A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)


Individuals who have been newly diagnosed with a brain tumor or have a brain tumor that is growing or has come back (recurred) after receiving known useful treatment may be eligible.

The purpose of this study is to test how much of an investigational drug called fimepinostat reaches tumors in patients with brain tumors. We want to find out what effects, good and/or bad fimepinostat has on you and your brain tumor.

Study details

There will be three groups in this study, Group A, Group B and Group C.

  • Group A is for patients who have been newly diagnosed with a brain tumor called diffuse intrinsic pontine glioma (DIPG).
  • Group B is for patients with recurrence of a brain tumor called medulloblastoma
  • Group C is for patients with recurrence of a brain tumor called high-grade glioma (HGG).

The doctors will discuss with you what group you will be in.

Study procedures

If you meet the qualifications to be included and decide to take part in this study, you will receive fimepinostat at a dose that has been studied in other cancers once a day for two days before your surgery, and once on the day of your surgery, before the surgery is performed.

After you have recovered from your surgery, you will continue to receive fimepinostat.

Participation in this study requires that blood and tissue samples undergo genetic testing, which the study teams can explain with you in detail.

There is limited experience with fimepinostat in humans. It is not known whether this drug works better than other treatments for your tumor. Research in the laboratory ha shown fimepinostat decreases the function of certain genetic mutations that are suspected to be active in causing the growth of the types of tumors being tested. We do not yet know whether blocking these mutations will be an effective treatment for your tumor.

You may receive fimepinostat for up to 1 year after your surgery, possibly longer if you benefit from the study drug and do not experience bad side-effects. After you are finished taking fimepinostat, we would like to monitor you for up to 5 years after your last dose of fimepinostat.


Patients must have one of the following histologically confirmed diagnoses (histologic confirmation from initial diagnosis acceptable, as appropriate):

  • Stratum A: Newly diagnosed diffuse intrinsic pontine glioma (DIPG) (World Health Organization [WHO] grade II-IV)- this stratum does not require tissue confirmation at time of enrollment, but diagnostic confirmation will be required to continue on study after biopsy
  • Stratum B: Recurrent medulloblastoma (WHO grade IV), any molecular subtype
  • Stratum C: Recurrent high-grade glioma (HGG), including anaplastic astrocytoma (WHO grade III) and glioblastoma (WHO grade IV)

Additional criteria apply.


For questions about the study, please reach out to Jennifer King:




Brain tumor - primary - adults, Brain tumor - children, Brain Tumors, medulloblastoma, Glioma

Principal Investigator

Sridharan Gururangan, FRCP



Contact Information


(352) 294-8374

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