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

NCT05549167 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 117

Last updated 2022-09-22

No results posted yet for this study

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.

Conditions

  • Juvenile Nasopharyngeal Angiofibroma

Interventions

DRUG

Sirolimus

Sirolimus 0,8 mg/m2, but \<2 mg per day, once a day (QD), per os, oral solution, 1mg/ml, 60 ml Optimal individual dosage of sirolimus will be determined under control of concentration in blood serum. Therapeutic concentration - 5-15 ng/ml. Blood samples will be collected until therapeutic concertation will be achieved in 2 consecutive samples, but no more than 4 samples (on the 4th, 7th, 10th, 14th days of admission).

Sponsors & Collaborators

  • Federal Research Institute of Pediatric Hematology, Oncology and Immunology

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
7 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-05-30
Primary Completion
2029-09-30
Completion
2029-12-30

Countries

  • Russia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05549167 on ClinicalTrials.gov