Using Sirolimus to Treat Juvenile Nasopharyngeal Angiofibroma in Children and Adolescents
Official title: Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)
Run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology · NCT05549167 · Data updated 2026-11-12
This study is testing if sirolimus can safely shrink juvenile nasopharyngeal angiofibroma tumors in children and teenagers aged 7 to 18.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 7 Years to 18 Years |
| Sex | Men only |
| Enrollment | 117 (estimated) |
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology Academic / other |
| Drugs / interventions | radiation |
| Locations | 1 site (Moscow) |
| Status | Recruiting |
| Started | May 30, 2022 |
| Main results expected | September 30, 2029 |
| Study ends | December 30, 2029 |
| Registry record updated | September 22, 2022 |
| Trial ID | NCT05549167 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are male children and adolescents aged 7-18 years with a confirmed diagnosis of primary or recurrent JNA.
✕Probably not a fit
Patients with JNA who do not meet the age or gender criteria, or those with inadequate liver or kidney function, may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study evaluates the efficacy and safety of sirolimus, an mTOR inhibitor, in treating juvenile nasopharyngeal angiofibroma (JNA) in children and adolescents aged 7 to 18 years. The trial consists of a pilot phase lasting three months, followed by an extended phase based on the treatment response. Participants will be assessed for changes in tumor volume using MRI, with historical controls for comparison. The study aims to provide insights into a new therapeutic approach for a condition that currently has limited treatment options.
Why it matters
Potential benefit: If successful, this treatment could offer a new, effective option for managing juvenile nasopharyngeal angiofibroma in young patients.
How similar studies have gone: While there is limited data on sirolimus specifically for JNA, the use of mTOR inhibitors has shown promise in other conditions, suggesting potential for success in this novel application.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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Research Institute of Pediatric Hematology, Oncology and Immunology
Moscow, Russia Recruiting
Study contacts
- Study coordinator: Igor m Vorozhtsov, MD,PhD
- Email: dr.vorozhtsov@gmail.com
- Phone: +79252019332
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.