NCT03095248

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

Phase 2 Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 3 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

In this research study the researchers want to learn more about the effects (both good and bad) the study drug selumetinib has on participants with neurofibromatosis type II (NF2) related tumor. The researchers are asking patients with NF2 related tumors to be in the study, because their hearing has decreased and/or their NF2 related tumor has started to grow. The goals of this study are: * Determine if selumetinib will stop NF2 related tumors from growing * Measure the changes in hearing after receiving selumetinib for 6 months. * Determine if selumetinib improves how participants feel (physically and emotionally) and how participants can perform daily activities. * Examine tumor tissue, if available, in a laboratory to see if NF2 related tumors have targets of selumetinib.

Detailed description

This is a Phase 2 trial to assess the hearing response rate and radiographic response of VS in children and young adults with NF2 who are treated with selumetinib. Dosing will be based on BSA calculated at the beginning of each course. Selumetinib is taken orally twice a day continuously. One course is equivalent to 28 days. Therapy may continue for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity. Dose modifications will be based on Body Surface Area (BSA). In December, 2022 the investigator closed the Stratum 1 arm. Stratum 2 is reserved for patients who exhibit growth of a tumor(s) besides vestibular schwannoma.

Conditions

• Neurofibromatosis 2
• Vestibular Schwannoma
• Meningioma
• Ependymoma
• Glioma

Interventions

Type	Name	Description
DRUG	Selumetinib	Continuous twice daily dosing; oral agent

Timeline

• Start date: 2017-05-08
• Primary completion: 2024-05-21
• Completion: 2024-05-21
• First posted: 2017-03-29
• Last updated: 2025-04-22
• Results posted: 2025-04-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study