A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
Status:
Recruiting
Trial end date:
2027-07-01
Target enrollment:
Participant gender:
Summary
In this research study the investigators want to learn more about the safety of the study
drug, PTC596 has when taken during radiation. The investigators also want to learn about the
effects, if any, these drugs have on children and young adults with brain tumors.
The investigators are asking people to be in this research study who have been diagnosed with
a high grade glioma (HGG) including diffuse intrinsic pontine glioma (DIPG) to be in the
research, because they have scheduled to have radiation to treat their cancer.
The study is divided into two parts. The goal of the first part is to find the dose of PTC596
that can be given with radiation without causing serious side effects. The purpose of this
surgical study is to 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.
The goals of the first part:
- Find the highest safe dose of PTC596 that can be given together with radiation therapy
without causing severe side effects;
- Learn what kind of side effects can be caused by PTC596 with radiation therapy;
- Learn more about the pharmacology of PTC596;
- Learn more about the biological effects of PTC596 on the cells in their body including
any changes to the tumor DNA;
- Determine whether PTC596 with radiation therapy is a beneficial treatment for their
tumor;
- Determine if there are any changes to participants quality of life when taking PTC596.
The goals of the surgical part are:
- Learn if PTC596 is able to reach the tumor in the brain;
- Learn what kind of side effects can be caused by PTC596 with radiation therapy;
- Learn more about the pharmacology of PTC596;
- Learn more about the biological effects of PTC596 on the cells in their body including
any changes to the tumor DNA;
- Determine whether PTC596 with radiation therapy is a beneficial treatment for their
tumor;
- Determine if there are any changes to their quality of life when taking PTC596.
Funding Source - FDA OOPD
Phase:
Phase 1
Details
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati Nationwide Children's Hospital