SCMC Trial on KHE With KMP (V.2020) (SCMC-KK2020)
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|ClinicalTrials.gov Identifier: NCT04409691|
Recruitment Status : Not yet recruiting
First Posted : June 1, 2020
Last Update Posted : June 9, 2020
|Condition or disease||Intervention/treatment||Phase|
|Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)||Drug: prednison and Sirolimus Drug: prednison||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance|
|Estimated Study Start Date :||July 1, 2020|
|Estimated Primary Completion Date :||January 31, 2023|
|Estimated Study Completion Date :||June 30, 2023|
|Active Comparator: prednison group||
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.
|Experimental: prednison+sirolimus group||
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
- response to treatment [ Time Frame: 6 months after taking the drug ]
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.
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.
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.
- side effect rate [ Time Frame: 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04409691
|Contact: Song Gufirstname.lastname@example.org|
|Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine|
|Shanghai, Shanghai, China, 200127|
|Contact: Song Gu, Doctor 18930830716 email@example.com|