Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)
Trial Parameters
Brief Summary
Juvenile nasopharyngeal angiofibroma (JNA) is a pathologically benign yet locally aggressive and destructive tumor that develops in the choana and nasopharynx. Historical treatment of JNA has included embolization, surgical resection, and radiation. mTOR signaling way demonstrated to be involved in regulation of growth and angiogenesis of JNA. Sirolimus, as mTOR inhibitor, is a potential target JNA therapy. The main purpose of the study is to evaluate the efficacy and safety of sirolimus in children and adolescents with primary or recurrent JNA. Efficacy will be estimated based on dynamics of the JNA progression. Historical control group will be used for comparison as standard therapy. Due to limited experience of sirolimus in JNA in routine practice, study should be conducted in 2 phases: pilot and extended. Decision regarding extended phase will be based on the results of pilot phase.
Eligibility Criteria
Inclusion Criteria: * Confirmed diagnosis of JNA. * Male gender. * Age 7-18 years. * Signed informed consent of the parents or the official representative for patients under 14 years of age, the patient and the patient's parents for persons from 14 to 18 years of age. * Adequate liver and kidney function. * Patients with primary JNA * Patients with tumor volume according to MRI \>35 ml. Group A1 (Extended phase:) - patients with decrease or stable tumor volume or an increase in tumor volume \< 20 %, after 3 months of sirolimus therapy. Group A2 (Extended phase:)- patients with an increase in tumor volume\> 20 % after 3 months of sirolimus therapy or the presence of other indications for surgical treatment, according to the investigator opinion. Historical control group - patients with diagnosed primary JNA, received treatment in Dmitry Rogachev's Center in the period from January 1, 2013 to April 15, 2022. * Group B RECCURRENT JNA, Patients with recurrent JNA after primary surgery, who