Back to resultsSCMC Trial on KHE With KMP (V.2020) (SCMC-KK2020)
Primary Purpose
Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
prednison and Sirolimus
prednison
Sponsored by
About this trial
This is an interventional treatment trial for Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
Eligibility Criteria
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
Sites / Locations
- Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
prednison group
prednison+sirolimus group
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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
Full Information
NCT ID
NCT04409691
First Posted
May 27, 2020
Last Updated
June 5, 2020
Sponsor
Shanghai Children's Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT04409691
Brief Title
SCMC Trial on KHE With KMP (V.2020)
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2020
Overall Recruitment Status
Unknown status
Study Start Date
July 1, 2020 (Anticipated)
Primary Completion Date
January 31, 2023 (Anticipated)
Study Completion Date
June 30, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shanghai Children's Medical Center
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
5. Study Description
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)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
prednison group
Arm Type
Active Comparator
Arm Title
prednison+sirolimus group
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
prednison and Sirolimus
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
prednison
Intervention Description
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.
Primary Outcome Measure Information:
Title
response to treatment
Description
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.
Time Frame
6 months after taking the drug
Secondary Outcome Measure Information:
Title
side effect rate
Description
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms
Time Frame
6 months after taking the drug
10. Eligibility
Sex
All
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Song Gu
Phone
18930830716
Email
gusong@shsmu.edu.cn
Facility Information:
Facility Name
Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200127
Country
China
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Song Gu, Doctor
Phone
18930830716
Email
gusong@shsmu.edu.cn
12. IPD Sharing Statement
Learn more about this trial
SCMC Trial on KHE With KMP (V.2020)
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