CONNECT 1702

What is the Purpose of this Study?

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.
What is the Condition Being Studied?
Diffuse Intrinsic Pontine Glioma, High-Grade Glioma, Radiotherapy

Who Can Participate in this Study?

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.

Age Group
Both
Participating Institutions

What is Involved?

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.

Study Details

Full Title
A Phase 1b Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
Principal Investigator
David Ashley
Rory David Deutsch Distinguished Professor of Neuro-Oncology

Protocol Number
IRB:
PRO00106705

NCT:
NCT03605550
ClinicalTrials.gov
View on ClinicalTrials.gov