Active not recruitingPHASE2INTERVENTIONAL

A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma

This study is for children and young adults with a type of brain cancer called medulloblastoma. Traditionally, treatment depended on how much cancer was left after surgery. However, we now know that medulloblastoma has different types, called 'molecular subgroups', that respond differently to treatment. This trial combines both the amount of cancer left and these molecular subgroups to create more personalised treatment plans. The goal is to see if lower doses of radiation and chemotherapy can be used for some patients, reducing side effects while maintaining cure rates. For others, it aims to find out if adding new or targeted medicines improves their chances of recovery.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

St. Jude Children's Research Hospital

Enrolment target

660

Start

23 Jun 2013

Estimated completion

13 Oct 2031

Medulloblastoma

What is this study about?

This research study is about finding better, more personalised ways to treat medulloblastoma, a type of brain cancer that mainly affects children and young adults. In the past, doctors generally decided on treatment based on how much of the tumour remained after surgery. However, we now understand that not all medulloblastomas are the same. In fact, they can be categorised into different 'molecular subgroups' based on their unique biological characteristics. These different subgroups can react differently to various treatments, meaning a one-size-fits-all approach might not be the most effective for everyone.

This study aims to combine these two important pieces of information – the amount of tumour left after surgery (called clinical risk) and the specific molecular subgroup of the tumour – to guide treatment decisions. By doing this, we hope to create treatment plans that are much more tailored to each individual patient. This personalised approach could mean that some patients, particularly those with a lower-risk type of medulloblastoma, might be able to receive less intense radiation and chemotherapy. The hope is that this will reduce difficult side effects while still achieving excellent results.

For other patients, especially those with more challenging forms of medulloblastoma, the study will explore whether adding new or targeted medications to standard treatments can improve their chances of getting better. Every patient in this study will have surgery to remove as much of the tumour as possible, followed by radiation therapy and chemotherapy. The exact type and amount of these treatments will depend on their specific clinical risk and molecular subgroup, as determined by tests on their tumor tissue.

Key takeaways

This study is exploring personalised treatment for medulloblastoma.
Treatment plans are based on your tumour's unique features and how much is removed.
It aims to reduce treatment intensity for some, and improve outcomes for others.
Surgery, radiation, and chemotherapy are part of all treatment plans.
Optional exercise and memory programs might be offered for some.

Who may be eligible?

This study is looking for patients diagnosed with medulloblastoma.

To join, participants generally need to be between 3 and 21 years old at the time they were diagnosed. However, if you are between 22 and 39 years old and have a specific type of medulloblastoma called SHH, you might also be able to join. It's important that you haven't had any previous radiation, chemotherapy, or other treatments for your brain tumour before starting this study, apart from certain steroid medications or surgery. You will also need to be able to start the study's treatment plan within 36 days of your surgery.

There are also some things that would mean you couldn't join. For example, if you have a different type of brain tumour, or other serious health problems that might make the study treatment unsafe for you. Females who could become pregnant must not be pregnant or breastfeeding and will need a negative pregnancy test.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have medulloblastoma?
Were you diagnosed between 3 and 21 years old (or 22-39 with a specific SHH type)?
Have you had no previous radiation or chemotherapy for your brain tumour?
Can you start treatment within 36 days of your surgery?
Are you able to manage daily activities reasonably well?

Answer every question to see your result.

What does participation involve?

If you join this study, you will first have surgery to remove as much of the tumour as possible. After surgery, your tumour tissue will be analysed to determine its specific molecular subgroup and combined with other factors to assign you to a specific treatment group. Every participant will receive a combination of radiation therapy to the brain and spine, and chemotherapy specific to their group. The type and amount of radiation and chemotherapy will vary depending on your assigned group.

Some groups might test lower doses of treatment or add new medications. For example, some participants with an SHH tumour might receive an additional targeted chemotherapy drug called vismodegib after their main chemotherapy is completed. The study also includes optional activities for some participants, such as supervised aerobic training (like exercise) during radiation and a computer program to help with memory after chemotherapy. You will have regular hospital visits for assessments, scans, and to receive your treatments. The total duration of your participation will depend on your assigned treatment plan and follow-up schedule.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and potential risks. A potential benefit of this study is receiving a treatment plan specifically tailored to your tumour's unique features, which might lead to better outcomes or fewer side effects compared to standard treatments. For some, it might mean access to newer medications not yet widely available. However, there are also risks associated with radiation and chemotherapy, including side effects like fatigue, nausea, hair loss, and potential long-term issues. The targeted drugs also have their own potential side effects. The research team will explain all known risks in detail. Always remember you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (23)

Lucille Packard Children's Hospital at Stanford University Medical Center
Verified postcode
Palo Alto, United States
Rady Children's Hospital
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San Diego, United States
Yale University
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New Haven, United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
University of Florida
Verified postcode
Gainesville, United States
Arnold Palmer Hospital for Children
Verified postcode
Orlando, United States
Children's Hospital and Clinics of Minnesota
Verified postcode
Minneapolis, United States
Duke Children's Hospital and Health Center
Verified postcode
Durham, United States
Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Medical University of South Carolina
Verified postcode
Charleston, United States
St. Jude Children's Research Hospital
Verified postcode
Memphis, United States
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States

Common questions

What is medulloblastoma?

Medulloblastoma is a type of cancerous brain tumour that usually starts in the lower back part of the brain, called the cerebellum. It is one of the most common brain tumours in children.

What does 'molecular subgroup' mean?

It means doctors look closely at the tumour's genes and features to understand its specific type. This helps them predict how it might behave and what treatments might work best.

Why is this study important?

This study is important because it's trying to make medulloblastoma treatment more personal. By matching the treatment to the specific type of tumour, we hope to improve patients' chances of getting well while also reducing side effects.

Will I get less treatment if I join this study?

For some patients with certain types of medulloblastoma, the study is testing if lower doses of radiation and chemotherapy can be used effectively. For other types, it might involve adding new medicines.

Can I choose which treatment I get?

No, your treatment group will be decided by your tumour's specific features and how much of it remains after surgery. This ensures the treatment is tailored based on the research plan.