Actively Recruiting
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Led by National Cancer Institute (NCI) · Updated on 2026-05-13
146
Participants Needed
1
Research Sites
510 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with a primary central nervous system tumor including glioma, medulloblastoma, ependymoma, or glioblastoma
- Age 18 years or older
- Karnofsky Performance Status of 70% or higher
- Tumor tissue available for genetic testing and correlative studies
- Completed prior surgery and radiation or chemotherapy as specified for tumor type
- Adequate organ and marrow function
- Able to self-report symptoms and physical function
- Willing and able to sign informed consent
- Women of child-bearing potential and men who can father children agree to use effective contraception during and after the study
- Breastfeeding participants agree to stop breastfeeding from study start through 6 months after last dose
You will not qualify if you...
- History of allergic reactions to drugs similar to PLX038
- Major surgery within 2 weeks before starting treatment
- Need for treatment with strong CYP3A or UGT1A1 inhibitors or inducers during study
- Previous treatment with pegylated topoisomerase inhibitors
- Significant gastrointestinal dysfunction from prior craniospinal irradiation
- Genetic condition causing severely reduced UGT1A1 activity
- Positive for HIV, Hepatitis B, or Hepatitis C infections
- Pregnancy confirmed by test at screening
- Unable to have MRI scans
- Other cancers that might interfere with study assessment
- Uncontrolled illness that prevents study compliance
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
N
NCI NOB Referral Group
CONTACT
J
Jing Wu, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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