Diffuse Midline Glioma study: ONC206
Official title ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors
ClinicalTrials.gov ID: NCT04732065
What this study is testing
What is ONC206?
An investigational oral medicine being studied for treating diffuse midline glioma (DMG) in children and young adults.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study looks at how well ONC206 works and what dose is safest for treating newly diagnosed or returning diffuse midline gliomas and other malignant brain tumors.
- Who it's for
- It is for adults or children who have these specific brain tumors and are willing to try a new treatment.
- What's involved
- Participants may receive the drug alone or with radiation, and will have regular medical visits and tests to monitor safety and tumor response.
- Time commitment
- The trial lasts several months, with scheduled visits for drug administration, imaging scans, and safety checks.
- Phase 1: an early, usually small safety study
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 21
You may be able to join if
- ARM A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age
- ARM A: Tumor tissue confirmation of DMG, H3K27 altered is mandatory and pathology must be consistent with a DMG, H3K27 altered.
- ARM A: Participants must have recovered from all acute side effects of prior therapy.
- ARM A: From the projected start of scheduled study treatment, the following time periods must have elapsed: 5 half-lives from any investigational...
- ARM B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age
You likely can't join if
- Arm A \& B: For tumors that do not have a pontine or spinal cord epicenter the following specific exclusion criteria apply: Thalamic DMG and...
- Arm C \& D: Patients who participated in trials investigating ONC201 in the upfront setting will not be eligible.
- Participants who are currently receiving another investigational drug are not eligible.
- Participants who are currently receiving other anti-cancer agents are not eligible.
- Participants with a known disorder that affects their immune system, such as human immunodeficiency virus (HIV) or hepatitis B or C, or an...
- Participants with uncontrolled infection.
See the full eligibility criteria
Who can join
- ARM A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy.
- ARM A: Tumor tissue confirmation of DMG, H3K27 altered is mandatory and pathology must be consistent with a DMG, H3K27 altered.
- ARM A: Participants must have recovered from all acute side effects of prior therapy.
- ARM A: From the projected start of scheduled study treatment, the following time periods must have elapsed: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide...
- ARM B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs.
- ARM B: Tumor tissue confirmation of DMG is mandatory and pathology must be consistent with a DMG, H3K27 altered.
- ARM C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are...
- ARM C: Patients must have undergone prior focal radiation therapy as part of their initial therapy and should be at least 6 months from prior radiation therapy.
- ARM C: Tumor tissue confirmation is mandatory and pathology must be consistent with a DMG, H3K27 altered.
- ARM C: Participants must have recovered from all acute side effects of prior therapy
- ARM C: From the projected start of scheduled study treatment, the following time periods must have elapsed: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide...
- ARM D: Children and young adults with recurrent primary malignant CNS tumors, excluding DMGs, (Dose escalation: 2-21 years of age
- ARM D: Prior tumor tissue confirmation is mandatory and pathology from the primary tumor must be consistent with malignant CNS tumor (diagnosis of ependymoma is allowed).Tissue at the time of progression is not required.
- ARM D: Participants must have recovered from all acute side effects of prior therapy
- ARM D: From the projected start of scheduled study treatment, the following time periods must have elapsed: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide...
- TARGET VALIDATION: Newly diagnosed children and adults (2 years of age and above) with imaging consistent with a DMG, H3K27 altered are eligible.
- TARGET VALIDATION: Children and young adults with recurrent primary malignant CNS tumors, including recurrent DMG, (2 years of age and above) who have evidence of progression but have not been treated for this...
- TARGET VALIDATION: Participants must undergo tumor tissue collection as part of their standard of care
- Participants who are receiving steroids must be on a stable or decreasing dose for at least 3 days prior to registration.
- Peripheral absolute neutrophil count (ANC) \>= neutrophil 1.0 g/l.
- Platelet count \>= 100 x 10\^9/L (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
- Serum creatinine \< 1.5 Upper Limit normal (ULN) based on age and gender.
- Total bilirubin \<= 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin \< 3 x ULN or direct bilirubin \< 1.5 x ULN.
- Alanine aminotransferase (ALT) \<= 3 x ULN.
- Aspartate aminotransferase (AST) \<= 3 x ULN.
- Patients with seizure disorder may be enrolled if seizure disorder is well controlled
- The effects of ONC206 on the developing human fetus is unknown. For this reason, females of child-bearing potential and males must agree to use adequate contraception.
- Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants =\< 16 years of age.
- Participants must be willing to provide adequate tissue. A minimum of 10-20 paraffin embedded unstained slides OR 1 block with tumor content of 40% or greater is required. Frozen tissue is also acceptable.
- A legal parent/guardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate
- Patients must be enrolled on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution
What rules you out
- Arm A \& B: For tumors that do not have a pontine or spinal cord epicenter the following specific exclusion criteria apply: Thalamic DMG and cerebellar, H3K27 altered that has undergone standard radiation without...
- Arm C \& D: Patients who participated in trials investigating ONC201 in the upfront setting will not be eligible. Prior ONC201 exposure as part of PNOC022 or expanded access programs will be allowed.
- Participants who are currently receiving another investigational drug are not eligible.
- Participants who are currently receiving other anti-cancer agents are not eligible.
- Participants with a known disorder that affects their immune system, such as human immunodeficiency virus (HIV) or hepatitis B or C, or an auto-immune disorder requiring systemic cytotoxic or immunosuppressive therapy...
- Participants with uncontrolled infection.
- Female participants of childbearing potential must not be pregnant or breast-feeding. Female participants of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy.
- Active illicit drug use or diagnosis of alcoholism.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to ONC206.
- Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant or family.
- Any participants with illnesses that may affect absorption of ONC206.
- Any participants on strong inhibitors or inducers of CYP3A4, 2D6, 1A2, 2C9 and 2C19 at least 14 days prior and throughout the study.
The study team makes the final eligibility decision.
Where it is enrolling
- San Francisco, California, United States
- Washington D.C., District of Columbia, United States
- Atlanta, Georgia, United States
- Ann Arbor, Michigan, United States
- Philadelphia, Pennsylvania, United States
- Utrecht, Netherlands
- Zurich, Canton of Zurich, Switzerland
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at San Francisco, California, United States; Washington D.C., District of Columbia, United States; Atlanta, Georgia, United States; Ann Arbor, Michigan, United States; Philadelphia, Pennsylvania, United States; Utrecht, Netherlands and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.