SCMC Trial on KHE With KMP (V.2020) (SCMC-KK2020)

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)

Study Overview

• Status: Not yet recruiting
• Conditions: Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
• Intervention / Treatment: Drug: prednison; Drug: prednison and Sirolimus

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Song Gu; Phone Number: 18930830716; Email: gusong@shsmu.edu.cn

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200127
      • Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
      • Contact: Song Gu, Doctor; Phone Number: 18930830716; Email: gusong@shsmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: prednison group	Drug: 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.
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Shanghai Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): July 1, 2020
• Primary Completion (Anticipated): January 31, 2023
• Study Completion (Anticipated): June 30, 2023

Study Registration Dates

• First Submitted: May 27, 2020
• First Submitted That Met QC Criteria: May 27, 2020
• First Posted (Actual): June 1, 2020

Study Record Updates

• Last Update Posted (Actual): June 9, 2020
• Last Update Submitted That Met QC Criteria: June 5, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Virus Diseases; Infections; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; DNA Virus Infections; Thrombocytopenia; Blood Platelet Disorders; Herpesviridae Infections; Sarcoma; Hemangioma; Neoplasms, Vascular Tissue; Sarcoma, Kaposi; Hemangioendothelioma; Kasabach-Merritt Syndrome; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: SCMC2020

Drug and device information, study documents

• 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