Clinical Trials for Ependymoma: Current Research and Treatment Options
There are currently 3 ongoing clinical trials investigating new treatment approaches for ependymoma, a type of tumor that develops in the brain or spinal cord. These studies are testing various combinations of chemotherapy drugs, targeted therapies, and immune-based treatments for patients ranging from young children to young adults. The trials are being conducted across multiple European countries and France, offering different treatment strategies for newly diagnosed patients as well as those whose tumors have returned or not responded to standard treatments.
Clinical trial locations
- Austria
- Belgium
- Czechia
- Denmark
- Finland
- France
- Study on Ependymoma Treatment in Children, Adolescents, and Young Adults Using Cisplatin, Cyclophosphamide, Etoposide, and a Drug Combination
- Study on the Effectiveness of Bevacizumab and Drug Combination for Children with Recurrent or Progressive Medulloblastoma, Ependymoma, and ATRT
- Study of Nivolumab with Chemotherapy for Children and Teenagers with Refractory or Relapsing Solid Tumors or Lymphoma
- Germany
- Greece
- Ireland
- Italy
- Netherlands
- Norway
- Spain
- Sweden
Study on Ependymoma Treatment in Children, Adolescents, and Young Adults Using Cisplatin, Cyclophosphamide, Etoposide, and a Drug Combination
This trial focuses on finding better treatment approaches for children, adolescents, and young adults who have been newly diagnosed with ependymoma. The study is designed to test different combinations of chemotherapy drugs and determine which approach works best based on how much of the tumor could be removed during surgery.
Who can participate: The trial is open to patients younger than 22 years old at diagnosis who have newly confirmed intracranial or spinal ependymoma. Participants must not have received previous chemotherapy or radiotherapy for their tumor. They must have good organ function, including normal bone marrow, liver, and kidney function. Those who have started menstruating must not be pregnant or breastfeeding and must use effective birth control during treatment and for six months afterward. Patients must be willing to attend all scheduled visits and follow the treatment plan.
Who cannot participate: The trial excludes patients who have already received chemotherapy or radiotherapy, those with other serious medical conditions that would prevent them from receiving treatment safely, and patients with certain subtypes of ependymoma not included in the study. Patients who cannot undergo post-operative MRI scans or who have infections are also excluded.
What the trial aims to discover: The study aims to determine which treatment combinations provide the best outcomes for patients with different surgical results. For patients whose tumors were completely removed, the trial tests chemotherapy followed by radiation therapy. For those with remaining tumor that cannot be reoperated, the study compares two different chemotherapy schedules. For very young children who cannot receive radiation, the trial tests whether adding a medication called valproate to chemotherapy can improve outcomes. The research team will monitor how long patients remain free from disease progression and overall survival.
Investigational drugs being tested: The main chemotherapy regimen is called VEC, which combines vincristine, etoposide, and cyclophosphamide. This is often used together with cisplatin, a platinum-based drug that damages cancer cell DNA. Some patients will also receive high-dose methotrexate, which interferes with cancer cell growth. Additionally, the trial tests valproate, primarily known as a seizure medication, which in this study is being evaluated for its potential to make chemotherapy more effective by changing how genes are expressed in tumor cells.
Study on the Effectiveness of Bevacizumab and Drug Combination for Children with Recurrent or Progressive Medulloblastoma, Ependymoma, and ATRT
This trial investigates treatment options for children whose brain tumors, including ependymoma, have either returned after treatment or continued to grow despite previous therapy. The study uses an approach called metronomic therapy, which involves giving lower doses of chemotherapy drugs more frequently to minimize side effects while maintaining effectiveness. The treatment also includes anti-angiogenic therapy, which aims to cut off the blood supply that tumors need to grow.
Who can participate: Children and young people under 20 years of age who were diagnosed with recurrent or progressive medulloblastoma, ependymoma, or atypical teratoid rhabdoid tumor can join this trial. The diagnosis must be confirmed through tissue examination, and there must be at least one tumor site that has not been previously re-treated. Participants must have a performance status score of at least 50, meaning they can perform most daily activities with some limitations. They need normal organ and bone marrow function, with specific requirements for liver enzymes, kidney function, white blood cells, and platelets. Patients who previously received similar immune-based treatments may be eligible if they had good responses.
Who cannot participate: The trial excludes patients who are not within the specified age range, cannot follow study procedures, or have other medical conditions that could interfere with the study or pose health risks. Pregnant or breastfeeding patients cannot participate, nor can those with severe allergies to the study drugs, active infections, or other serious illnesses.
What the trial aims to discover: The study will evaluate how well different combinations of chemotherapy drugs work when given in a metronomic schedule. It aims to determine which treatment combination is most effective at controlling tumor growth and improving quality of life. The trial will assess response rates at six months and monitor patients for up to three years to track overall survival and progression-free survival.
Investigational drugs being tested: The trial tests several drugs including cytarabine (injected into the spinal fluid), celecoxib (an oral anti-inflammatory drug), cyclophosphamide (an oral chemotherapy drug), etoposide (given through intravenous infusion), bevacizumab (an intravenous drug that blocks blood vessel formation), fenofibrate (an oral medication), and thalidomide (an oral drug). These medications work together to attack cancer cells from different angles while also preventing the formation of new blood vessels that tumors need to survive and grow.
Study of Nivolumab with Chemotherapy for Children and Teenagers with Refractory or Relapsing Solid Tumors or Lymphoma
This trial explores the use of immune checkpoint inhibitor therapy combined with chemotherapy for children and teenagers whose solid tumors, which may include ependymoma, have not responded to standard treatments or have returned after previous therapy. The study tests whether adding an immune-based medication called nivolumab to metronomic chemotherapy can improve treatment outcomes.
Who can participate: Patients under 18 years of age with confirmed solid malignant tumors or lymphoma that are either getting worse despite treatment or not responding to standard therapy can participate. Older patients diagnosed with pediatric cancers before age 18 may also be included after discussion with the research team. Participants must have a performance status of 70% or higher, meaning they can carry out most daily activities with some limitations. They need adequate organ function including normal blood counts, heart, kidney, and liver function. Females who can become pregnant must have a negative pregnancy test within seven days before starting treatment. Patients must be able to attend follow-up visits and manage potential side effects.
Who cannot participate: The trial excludes patients outside the specified age range, those who cannot follow study procedures or take medications as required, pregnant or breastfeeding patients, those with severe allergic reactions to study drugs, and patients with active infections or other serious illnesses that could affect participation.
What the trial aims to discover: The study has two stages. The first stage determines which of three chemotherapy regimens is safest to use with nivolumab. The regimens being tested are cyclophosphamide with vinblastine, capecitabine alone, or a combination of all three drugs. The second stage focuses on the most promising regimen from the first stage to evaluate how well it works at preventing cancer progression, both with and without nivolumab.
Investigational drugs being tested: The trial tests nivolumab, an immune checkpoint inhibitor that helps the immune system recognize and attack cancer cells by blocking a protein called PD-1. The chemotherapy drugs include cyclophosphamide, which damages cancer cell DNA to prevent growth and division, vinblastine, which disrupts cell division by interfering with the cell’s internal structure, and capecitabine, an oral drug that converts to 5-fluorouracil in the body and interferes with cancer cell DNA and RNA production.
Summary
The three ongoing clinical trials for ependymoma reflect different treatment approaches based on disease stage and patient age. The largest trial, conducted across 13 European countries, focuses exclusively on newly diagnosed patients and tests various chemotherapy combinations tailored to surgical outcomes and patient age. Two other trials address the challenging situation of recurrent or progressive disease, with one conducted across seven European countries and another in France.
There is notable geographic concentration of trials in France, Austria, Denmark, Czechia, Norway, and Spain, which participate in multiple studies. This distribution provides patients in these countries with several treatment options depending on their specific situation.
The trials employ different therapeutic strategies. Traditional chemotherapy combinations remain the foundation of treatment, particularly cisplatin, cyclophosphamide, and etoposide. Newer approaches include anti-angiogenic therapy aimed at cutting off tumor blood supply, immune checkpoint inhibitors that help the immune system fight cancer, and metronomic chemotherapy using lower, more frequent doses to reduce side effects.
An interesting aspect is the investigation of valproate, traditionally used for seizures, as a potential enhancer of chemotherapy effectiveness. This represents an example of drug repurposing in cancer treatment.
These trials offer hope for improved outcomes, particularly for young patients whose tumors cannot be completely removed surgically or who experience recurrence. Families interested in participation should discuss eligibility with their treating physicians.
