Purpose of this Study
We are doing this study to find out if the combination of an experimental drug called ONC201 (the study drug) with paxalisib is an effective option for patients with a diffuse midline glioma (DMG). We also want to see how much of these drugs are absorbed by tumor tissue in patients with brain tumors.
Who Can Participate?
Eligibility
Children and adults ages 2-29 who are diagnosed with DMG and:
- Have not started treatment yet; OR
- Have finished radiation therapy and have no evidence of progression; OR
- Have finished a full round of standard therapy and show evidence of progression
For more information about who can join this study, please contact the study team at 919-684-5301.
- Have not started treatment yet; OR
- Have finished radiation therapy and have no evidence of progression; OR
- Have finished a full round of standard therapy and show evidence of progression
For more information about who can join this study, please contact the study team at 919-684-5301.
Age Range
2-39
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
If you choose to join this study, you will go through a study screening period that lasts about 14 days and includes:
- Physical exams
- Blood draws and a urine sample
- An Imaging scan (MRI)
- A lumbar puncture (spinal tap)
- An electrocardiogram (ECG) and echocardiogram (ECHO)
If you are eligible to join the study based on your screening results, you will proceed to the study regimen phase. The procedures during this phase will depend on the routine care that your study doctor believes is appropriate. The study regimen phase has 3 parts, and you may be eligible to participate in some or all of the parts.
If your doctor believes that you should have surgery to resect your tumor, you will participate in Part 1 of the study regimen. In this part, you will get 2 doses of the study drug before your tumor is resected.
If your doctor believes that radiation therapy is appropriate for you, you will participate in Part 2 of the study regimen. In this part, you will take the study drug at the same time you get radiation therapy.
Everyone who joins the study will participate in Part 3. During this part, you will take the study drug in combination with paxalisib.
Both the study drug and paxalisib are pills that you take by mouth. In addition to the drug regimen and surgery and/or radiation (if applicable), participation in the study will also involve:
- Physical exams
- Blood draws
- Stool samples
- Imaging scans (MRI)
- Heart scans (ECG)
- Questionnaires
You will stay on the study regimen until you and your doctor decide that it is not beneficial for you to continue.
About 14 days after you stop the study regimen, we will schedule you to come to the clinic for an end-of-study visit.
About 30 days after you finish the study regimen, someone from the study team will contact you to see how you are doing and ask about any medicines you are taking and if you are having any side effects.
About every 3 months after you finish the study, someone from the study team will contact you to see how you are doing and ask you to complete a questionnaire.
- Physical exams
- Blood draws and a urine sample
- An Imaging scan (MRI)
- A lumbar puncture (spinal tap)
- An electrocardiogram (ECG) and echocardiogram (ECHO)
If you are eligible to join the study based on your screening results, you will proceed to the study regimen phase. The procedures during this phase will depend on the routine care that your study doctor believes is appropriate. The study regimen phase has 3 parts, and you may be eligible to participate in some or all of the parts.
If your doctor believes that you should have surgery to resect your tumor, you will participate in Part 1 of the study regimen. In this part, you will get 2 doses of the study drug before your tumor is resected.
If your doctor believes that radiation therapy is appropriate for you, you will participate in Part 2 of the study regimen. In this part, you will take the study drug at the same time you get radiation therapy.
Everyone who joins the study will participate in Part 3. During this part, you will take the study drug in combination with paxalisib.
Both the study drug and paxalisib are pills that you take by mouth. In addition to the drug regimen and surgery and/or radiation (if applicable), participation in the study will also involve:
- Physical exams
- Blood draws
- Stool samples
- Imaging scans (MRI)
- Heart scans (ECG)
- Questionnaires
You will stay on the study regimen until you and your doctor decide that it is not beneficial for you to continue.
About 14 days after you stop the study regimen, we will schedule you to come to the clinic for an end-of-study visit.
About 30 days after you finish the study regimen, someone from the study team will contact you to see how you are doing and ask about any medicines you are taking and if you are having any side effects.
About every 3 months after you finish the study, someone from the study team will contact you to see how you are doing and ask you to complete a questionnaire.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
Yes
Study Details
Full Title
PNOC022: A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression
Principal Investigator
David
Ashley
Protocol Number
PRO00110922
NCT ID
NCT05009992
Phase
II/III
Enrollment Status
OPEN TO ACCRUAL