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PLX038 for Brain and Spinal Cord Tumors with MYC or MYCN Amplifications

This study is testing a drug called PLX038 for people with certain brain and spinal cord tumors. Many of these tumors have changes in genes called MYC or MYCN, which can make the cancer grow. PLX038 is given through a vein (intravenously) once every 21 days. The study aims to find the best dose of PLX038 and see how well it works in people aged 18 and older with these types of tumors. Some participants will need to have tumors with MYC or MYCN gene changes. The study will look at how the drug affects the tumor over 5 years.

Study design
This interventional study plans to enroll 146 participants. It is designed in two parts: Phase I to find the right dose, and Phase II to assess the drug's effectiveness.
What's involved
You would undergo screening, including a physical exam, blood tests, imaging scans, and a heart function test. PLX038 is given intravenously on day 1 of each 21-day cycle.
Compensation
Not stated in the trial record.
Follow-up
The study will assess the efficacy of PLX038 for up to 5 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06161519

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

Recruiting
PHASE1Ages 18+InterventionalTreatment
National Cancer Institute (NCI)
~146 participants
Updated 2026-08-12 on ClinicalTrials.gov
What's tested:PLX038

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase I: To confirm the RP2D of PLX038 in participants with progressive or recurrent primary CNS tumors
Measured over Days 1-42 (cycles 1-2)
+1 more outcome measured

Conditions

Glioma
Medulloblastoma
Ependymoma
Glioblastoma

Explore related trials

Childhood Central Nervous System NeoplasmEpendymomaGlioblastomaGliomaMedulloblastomaSpine tumorMYC GeneMYCN Gene

NCT06161519

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • National Institutes of Health Clinical Center

    Bethesda, Marylandstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Jing Wu, M.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)

Who to contact

NCI NOB Referral Group
[email protected]
(866) 251-9686
Email the study team

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Eligibility criteria

Inclusion

Participants must have documented pathologic diagnosis of confirmed primary central nervous system (CNS) tumor with one of the below diagnoses:
Cohort Phase I: Any recurrent or progressive primary CNS tumor, regardless of molecular features.
Cohort Phase IIA: Newly diagnosed MYCN amplified ependymoma after surgery and radiation.
Cohort Phase IIB:
Recurrent or progressive MYCN amplified ependymoma, OR
Recurrent or progressive medulloblastoma with MYC or MYCN amplifications
Cohort Phase IIC: Any other recurrent or progressive primary CNS tumor with MYC or MYCN amplifications.
Cohort Phase IID: Any recurrent glioblastoma without MYC or MYCN amplifications.
Participants must have archival tumor tissue (either a block or 15 formalin-fixed paraffin-embedded (FFPE) unstained slides) available for NCI LP review of MYC or MYCN amplification status (if necessary) and for correlative studies:
Cohorts Phase I, Phase IIB, Phase IIC, and Phase IID: tumor tissue obtained at any point before trial treatment initiation, but preferably from most recent surgical resection before study treatment initiation.
Cohort Phase IIA: tumor tissue obtained at original diagnosis.
Participants in 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.
Participants in 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).
Age \>= 18 years.
Karnofsky \>= 70%. NOTE: Participants with severe paraparesis/paraplegia who need minimal assistance for self-care due to their motor deficit but are otherwise functionally independent will be eligible.
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
Women of child-bearing potential (WOCBP) and those who can father children must agree to use effective contraception (barrier, hormonal contraception, intrauterine device (IUD), surgical sterilization, barrier at the study entry, for the duration of study treatment and up to 6 months (WOCBP) and 3 months (those who can father children) after the last dose of study treatment.
Breastfeeding participants must be willing to discontinue breastfeeding from study treatment initiation through 6 months after the last dose of the study drug.
Ability to self-report symptoms and physical function as determined by assessment of the clinical team performed at screening.
Participants must be able to understand and willing to sign a written informed consent document.

Exclusion

History of allergic reactions to compounds of similar chemical composition to PLX038.
Major surgery within 2 weeks prior to study treatment initiation. NOTE: The surgery is considered major if a mesenchymal barrier is opened (pleural cavity, peritoneum, meninges).
Participants who require treatment with strong inhibitors or inducers of CYP3A or with UGT1A1 inhibitors during the planned period of investigational treatment with PLX038. Lists including medications and substances known or with the potential to interact with CYP3A or UGT1A1 are provided in https://drug-interactions.medicine.iu.edu/maintable.
History of treatment with pegylated topoisomerase inhibitors.
Has unresolved or persistent grade 2 or higher GI toxicity from any type of RT at screening
Participants with history of homozygous for the UGT1A1\*28 variant allele with severely reduced UGT1A1 activity.
Participants positive for Human immunodeficiency virus (HIV), Hepatitis C virus (HCV), and Hepatitis B virus (HBV).
Pregnancy (confirmed with beta human chorionic gonadotropin (beta-HCG) serum or urine pregnancy test performed in females of childbearing potential at screening).
Participants unable to have MRIs.
Prior or concurrent malignancy unless its natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen (https://deainfo.nci.nih.gov/advisory/ctac/1117/4-JournalClinicalOncology.pdf, https://ctep.cancer.gov/protocolDevelopment/docs/CTEP\_Broadened\_Eligibility\_Criteria\_Guidance.pdf)
Uncontrolled intercurrent illness evaluated by history, weight, and physical exam that would limit compliance with study requirements.

What this trial measures

  • Phase I: To confirm the RP2D of PLX038 in participants with progressive or recurrent primary CNS tumorsDays 1-42 (cycles 1-2)

    Number of Dose Limiting Toxicities (DLT).

  • Phase II: To assess the efficacy of PLX038 at RP2D in primary CNS tumors containing MYC or MYCN amplifications5 years

    Adjuvant cohort: Defined as the time from the PLX038 treatment start date to the date of confirmed progression/death or last follow-up. Kaplan-Meier method will be used to estimate the survival function. The median PFS as well as the 95% CI will be summarized.Recurrent cohorts: Defined as the percentage of participants having CR, PR, or SD \>= 6 months as determined by investigator per RANO and/or RECIST v1.1. The DCR and its 95% exact binomial CI will be summarized.