Ongoing Clinical Trials for Malignant Ependymoma
Currently, there is 1 ongoing clinical trial for malignant ependymoma, a serious type of brain tumor that can affect children and young adults. This trial is testing a combination of two medications, axitinib and etoposide, in patients whose tumors have not responded to standard treatments or have returned after treatment. The trial is being conducted in France and aims to find better treatment options for this challenging condition.
Clinical trial locations
Study of Axitinib and Etoposide for Children and Young Adults with Refractory or Relapsing Medulloblastoma and Ependymoma
This clinical trial is testing a combination of two medications for children and young adults with brain tumors that have either not responded to previous treatments or have come back after initial treatment. The study focuses on two specific types of brain tumors: malignant ependymoma and medulloblastoma.
Main inclusion criteria:
- Patients must have a confirmed diagnosis of either ependymoma or medulloblastoma
- Age between 4 and 25 years old
- Weight must be more than 20 kg (about 44 pounds)
- The tumor must be measurable according to specific medical guidelines
- The disease must be getting worse or not responding to standard treatments, or no effective standard treatment exists
- Patients must be able to swallow capsules and take medicines by mouth
- Life expectancy of at least 3 months
- Adequate organ function including blood health, heart function, kidney and liver function
- Performance status score of 70% or higher, meaning they can do most daily activities
- If female and able to have children, must have a negative pregnancy test and agree to use birth control during the study and for 6 months after
Main exclusion criteria:
- Patients with a different type of cancer than ependymoma or medulloblastoma
- Patients who are pregnant or breastfeeding
- Patients with other serious health conditions that might interfere with the study
- Patients unable to take the study medications as prescribed or follow study procedures
- Patients unable to attend study visits
Focus and goal of the trial:
The study is being conducted in two stages. In the first stage, researchers will work to find the best and safest dose of axitinib when used together with a fixed dose of etoposide. This is called the dose escalation phase, and it helps ensure that patients receive a dose that is effective but doesn’t cause too many side effects. In the second stage, the focus shifts to evaluating how well this combination of medications works in preventing the tumors from growing or spreading further. The main goal is to improve progression-free survival, which means keeping the disease from getting worse for as long as possible.
Throughout the trial, patients will be carefully monitored with regular assessments to check how the tumor is responding to treatment and to watch for any side effects. The study will track important measures like overall survival and progression-free survival from the start of treatment until the end of the study.
Investigational drugs:
The trial uses two medications: axitinib (brand name Inlyta) and etoposide. Axitinib is a targeted therapy that works by blocking certain proteins that help cancer cells grow and develop blood vessels. By blocking these proteins, it may slow down or stop the growth of the tumor. Etoposide is a chemotherapy drug that interferes with the DNA of cancer cells, preventing them from dividing and growing. In this study, etoposide is given in a special way called “oral metronomic dosing,” which means it is taken by mouth in small, regular doses over time to maintain a steady level of the drug in the body. Both medications are taken orally, making treatment more convenient for young patients.
Summary
For patients with malignant ependymoma, there is currently one active clinical trial available, conducted in France. This trial represents an important research effort for children and young adults whose tumors have not responded to standard treatments or have returned after initial therapy. The study focuses on combining two medications, axitinib and etoposide, both of which work through different mechanisms to fight cancer cells. The trial’s two-stage design allows researchers to first establish the safest dosing and then evaluate effectiveness, providing a thorough approach to developing new treatment options for this challenging condition. Patients interested in participating should discuss with their healthcare team whether they meet the criteria and whether this trial might be a suitable option for their situation.



