120 Participants Needed

PLX038 for Brain Tumors

NN
MP
JW
Overseen ByJing Wu, M.D.
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: National Cancer Institute (NCI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you cannot take drugs that strongly affect certain liver enzymes (CYP3A or UGT1A1). It's best to discuss your current medications with the trial team to see if any adjustments are needed.

What makes the drug PLX038 unique for treating brain tumors?

PLX038 is unique because it is a PEGylated form of SN-38, which is the active metabolite of the chemotherapy drug irinotecan, and it is designed to improve delivery and reduce side effects compared to traditional forms. This formulation may offer better penetration into brain tumors and potentially enhance treatment effectiveness.12345

What data supports the effectiveness of the drug PLX038 for brain tumors?

Research shows that similar treatments using PEG (a component of PLX038) combined with other drugs like paclitaxel have been effective in treating glioblastoma, a type of brain tumor, by enhancing drug delivery and increasing survival rates in animal models.13678

Who Is on the Research Team?

JW

Jing Wu, M.D.

Principal Investigator

National Cancer Institute (NCI)

Are You a Good Fit for This Trial?

Adults with certain brain or spinal cord tumors, specifically those with changes in MYC or MYCN genes. They must have had previous specific treatments, be able to perform most daily activities (Karnofsky >= 70%), and have proper organ function. Women who can bear children and men must use contraception; breastfeeding women must stop breastfeeding.

Inclusion Criteria

I am mostly able to care for myself.
I am breastfeeding but willing to stop for the trial.
I can describe my own symptoms and how well I can move.
See 6 more

Exclusion Criteria

Pregnancy
History of allergic reactions to compounds of similar chemical composition to PLX038
I have not had major surgery in the last 2 weeks.
See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment

Participants receive PLX038 intravenously every 3 weeks for up to 10 cycles, with additional self-administered medication to reduce infection risk

7 months
Every 3 weeks (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years
Every few months (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • PLX038
Trial Overview The trial is testing PLX038, a drug for brain/spinal cord tumors with genetic changes. Participants receive PLX038 via IV on day one of each 21-day cycle and self-inject another drug at home after three days to prevent infection. The treatment lasts up to seven months, followed by check-ups for five years.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Phase IIExperimental Treatment1 Intervention
Group II: Phase IExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

The study demonstrated that a PLGA/PEG paste can effectively deliver temozolomide and etoposide directly into the cavity after surgical resection of high-grade gliomas, leading to a significant survival benefit in animal models.
In vivo results showed that over half of the treated animals were disease-free, indicating the potential of this delivery method to enhance the efficacy of chemotherapy when combined with adjuvant radiotherapy.
Overall Survival in Malignant Glioma Is Significantly Prolonged by Neurosurgical Delivery of Etoposide and Temozolomide from a Thermo-Responsive Biodegradable Paste.Smith, SJ., Tyler, BM., Gould, T., et al.[2020]
The study identified an optimal dosing schedule for pegylated interferon alpha (PEG-IFN-alpha) at 10 KU twice a week, which significantly reduced cell proliferation and angiogenesis in glioblastoma cells in a mouse model.
Combining PEG-IFN-alpha with paclitaxel not only inhibited tumor growth effectively but also increased apoptosis in tumor-associated endothelial cells, suggesting a promising new treatment strategy for glioblastoma patients.
Synergistic effect and condition of pegylated interferon alpha with paclitaxel on glioblastoma.Son, MJ., Song, HS., Kim, MH., et al.[2018]
Co-loading paclitaxel and etoposide in methoxy poly(ethylene glycol)-poly(lactic-co-glycolic acid) nanoparticles significantly enhanced their cytotoxic effects against glioma cell lines, leading to lower IC50 values and increased cell apoptosis compared to the free drugs.
In vivo studies in rats with intracranial glioma showed that the co-loaded nanoparticles resulted in a median survival of 76 days, which is significantly longer than the 36 days for free paclitaxel and 37 days for free etoposide, along with improved tumor regression as confirmed by MRI.
Effect of Paclitaxel/etoposide co-loaded polymeric nanoparticles on tumor size and survival rate in a rat model of glioblastoma.Maleki, H., Hosseini Najafabadi, MR., Webster, TJ., et al.[2021]

Citations

Overall Survival in Malignant Glioma Is Significantly Prolonged by Neurosurgical Delivery of Etoposide and Temozolomide from a Thermo-Responsive Biodegradable Paste. [2020]
Synergistic effect and condition of pegylated interferon alpha with paclitaxel on glioblastoma. [2018]
Effect of Paclitaxel/etoposide co-loaded polymeric nanoparticles on tumor size and survival rate in a rat model of glioblastoma. [2021]
Enhanced anti-glioblastoma efficacy by PTX-loaded PEGylated poly(ɛ-caprolactone) nanoparticles: In vitro and in vivo evaluation. [2018]
Anti-glioblastoma efficacy and safety of paclitaxel-loading Angiopep-conjugated dual targeting PEG-PCL nanoparticles. [2018]
Anti-tumor effect of beta-elemene in glioblastoma cells depends on p38 MAPK activation. [2021]
Nose-to-brain delivery of temozolomide-loaded PLGA nanoparticles functionalized with anti-EPHA3 for glioblastoma targeting. [2020]
Combination of p38 MAPK inhibitor with PD-L1 antibody effectively prolongs survivals of temozolomide-resistant glioma-bearing mice via reduction of infiltrating glioma-associated macrophages and PD-L1 expression on resident glioma-associated microglia. [2021]
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