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Ongoing Clinical Trials for Medulloblastoma

This article provides information about 7 ongoing clinical trials investigating new treatments for medulloblastoma, a type of brain tumor that primarily affects children. These trials are being conducted across multiple European countries and are testing various combinations of chemotherapy drugs, radiation therapy approaches, and targeted medications to improve outcomes for patients with different risk levels and subtypes of the disease.

Clinical trial locations

Study of drug combination therapy for high-risk medulloblastoma in patients over 3 years of age: carboplatin, cisplatin, cyclophosphamide, lomustine, temozolomide, thiotepa, vincristine

This trial focuses on treating high-risk forms of the disease in patients aged 3 years and older. The study is designed for patients with sonic hedgehog subgroup tumors or non-SHH/non-WNT (Groups 3 and 4) tumors with additional high-risk features.

Main inclusion criteria: Patients must be at least 3 years old at diagnosis and have confirmed high-risk disease. They need to have adequate liver, kidney, and blood function, with normal blood cell counts including sufficient neutrophils and platelets. Good hearing in at least one ear is required. Patients must not have received previous treatment except for surgery or one course of urgent initial chemotherapy. Tumor tissue and blood samples must be provided for analysis. Women who can become pregnant must have a negative pregnancy test and agree to use effective contraception during treatment.

Main exclusion criteria: Patients who have received prior cancer treatment such as chemotherapy, radiotherapy, or immunotherapy before joining the study cannot participate. Those with a history of other cancers within the past 5 years, severe heart problems, severe kidney or liver problems, or active uncontrolled infections are excluded. Pregnant or breastfeeding women, patients unable to undergo MRI scans, those with known allergies to study medications, or anyone participating in another clinical trial within 30 days are not eligible.

Study focus: The trial aims to determine whether specialized radiation therapy called hyperfractionated-accelerated radiotherapy or high-dose therapy with thiotepa can improve outcomes compared to standard radiation therapy. It also evaluates different maintenance chemotherapy approaches. The treatment involves multiple medications administered through various routes, with some given intravenously and others taken orally. Patients are monitored throughout treatment to track disease progression and overall survival.

Investigational drugs: Thiotepa is the key investigational medication, used as a chemotherapy agent in high-dose therapy. It works by interfering with cancer cell DNA to stop tumor growth. The trial also compares conventional radiation therapy delivered once daily with hyperfractionated-accelerated radiotherapy delivered multiple times per day. Various maintenance chemotherapy approaches are being tested to determine the most effective long-term treatment strategy.

Study on Preventing Hypothyroidism in Patients with Medulloblastoma and Pediatric Lymphoma Using Levothyroxine During Radiation Therapy

This trial examines whether taking levothyroxine during radiation therapy can help prevent hypothyroidism, a condition where the thyroid gland does not produce enough hormones. Patients will be monitored for three years after radiation treatment to assess thyroid function.

Main inclusion criteria: Patients must have medulloblastoma, Hodgkin lymphoma, or non-Hodgkin lymphoma and be preparing to receive radiation therapy that includes the thyroid gland area. They must not have had any previous primary or secondary hypothyroidism. Written informed consent must be provided by patients and their guardians. The study is open to male and female patients across various age groups, including children, adolescents, and adults.

Main exclusion criteria: Patients who do not have Hodgkin lymphoma, non-Hodgkin lymphoma, or medulloblastoma cannot participate. Those not within the specified age range are excluded, though the study is generally open to children and young adults. Gender is not a reason for exclusion, and vulnerable populations are not excluded from participation.

Study focus: The study investigates whether levothyroxine can reduce the risk of developing hypothyroidism after radiation therapy. Some patients will receive levothyroxine during their radiation treatment, while others will not, allowing researchers to compare outcomes. The primary goal is to determine if the medication can help protect the thyroid gland during treatment and improve long-term health outcomes. Participants are monitored for the onset of hypothyroidism through regular blood tests measuring thyroid-stimulating hormone levels.

Investigational drugs: Levothyroxine is being tested to see if it can lower the chances of hypothyroidism in patients receiving radiation treatment. The medication works by suppressing the production of thyroid-stimulating hormone, which may help reduce the risk of thyroid problems. It is administered orally in tablet form, with each tablet containing 25 micrograms of the active ingredient.

Study on the Effectiveness of Bevacizumab and Drug Combination for Children with Recurrent or Progressive Medulloblastoma, Ependymoma, and ATRT

This study explores treatments for children with tumors that have returned or are getting worse. It tests a combination of medications that target the blood vessels supplying the tumor, known as anti-angiogenesis therapy.

Main inclusion criteria: Patients must have recurrent or progressive disease with at least one untreated site. The original diagnosis or relapse must be confirmed through tissue examination. Participants can be male or female and must be under 20 years old at the time of original diagnosis. Normal organ and bone marrow function is required, including specific levels of liver enzymes, kidney function markers, white blood cells, and platelets. If certain blood counts are low, treatment will start at a reduced dose. Patients must have a performance status score of at least 50, measuring their ability to perform daily activities. Written informed consent from patients and parents is required.

Main exclusion criteria: The trial excludes patients who do not have recurrent or progressive medulloblastoma, ependymoma, or atypical teratoid rhabdoid tumor. Those not within the specified age range or who are considered part of a vulnerable population requiring special protection cannot participate.

Study focus: The trial aims to determine how well these treatments work in stopping tumor growth by continuously attacking cancer cells over time while minimizing side effects. The approach involves giving lower doses of chemotherapy drugs more frequently, known as metronomic therapy. The study also uses anti-angiogenic therapy to stop the formation of new blood vessels that tumors need to grow, essentially starving the tumor by cutting off its blood supply.

Investigational drugs: The medications being tested include cytarabine, celecoxib, cyclophosphamide, etoposide, bevacizumab, fenofibrate, and thalidomide. Some are given as injections while others are taken orally as capsules. The study monitors how tumors respond to treatment, whether they shrink, stay the same, or grow, and how long patients remain free of tumor growth.

Study on Medulloblastoma Treatment in Children Aged 3-5 Using Carboplatin and Drug Combination

This trial explores different treatment approaches for children with various risk levels of the disease. The treatments being tested include combinations of radiation therapy and chemotherapy tailored to specific risk levels and biological characteristics.

Main inclusion criteria: Patients should be at least 3 to 5 years old at diagnosis, depending on the country, and less than 16 years for some groups or less than 22 years for others. They must have confirmed disease through tests. For certain groups, the tumor must not have specific genetic changes in genes like TP53, PTCH, SUFU, BRCA2, and PALB2. Patients must not have received any previous treatment except for surgery, and must not have significant hearing problems. Post-surgery treatment should start within 28 days and no later than 40 days after surgery. Normal liver, kidney, and blood function is required. High-quality tumor and blood samples must be provided for research, and written consent from patients or guardians is necessary.

Main exclusion criteria: Patients with a different type of cancer, those outside the specified age range, or those not meeting the standard-risk or high-risk biological profile criteria cannot participate. Patients with genetic mutations not part of the study’s focus, those unable to undergo required treatments, those unable to follow study procedures or attend regular follow-up visits, pregnant or breastfeeding patients, and those with other serious health conditions that could interfere with treatment are excluded.

Study focus: The study aims to determine the effectiveness of different treatment combinations in improving survival rates. Participants are divided into different groups based on risk level and biological characteristics. Some groups receive a combination of radiation and chemotherapy, while others receive chemotherapy alone. The study assesses event-free survival (the length of time participants remain free from cancer-related events) and overall survival.

Investigational drugs: The trial tests carboplatin, methotrexate, cisplatin, cyclophosphamide, doxorubicin hydrochloride, vinblastine sulfate, vincristine, and lomustine. These drugs are administered in different forms including solutions for infusion or injection and capsules. The study also examines different radiation therapy approaches, including neuraxis irradiation and boost to the primary tumor, along with reduced-intensity chemotherapy and modified maintenance chemotherapy for different risk groups.

Study on the Safety and Effectiveness of Methotrexate and Drug Combination for Children and Young Adults with High-Risk Metastatic Medulloblastoma

This trial focuses on studying the safety and effectiveness of chemotherapy combined with radiation therapy for patients with metastatic disease and other similar types of aggressive brain tumors that can spread to other parts of the brain and spinal cord.

Main inclusion criteria: Patients must have a first-time diagnosis of high-risk disease or other embryonal tumors according to the WHO 2016 classification. They must not have received any previous treatment like chemotherapy or radiotherapy, except for surgery. Written informed consent must be obtained from patients, parents, or legal representatives. Patients must be available for treatment and able to follow study rules and procedures. Both males and females aged over 3 years and under 21 years at diagnosis can participate. Patients must have a life expectancy of 12 months or more and a Karnofsky/Lansky score of 40% or higher. Adequate blood cell levels, liver function, and kidney function are required.

Main exclusion criteria: Patients with a different type of cancer than those being studied, those outside the specified age range for children and young adults, those unable to undergo the tested treatments, and those not considered part of the vulnerable population selected for the study are excluded.

Study focus: The study evaluates the safety of using both standard and high doses of chemotherapy drugs along with radiation therapy targeting the brain and spinal cord. Researchers track participants’ progress including progression-free survival and overall survival. The study also monitors neurological and psychological health and any other health issues that may arise from treatment.

Investigational drugs: The medications include methotrexate, carboplatin, lomustine, cyclophosphamide, etoposide, thiotepa, and vinorelbine. Most are given through an intravenous line, except for lomustine which is taken orally. The trial tests both standard chemotherapy and high-dose chemotherapy approaches, followed by craniospinal irradiation to eliminate any remaining cancer cells.

Study on Personalized Treatment for Post-Pubertal Patients with Newly Diagnosed Medulloblastoma Using Sonidegib, Cisplatin, Lomustine, and Vincristine

This trial explores a personalized treatment approach based on patient age and specific tumor characteristics. It focuses on newly diagnosed patients who are post-pubertal or adults and involves adjusting radiation therapy and chemotherapy doses while introducing an investigative drug called sonidegib.

Main inclusion criteria: Patients must provide written informed consent before joining. Health should be stable within two weeks before enrollment, with a Karnofsky scale score between 50 and 100. The NANO-score for neurological function should be between 0 and 9. Patients should have standard-risk disease confirmed by MRI scans showing most of the tumor has been removed with no signs of spread. Full recovery from surgery or complications is required. Baseline MRI scans of the brain and spine are necessary. Normal liver, kidney, and blood functions within two weeks of joining are required. Women who can have children must have a negative pregnancy test and use two methods of birth control. Men must use condoms during treatment and for 12 months after. Tumor tissue and blood samples must be available for review. The tumor must be of a specific molecular subtype and not have spread beyond certain areas.

Main exclusion criteria: Patients without a diagnosis, those not in the SHH-activated subgroup, those who are not post-pubertal, and those who do not have a standard risk level cannot participate.

Study focus: The trial compares personalized treatment to standard therapy in patients with a specific type called SHH-activated. The main goal is to see if the personalized treatment can help patients live longer without the cancer getting worse. The study involves regular monitoring and assessments to track disease progress and any side effects. Participants undergo various tests including MRI scans to evaluate tumor response. The trial also assesses the impact on quality of life and cognitive functions.

Investigational drugs: Sonidegib is the experimental medication being tested. Patients also receive cisplatin administered as a concentrate for infusion, lomustine taken orally, and vincristine given as an injection. A placebo group is included to help determine the effectiveness of the new treatment approach. Sonidegib works by inhibiting the Hedgehog signaling pathway, which is involved in cancer cell growth and spread.

Study on Ribociclib, Topotecan, and Temozolomide for Children and Young Adults (12 months – 21 years) with Relapsed or Refractory Neuroblastoma and Solid Tumors

This trial studies treatment for children and young adults aged 12 months to 21 years with relapsed or refractory neuroblastoma and other solid tumors. The study uses a combination of medications including ribociclib, temozolomide, and topotecan hydrochloride to see if they can effectively treat these types of cancers.

Main inclusion criteria: Participants must sign an informed consent document, with guardians signing for children. Age must be between 12 months and 21 years. Patients must have certain types of solid tumors that have not responded to standard treatments or for which no effective standard treatment exists, including neuroblastoma and medulloblastoma. Those with central nervous system disease taking corticosteroids must be on a stable dose for at least 7 days. Patients must have measurable disease and be able to sit upright in a wheelchair. Life expectancy must be at least 12 weeks. Adequate bone marrow and organ function is required, including specific levels of neutrophils, platelets, hemoglobin, normal bilirubin levels, normal liver and kidney function, normal heart function, and normal levels of potassium, magnesium, and calcium.

Main exclusion criteria: Patients who have not recovered from previous treatments or surgeries, those with other serious health conditions, pregnant or breastfeeding patients, those unable to follow study procedures, those who participated in another clinical trial recently, those with allergies to study medications, those with uncontrolled infections, those with certain heart problems, those taking interfering medications, and those with a history of drug or alcohol abuse are excluded.

Study focus: The study is divided into two phases. In the first phase, researchers determine the best dose of ribociclib to use with the other medications. In the second phase, the study evaluates how well this combination works compared to a placebo. Participants receive medications either orally or through intravenous infusion and are closely monitored for side effects and tumor response.

Investigational drugs: Ribociclib works by blocking proteins that cancer cells need to grow and divide. Topotecan is a chemotherapy drug that interferes with cancer cell DNA to prevent growth and multiplication. Temozolomide damages cancer cell DNA to stop growth and spread. The trial tests these medications in combination to determine if they can improve treatment outcomes.

Summary

The 7 ongoing clinical trials for medulloblastoma reflect a comprehensive approach to treating this complex brain tumor across different patient populations and disease stages. Several notable patterns emerge from these studies.

Geographically, Germany, Italy, and Spain show the highest concentration of trials, each hosting four or more studies. This suggests these countries have established strong research infrastructure for pediatric brain tumor treatment. The trials are predominantly focused on pediatric patients, with most studies targeting children aged 3 years and older, though one trial extends eligibility to young adults up to 21 years.

A significant focus exists on high-risk disease, with multiple trials specifically designed for patients with aggressive tumor characteristics or metastatic spread. The treatment approaches vary considerably, ranging from intensive multi-drug chemotherapy regimens combined with radiation therapy to more targeted personalized treatments based on molecular subtypes such as SHH-activated tumors.

Common chemotherapy agents appearing across multiple trials include carboplatin, cisplatin, lomustine, and vincristine, suggesting these remain important components of treatment protocols. Several trials are exploring innovative approaches such as high-dose chemotherapy with thiotepa, metronomic therapy combined with anti-angiogenic agents, and targeted drugs like sonidegib and ribociclib.

Radiation therapy remains a central component of most protocols, with some trials comparing different radiation approaches including conventional versus hyperfractionated-accelerated techniques. One trial takes a unique approach by focusing on preventing treatment-related side effects, specifically hypothyroidism, rather than directly targeting the tumor.

The trials generally require patients to have adequate organ function and acceptable performance status, with most excluding those who have received prior cancer treatment. This emphasis on treatment-naive patients suggests these studies are working to establish optimal first-line treatment strategies for different disease presentations.

Ongoing Clinical Trials on Medulloblastoma

  • Study of drug combination therapy for high-risk medulloblastoma in patients over 3 years of age: carboplatin, cisplatin, cyclophosphamide, lomustine, temozolomide, thiotepa, vincristine

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark Finland Germany +4

  • Study on Preventing Hypothyroidism in Patients with Medulloblastoma and Pediatric Lymphoma Using Levothyroxine During Radiation Therapy

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on the Effectiveness of Bevacizumab and Drug Combination for Children with Recurrent or Progressive Medulloblastoma, Ependymoma, and ATRT

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Czechia Denmark France Norway Spain +1

  • Comparing two treatment regimens with drug combination for newly diagnosed low-risk medulloblastoma to evaluate cognitive function outcomes

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Denmark France Germany The Netherlands

  • Study on Medulloblastoma Treatment in Children Aged 3-5 Using Carboplatin and Drug Combination

    Not yet recruiting

    4 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Finland France Germany +4

  • Study on the Safety and Effectiveness of Methotrexate and Drug Combination for Children and Young Adults with High-Risk Metastatic Medulloblastoma

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on Ribociclib, Topotecan, and Temozolomide for Children and Young Adults (12 months – 21 years) with Relapsed or Refractory Neuroblastoma and Solid Tumors

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark France Germany Hungary Italy +1

  • Study on Personalized Treatment for Post-Pubertal Patients with Newly Diagnosed Medulloblastoma Using Sonidegib, Cisplatin, Lomustine, and Vincristine

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

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