SCMC Trial on KHE With KMP (V.2020)
- Conditions
- Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
- Interventions
- Drug: prednison
- Registration Number
- NCT04409691
- Lead Sponsor
- Shanghai Children's Medical Center
- Brief Summary
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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 20
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description prednison+sirolimus group prednison and Sirolimus - prednison group prednison -
- Primary Outcome Measures
Name Time Method response to treatment 6 months after taking the drug 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.
- Secondary Outcome Measures
Name Time Method side effect rate 6 months after taking the drug Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms
Trial Locations
- Locations (1)
Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
🇨🇳Shanghai, Shanghai, China