tumor is harder. |
6 months after taking the drug | |
Secondary | side effect rate | Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms | 6 months after taking the drug |