Ongoing Clinical Trials for Recurrent Medulloblastoma
Currently, there are 2 clinical trials investigating new treatments for recurrent medulloblastoma. These studies are testing different approaches including targeted therapies combined with chemotherapy and innovative cell-based treatments for patients whose tumors have returned or not responded to standard treatment.
Clinical trial locations
- France
- Spain
Study of Axitinib and Etoposide for Children and Young Adults with Refractory or Relapsing Medulloblastoma and Ependymoma
This trial is testing a combination of two medications, axitinib (also known by its brand name Inlyta) and etoposide, for children and young adults whose brain tumors have either not responded to standard treatments or have returned after treatment. The study is conducted in two stages, first finding the best dose of axitinib when combined with etoposide, then evaluating how well this combination prevents the tumors from growing or spreading further.
Who can participate:
- Patients between 4 and 25 years old
- Weight more than 20 kg (about 44 pounds)
- Confirmed diagnosis of either ependymoma or medulloblastoma that is getting worse or not responding to standard treatments
- Able to take medicines by mouth and swallow capsules easily
- Adequate blood, heart, kidney, and liver function
- Performance status score of 70% or higher (able to do most daily activities)
- Life expectancy of at least 3 months
- Measurable tumor according to specific guidelines
- For females of childbearing age: negative pregnancy test and agreement to use birth control during the study and for 6 months after
Who cannot participate:
- Patients with a different type of cancer than ependymoma or medulloblastoma
- Patients who are pregnant or breastfeeding
- Patients who have other serious health conditions that might interfere with the study
- Patients who cannot follow the study procedures or attend study visits
- Known allergies to the study treatment
Focus of the trial: The study aims to determine the maximum tolerated dose of axitinib when combined with oral metronomic etoposide and evaluate the effectiveness of this combination in preventing disease progression. Axitinib works by blocking certain proteins that promote cancer growth, while etoposide is a chemotherapy drug that interferes with cancer cell division. Both medications are taken by mouth, with etoposide given in small, regular doses over time to maintain steady levels in the body.
Investigational drugs: Axitinib (a tyrosine kinase inhibitor that blocks proteins helping cancer cells grow) and etoposide (a chemotherapy drug that interferes with DNA replication in cancer cells).
Study on the Safety and Efficacy of AloCelyvir for Children, Adolescents, and Young Adults with Diffuse Intrinsic Pontine Glioma or Medulloblastoma
This trial is evaluating a special cell-based therapy called AloCelyvir for young patients with recurrent or worsening brain tumors. AloCelyvir uses modified bone marrow cells combined with a virus called ICOVIR-5 to potentially fight the cancer. For patients with medulloblastoma that has returned or worsened, the study tests AloCelyvir on its own, given through an intravenous infusion directly into a vein.
Who can participate:
- Patients between 1 and 21 years old
- For medulloblastoma patients: must have already tried surgery, radiation therapy, and chemotherapy, and these treatments must not have worked (relapsed or refractory disease)
- Must have recovered from side effects of previous treatments to a mild level (except hearing loss, hair loss, and nerve damage)
- Disease must be measurable or evaluable according to specific medical criteria
- Adequate blood counts: platelet count at least 75,000/µL, neutrophil count at least 500/µL, hemoglobin at least 8 g/dL
- Good kidney function with glomerular filtration rate greater than 60 mL/min/1.73 m²
- Adequate liver function
- Functional status of at least 50%
- Life expectancy of at least 8 weeks
- For sexually active patients of childbearing age: must use highly effective contraceptive methods
- For females of childbearing age: negative pregnancy test required
Who cannot participate:
- Patients without a diagnosis of diffuse intrinsic pontine glioma or medulloblastoma that is worsening or coming back
- Patients who cannot safely receive AloCelyvir treatment
- Patients who cannot comply with the treatment schedule and visits
Focus of the trial: The study evaluates the safety and effectiveness of AloCelyvir as a standalone treatment for medulloblastoma that has returned or worsened after standard treatment. Regular visits are scheduled to monitor health and response to treatment, including physical exams, blood tests, and other necessary assessments. The goal is to understand how well the treatment works and ensure it is safe for use in children, adolescents, and young adults.
Investigational drug: AloCelyvir, a cell-based therapy combining mesenchymal allogenic cells with ICOVIR-5 virus, administered as an infusion. This advanced treatment is designed to use modified cells to potentially attack cancer cells.
Summary
Currently, two clinical trials are actively recruiting patients with recurrent medulloblastoma, offering different treatment approaches. One trial is taking place in France and focuses on combining targeted therapy with chemotherapy, while the other is conducted in Spain and tests an innovative cell-based treatment approach.
Both studies are designed specifically for pediatric and young adult populations, with age ranges from 1 to 25 years old. They target patients whose tumors have not responded to standard treatments or have returned after initial therapy. The French trial tests a combination of oral medications (axitinib and etoposide), while the Spanish trial evaluates an intravenous cell therapy (AloCelyvir).
These trials represent different therapeutic strategies: one using targeted inhibitors combined with chemotherapy, and the other employing modified cells to potentially fight cancer. Both studies emphasize careful monitoring of safety and effectiveness, requiring patients to have adequate organ function and the ability to comply with regular study visits and assessments.
