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OncoMatch/Clinical Trials/NCT06161519

PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications

Is NCT06161519 recruiting? Yes, currently enrolling (Sep 2026). This Phase 1/2 trial studies PLX038 for glioma.

Phase 1/2RecruitingNational Cancer Institute (NCI)NCT06161519Data as of Sep 2026

Treatment: PLX038Background: About 90,000 new cases of brain and spinal cord tumors are diagnosed annually in the United States. Most of these tumors are benign; however, about 30% are malignant, and 35% of people with malignant tumors in the brain and spinal cord will die within 5 years. Many of these people have changes in certain genes (MYC or MYCN) that drive the development of their cancers. Objective: To test a study drug (PLX038) in people with tumors of the brain or spinal cord. Eligibility: People aged 18 years or older with a tumor of the brain or spinal cord. Some participants must also have tumors with changes in the MYC or MYCN genes. Design: Participants will be screened. They will have a physical exam and blood tests. They will have imaging scans and a test of their heart function. They may need to have a biopsy: A sample of tissue will be removed from their tumor. PLX038 is given through a tube attached to a needle inserted into a vein in the arm. All participants will receive PLX038 on the first day of each 21-day treatment cycle. They will take a second drug 3 days later to help reduce the risk of infection; for this drug, participants will be shown how to inject themselves under the skin at home. Blood tests, imaging scans, and other tests will be repeated during study visits. Hair samples will also be collected during these visits. Some participants may have an additional biopsy. Study treatment will continue up to 7 months. Follow-up visits will continue every few months for up to 5 years.

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Extracted eligibility criteria

Treatments studied

Other

PLX038

Cancer type

Glioblastoma

Biomarker criteria

Required: MYCN amplification

Cohort Phase IIA: Newly diagnosed MYCN amplified ependymoma after surgery and radiation

Required: MYCN amplification

Cohort Phase IIB: Recurrent or progressive MYCN amplified ependymoma

Required: MYC amplification

Cohort Phase IIB: Recurrent or progressive medulloblastoma with MYC or MYCN amplifications

Required: MYCN amplification

Cohort Phase IIB: Recurrent or progressive medulloblastoma with MYC or MYCN amplifications

Required: MYC amplification

Cohort Phase IIC: Any other recurrent or progressive primary CNS tumor with MYC or MYCN amplifications

Required: MYCN amplification

Cohort Phase IIC: Any other recurrent or progressive primary CNS tumor with MYC or MYCN amplifications

Required: MYC wild-type

Cohort Phase IID: Any recurrent glioblastoma without MYC or MYCN amplifications

Required: MYCN wild-type

Cohort Phase IID: Any recurrent glioblastoma without MYC or MYCN amplifications

Demographics

Ages ≤ 120

Prior therapy

Cannot have received: pegylated topoisomerase inhibitor

History of treatment with pegylated topoisomerase inhibitors

Lab requirements

Blood counts

leukocytes >=3,000/microliter; absolute neutrophil count >1,500/microliter; platelets >100,000/microliter; hemoglobin >= 9 g/dL (may be transfused within 2 weeks prior to treatment to achieve this level)

Kidney function

creatinine within normal institutional limits OR eGFR >= 60 mL/min/1.73 m^2 for participants with creatinine levels above institutional normal

Liver function

total bilirubin within normal institutional limits; AST/ALT <2.5 X institutional upper limit of normal (ULN)

Participants must have adequate organ and marrow function as defined below: leukocytes >=3,000/microliter; absolute neutrophil count >1,500/microliter; platelets >100,000/microliter; hemoglobin >= 9 g/dL (may be transfused within 2 weeks prior to treatment to achieve this level); total bilirubin within normal institutional limits; AST/ALT <2.5 X institutional upper limit of normal (ULN); creatinine within normal institutional limits OR eGFR >= 60 mL/min/1.73 m^2 for participants with creatinine levels above institutional normal

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • National Institutes of Health Clinical Center · Bethesda, Maryland

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Frequently asked questions

Is NCT06161519 currently recruiting?

Yes, this trial is currently recruiting patients.

Are there prior therapy exclusions?

Yes. Prior pegylated topoisomerase inhibitor disqualifies patients from enrollment.

Does this trial require MYCN?

Yes, MYCN amplification is a required biomarker for enrollment.

Does this trial require MYCN?

Yes, MYCN amplification is a required biomarker for enrollment.

Does this trial require MYC?

Yes, MYC amplification is a required biomarker for enrollment.

Is there an age limit?

Yes. Patients must be 120 years or younger.

Could you qualify for this trial?

Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.

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Related pages

Glioblastoma trials