Current Research Studies

Cancer – CONNECT1702: Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

Condition or Therapy:

diffuse intrinsic pontine glioma, high-grade glioma, radiotherapy

Category:

Cancer and Blood Disorders
Study Number: CONNECT1702 

What is the goal of this study?

In this phase 1 study, the investigators want to learn more about the safety of the study drug, PTC596, 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), 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 are:

  • 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.

    Who can join the study?

    This study may be a good fit for children and young adults who:

    • Are between 1 and 21 years old, and 
    • 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

    What will happen if my child takes part in this study?

    You can read more about this study on clinicaltrials.gov.

    Who can I contact for more information?

    To learn more, call 206-987-2106 or email the study coordinator.


    Study Location(s):

    Seattle Children's Main Hospital Campus

    Principal Investigator:

    Dr. Sarah Leary
    Research Center: Center for Clinical and Translational Research