SCMC Trial on KHE With KMP (V.2020)

Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) Clinical Trial

— SCMC-KK2020  

Official title:

A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04409691
• Other study ID #: SCMC2020
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: July 1, 2020
• Est. completion date: June 30, 2023

Study information

• Verified date: June 2020
• Source: Shanghai Children's Medical Center
• Contact: Song Gu
• Phone: 18930830716
• Email: gusong[@]shsmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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)

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Hemangioendothelioma
• Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
• Kasabach-Merritt Syndrome
• Sarcoma, Kaposi

Intervention

Drug:
• prednison and Sirolimus
If the intravenous prednisolone 4 mg / kg / d (one time in the morning and one time in the evening, 2mg / kg body weight each time) is effective, it will be gradually reduced to 2mg / kg / D (one time in the morning and one time in the evening, 1mg / kg body weight each time), gradually converted to oral prednisone of equal dose, and the hormone will be removed within 4-6 weeks. At the same time, the dosage of sirolimus oral liquid is 0.8 mg / m2 twice a day, with an interval of 12 hours, maintaining the blood concentration of 8-15 ng / ml. if there is no intolerable side effect, the treatment will last for 6 months
• prednison
Prednison is taken at a dose of 4mg/kg/d. If the hormone treatment is effective, it will be gradually reduced to 2mg / kg / d (one time in the morning and one time in the evening, 1 mg / kg body weight each time), and repeated after 2 months of continuous treatment and 1 month of drug withdrawal.

Locations

Country	Name	City	State
China	Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine	Shanghai	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Shanghai Children's Medical Center

Country where clinical trial is conducted

China, 

Outcome

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