NCT05549167 Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)
| NCT ID | NCT05549167 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology |
| Condition | Juvenile Nasopharyngeal Angiofibroma |
| Study Type | INTERVENTIONAL |
| Enrollment | 117 participants |
| Start Date | 2022-05-30 |
| Primary Completion | 2029-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 117 participants in total. It began in 2022-05-30 with a primary completion date of 2029-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05549167 clinical trial?
This trial is open to male participants only, aged 7 Years or older, up to 18 Years, studying Juvenile Nasopharyngeal Angiofibroma. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05549167 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 117 participants.
Is NCT05549167 currently recruiting?
Yes, NCT05549167 is actively recruiting participants. Contact the research team at dr.vorozhtsov@gmail.com for enrollment information.
Where is the NCT05549167 trial being conducted?
This trial is being conducted at Moscow, Russia.
Who is sponsoring the NCT05549167 clinical trial?
NCT05549167 is sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. The trial plans to enroll 117 participants.