CONNECT 1702 (Glioma in Children and Young Adults)

We are doing this study to learn more about the safety of the study drug, PTC596, when taken during radiation. We also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors.

Diffuse Intrinsic Pontine Glioma, High-Grade Glioma, Radiotherapy

Children and adults 1-21 years old who:
- Are newly diagnosed with diffuse intrinsic pontine gliomas (DIPGs), defined as tumors with a pontine epicenter and diffuse involvement of at least 2/3 of the pons without histologic confirmation
- Have brainstem tumors that do not meet radiographic criteria or are not considered to be typical diffuse intrinsic pontine gliomas, if the tumors are biopsied and proven to be high-grade gliomas (such as anaplastic astrocytoma, glioblastoma, H3K27-mutant diffuse midline glioma) or diffuse astrocytoma
- Are newly diagnosed with non-brainstem high-grade glioma (HGG)
- Have had histologically verified high-grade glioma such as anaplastic astrocytoma, glioblastoma, H3 K27 mutant diffuse midline glioma, etc

For more information about who can participate in this study, please contact the study team.

The study is separated into two parts: Part A and Part B

- All participants will receive standard radiation therapy to treat your brain tumor and the study drug PTC596, a pill taken by mouth.

Phase I (Part A):
Find the dose of PTC596 that can be given with radiation without causing serious side effects.
- Have blood drawn to measure the amount of PTC596 in your body during Cycle 1 and Cycle 2.
- Take study drug two times each week during the 6-7 weeks of radiation.
- After you finish radiation, you may continue to take the study drug twice a week for up to 25 cycles (each cycle is 4 weeks) as long as you do not have any bad side effects and your brain tumor does not get worse.

Surgical (Part B)
We will test the amount of a study drug that may be found in the tumor and blood when given prior to and during a planned surgery for removal of the recurrent tumor.
- Take the study drug for the first time 1-4 days before your planned surgery.
- Take the second dose up to 12 hours before your surgery.
- Take study drug two times each week during the 6-7 weeks of radiation.
- After you finish radiation, you may continue to take the study drug twice a week for up to 25 cycles (each cycle is 4 weeks) as long as you do not have any bad side effects and your brain tumor does not get worse.