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Trial record 27 of 36 for:    "Viral Infectious Disease" | "Everolimus"

Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma

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ClinicalTrials.gov Identifier: NCT04077515
Recruitment Status : Recruiting
First Posted : September 4, 2019
Last Update Posted : September 4, 2019
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Fudan University

Brief Summary:
to evaluate the safety and efficacy of Low-dose sirolimus in Kaposiform Hemangioendothelioma in Chinese children by a prospective, randomized open trial.

Condition or disease Intervention/treatment Phase
Hemangioma Kaposiform Hemangioendothelioma Drug: Sirolimus(0.8mg/m2) Drug: Sirolimus(0.7mg/m2) Phase 4

Detailed Description:
The clinically commonly used dose of sirolimus for Kaposiform Hemangioendothelioma is 0.8 mg/m2 administered twice daily, and the blood concentration can be maintained at 10-15 ng/ml according to the pharmacokinetic formula.Related research reports that maintaining low blood concentration of sirolimus is effective in the treatment of certain vascular malformations and hemangioma, and complications are less. In the clinical practice, we found that the blood concentration was maintained at 7-10 ng/ml, and the patients still achieved good results and the chance of infections decreased. Therefore, this clinical trial was designed.In this trial, two different dosing regimens with corresponding blood concentration were designed to compare the safety and efficacy.In the high concentration group, the sirolimus dosage was adjusted monthly to achieve trough levels between 10 and 15 ng/mL(excluding 10 ng/ml), and it is still used at 0.8 mg/m2 administered twice daily.The low concentration group is 7-10 ng/ml (including 10 ng/ml), and the initial use of sirolimus is 0.7mg/m2 administered twice daily.The dose was adjusted according to the formula after two weeks.The follow-up and evaluation were performed according to a strictly established follow-up schedule after taking the drug.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 152 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma:A Prospective, Randomized Open Trial
Actual Study Start Date : May 1, 2019
Estimated Primary Completion Date : July 1, 2021
Estimated Study Completion Date : December 31, 2021


Arm Intervention/treatment
Active Comparator: high blood concentration group
The blood concentration is maintained at 10-15ng/ml (not including 10ng/ml).
Drug: Sirolimus(0.8mg/m2)
The initial use of sirolimus is 0.8mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.
Other Name: Rapamycin

Experimental: low blood concentration group
The blood concentration is maintained at 7-10ng/ml (including 10ng/ml).
Drug: Sirolimus(0.7mg/m2)
The initial use of sirolimus is 0.7mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.
Other Name: Rapamycin




Primary Outcome Measures :
  1. response to treatment [ Time Frame: 1 year after taking the drug ]

    it is a composit outcome (categorial variable), including complete responses, partial response and no response.

    The definitions are :

    Complete Response:

    1. platelets counts is greater than 100×10^9/L.
    2. significant volume reduction is greater than 80%.
    3. Fibrinogen levels at 2-4g/L.
    4. The surface skin of the tumor is lighter or the tumor is softer significantly.

    Partial Response:

    1. platelets counts is greater than 40×10^9/L.
    2. significant volume reduction is greater than 50%.
    3. Fibrinogen levels at less than 50% reduction from baseline.
    4. The surface skin of the tumor and palpation of the tumor have no change or less change.

    No Response:

    1. platelets counts is less than 40×10^9/L.
    2. significant volume reduction is less than 50% or the tumor is bigger.
    3. Fibrinogen levels at grater then 50% reduction from baseline.
    4. The surface skin of the tumor is darker or the tumor is harder.


Secondary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 1 year after taking the drug ]
    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 or without 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): NCT04077515


Contacts
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Contact: Kai Li, MD, PhD +86 02164931114 likai2727@163.com

Locations
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China
Children's Hospital of Fudan University Recruiting
Shanghai, China, 210012
Contact: Kai Li, MD, PhD    +86 02164931114    likai2727@163.com   
Principal Investigator: Kai Li, MD, PhD         
Sub-Investigator: Weili Yan, MD,PhD         
Sub-Investigator: Ying Gong, MD,PhD         
Sub-Investigator: Wei Yao, MD, PhD         
Sub-Investigator: Zuopeng Wang, MD,PhD         
Sponsors and Collaborators
Children's Hospital of Fudan University
Investigators
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Principal Investigator: Kai Li, MD, PhD Children's Hospital of Fudan University

Publications:
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Responsible Party: Children's Hospital of Fudan University
ClinicalTrials.gov Identifier: NCT04077515     History of Changes
Other Study ID Numbers: 2019-161
First Posted: September 4, 2019    Key Record Dates
Last Update Posted: September 4, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Children's Hospital of Fudan University:
Kaposiform Hemangioendothelioma
Kasabach-Merritt Syndrome
Sirolimus
Rapamycin
Additional relevant MeSH terms:
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DNA Virus Infections
Virus Diseases
Everolimus
Sarcoma, Kaposi
Hemangioma
Hemangioendothelioma
Kasabach-Merritt Syndrome
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Herpesviridae Infections
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