OncoMatch/Clinical Trials/NCT06161519
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Is NCT06161519 recruiting? Yes, currently enrolling (May 2026). This Phase 1/2 trial studies PLX038 for glioma.
Treatment: PLX038 — Background: 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.
Check if I qualifyExtracted eligibility criteria
Cancer type
Glioblastoma
Biomarker criteria
Required: MYCN amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
MYCN amplified ependymoma
Required: MYCN amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
MYCN amplified ependymoma (recurrent or progressive)
Required: MYCN amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
medulloblastoma with MYCN amplification
Required: MYC amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
medulloblastoma with MYC amplification
Required: MYCN amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
any other recurrent or progressive primary CNS tumor with MYCN amplification
Required: MYC amplification (fold change (FC) of >= 2.5X (5 copies) with a minimum tumor content of 20%)
any other recurrent or progressive primary CNS tumor with MYC amplification
Required: MYCN wild-type
Any recurrent glioblastoma without MYC or MYCN amplifications
Required: MYC wild-type
Any recurrent glioblastoma without MYC or MYCN amplifications
Excluded: UGT1A1 *28 homozygous
Participants with history of homozygous for the UGT1A1*28 variant allele with severely reduced UGT1A1 activity
Prior therapy
Must have received: surgery and radiation therapy — newly diagnosed MYCN amplified ependymoma (Cohort Phase IIA)
must have completed surgery followed by radiation at least 4 weeks and no more than 10 weeks from the last dose of radiation prior to study treatment initiation
Must have received: cytotoxic chemotherapy or radiation therapy — Cohorts Phase I, Phase IIB, Phase IIC, and Phase IID
must have completed prior cytotoxic chemotherapy or radiation at least 4 weeks prior to study treatment initiation (at least 6 weeks if the last regimen included lomustine (CCNU) or carmustine (BCNU); at least 3 weeks if the last regimen included bevacizumab; at least 4 weeks if the last regimen included a checkpoint inhibitor or any other type of immunotherapy or cellular therapy; at least 5 half-lives if the last regimen included any investigational agent(s). Participants previously treated with PHOTON radiation to at least 2 segments of the spine must have completed radiation at least 12 months before study treatment initiation.
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 * aspartate aminotransferase (AST) / alanine aminotransferase (ALT) <2.5 X institutional upper limit of normal (ULN) * creatinine within normal institutional limits OR * estimated glomerular filtrate rate (eGFR) using chronic kidney disease epidemiology collaboration) (CKD-EPI) equation:>= 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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