Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)

• Status: Recruiting
• Trial end date: 2029-12-30
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Treatments: Sirolimus

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 have not previously received sirolimus therapy.

• Historical control group - patients with diagnosed recurrent JNA, received treatment in Dmitry Rogachev' s Center in the period from January 1, 2013 to April 15, 2022.

Exclusion Criteria:

Hypersensitivity to sirolimus or its analogues. The presence of acute or chronic infections, including opportunistic infections.

Hepatic and/or renal insufficiency. The need for concomitant use of inducers (e.g. rifampicin, rifabutin) or inhibitors (e.g. ketoconazole) of the cytochrome CYP3A4 system Previous therapy with sirolimus or other mTOR inhibitors. Indications to palliative therapy, according to investigator's opinion. Participation in other clinical trials.