Clinical Trial: Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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 Summary:

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 age: For patients with NF2 who are 3 to < 18 years of age, dosing will be based on BSA. Dosing is based on BSA calculated at the beginning of each course. For patients with NF2 who are ≥ 18 to 45 years of age, dosing will be the standard adult dose of 75 mg BID.

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.

There will be two treatment strata. Stratum 1 is for those patients who have a target vestibular schwannoma which is causing hearing loss. Stratum 2 will be reserved for patients who exhibit growth of a tumor(s) besides vestibular schwannoma and are therefore not eligible for stratum 1.


Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome:

  • Change in hearing response at 24 weeks [ Time Frame: 24 weeks ]
    Word recognition scores
  • Response rate of other NF2 related tumors [ Time Frame: Through study completion up to 2 years ]
    Radiographic change in tumor measurements


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Response rate of vestibular schwannomas [ Time Frame: Through study completion up to 2 years ]
    Radiographic change in tumor measurements
  • Change in quality of life [ Time Frame: Through study completion up to 2 years ]
    Neurofibromatosis 2 impact quality of life questionnaire


Original Secondary Outcome: Same as current

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: March 8, 2017
Date Started: May 8, 2017
Date Completion: May 2021
Last Updated: May 9, 2017
Last Verified: May 2017