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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04409691
Recruitment Status : Not yet recruiting
First Posted : June 1, 2020
Last Update Posted : June 9, 2020
Sponsor:
Information provided by (Responsible Party):
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)

Condition or disease Intervention/treatment Phase
Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) Drug: prednison and Sirolimus Drug: prednison Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


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




Primary Outcome Measures :
  1. response to treatment [ Time Frame: 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 :
  1. 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



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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Contacts
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Contact: Song Gu 18930830716 gusong@shsmu.edu.cn

Locations
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China, Shanghai
Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai, China, 200127
Contact: Song Gu, Doctor    18930830716    gusong@shsmu.edu.cn   
Sponsors and Collaborators
Shanghai Children's Medical Center
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Responsible Party: Shanghai Children's Medical Center
ClinicalTrials.gov Identifier: NCT04409691    
Other Study ID Numbers: SCMC2020
First Posted: June 1, 2020    Key Record Dates
Last Update Posted: June 9, 2020
Last Verified: June 2020

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