Efficacy and Safety of Sirolimus to Vascular Anomalies

  • STATUS
    Recruiting
  • End date
    Dec 31, 2021
  • participants needed
    126
  • sponsor
    West China Hospital
Updated on 26 January 2021

Summary

To evaluate the safety and efficacy of Sirolimus in complicated vascular anomalies in Chinese children

Description

Vascular anomalies are composed of vascular tumors and vascular malformations. The prognosis of vascular anomalies is significantly variable. Most of them had a benign course. However, complicated vascular anomalies can lead to disfigurement, organ disfunction and life-threatening with significant morbidity and mortality. Traditional treatments, including steroids, vincristine, cyclophosphamide and surgery, had limited response to complicated vascular anomalies. In the past few years, the inhibitor of the mammalian target of rapamycin (mTOR) signaling pathway-sirolimus has emerged as a treatment for severe vascular anomalies. Besides, preclinical studies also showed that the Phosphoinositide 3-kinase (PI3K)/protein kinase B (Akt)/mTOR pathway play an important role in the development of vascular tumors and vascular malformations. However, the exact efficacious rate and complications of sirolimus are still unknow in china because of the lack of large scale of prospective studies. Therefore, it's important to perform this prospective study to determine the safety and efficacy of sirolimus in the treatment of Chinese children with complicated vascular anomalies, and this study will also make contributions to the diagnoses and treatments of vascular anomalies.

Details
Condition Vascular anomaly, vascular malformation
Treatment Sirolimus
Clinical Study IdentifierNCT03583307
SponsorWest China Hospital
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

All patients included in the present research must be diagnosed with one of the following vascular anomalies
Kaposiform Hemangioendotheliomas without Kasabach-Merritt Phenomenon
Tufted Angioma without Kasabach-Merritt Phenomenon
Capillary Malformations
Lymphatic Malformations
Venous Malformations
Capillary-Venous Malformation (CVM)
Capillary-Lymphatic Malformation (CLM)
Lymphatic-Venous Malformation (LVM)
Capillary-Lymphatic-Venous Malformation (CLVM)
Multifocal Lymphangiomatosis and Thrombocytopenia (MLT)
Patients must be 0 - 18 years of age at the time of study entry
Without functional impairment requiring treatment of corticosteroid
Organ function requirements
Adequate liver function Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and alanine transaminase (ALT) and aspartate aminotransferase (AST) less than or equal to 2.5 x upper limit normal (ULN) for age
Adequate renal function 0-5 years of age maximum serum creatinine (mg/dL) of 0.8 6-10 years of age maximum serum creatinine (mg/dL) of 1.0 11-15 years of age maximum serum creatinine (mg/dL) of 1.2 16-18 years of age maximum serum creatinine (mg/dL) of 1.5
Adequate bone marrow function
Absolute Neutrophil Count (ANC) greater than or equal to 1 x 10 to the
ninth/Liter
Consent of parents (or the person having parental authority in families): Signed and dated written informed consent

Exclusion Criteria

Allergy to sirolimus or other rapamycin analogues
Allergy to sirolimus or other rapamycin analogues
Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization
Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected
Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration)
Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus
Patients who have a history of malignancy
Patients with an inability to participate or to follow the study treatment and assessment plan
Patients who have a history of treatment with sirolimus or other mTOR inhibitor
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