RecruitingPHASE2INTERVENTIONAL

DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

This study is investigating a drug called Difluoromethylornithine (DFMO) for children and young adults (up to 21 years old) who have medulloblastoma, a type of brain tumour. It's for patients whose tumour is considered high-risk or very high-risk, or if it has come back after previous treatments. The study aims to see if taking DFMO daily for about two years can act as a "maintenance therapy" – a treatment given to help prevent the cancer from returning after initial successful treatment. This is an open-label study, meaning both doctors and patients will know they are receiving DFMO. It will involve three different groups of patients, depending on their specific tumour type and situation.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Giselle Sholler

Enrolment target

118

Start

29 Mar 2021

Estimated completion

01 Mar 2029

Medulloblastoma

What is this study about?

This study is looking into a medication called Difluoromethylornithine, or DFMO for short. It's for children and young adults up to 21 years old who have a type of brain tumour called medulloblastoma. The study is particularly interested in patients whose tumour is categorised as 'high-risk' or 'very high-risk', or if their tumour has returned after previous treatments. The main goal is to see if DFMO can be used as a 'maintenance therapy'. This means giving the drug after the main treatment for the cancer is finished, with the hope that it will help stop the cancer from coming back.

Medulloblastoma is a serious brain tumour, and doctors are always looking for new ways to improve treatments and prevent the cancer from recurring. This study is an 'open-label' one, meaning everyone involved – patients, families, and doctors – will know that DFMO is being given. There's no ‘placebo’ group in this particular study where people wouldn't know if they were getting active treatment.

Researchers will divide the patients into three groups based on the specific characteristics of their medulloblastoma. This allows them to study how DFMO works in different situations. By carefully watching how patients respond over two years, they hope to learn if DFMO can offer new hope in keeping this type of cancer away.

Key takeaways

This study is for children and young adults (0-21) with medulloblastoma.
It tests a drug called DFMO as a 'maintenance' treatment to prevent cancer recurrence.
DFMO is taken by mouth twice daily for about two years.
Participants will be closely monitored with regular check-ups and scans.
The study is for specific types of medulloblastoma: high-risk, very high-risk, or if it has relapsed.

Who may be eligible?

To be considered for this study, patients must be between 0 and 21 years old when they were first diagnosed with medulloblastoma. Doctors will need to confirm the exact type of medulloblastoma using special tests. This includes looking at specific features of the tumour to place it into a risk group (high-risk, very high-risk, or if it has come back).

Before starting the study, patients will need to have had recent scans of their brain and spine to check the tumour status. Importantly, patients should either have no signs of active cancer, or any remaining tumour should be small and stable (not growing). There are also specific timeframes for when previous cancer treatments must have finished before a patient can join this study.

Patients also need to be reasonably well enough to take part. This is checked using a special scale (Lansky or Karnofsky score), and they must have an expected lifespan of at least two months. Generally, if they've recently had chemotherapy, the study drug should start no more than 60 days afterwards.

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.

Are you/the patient aged 0 to 21 years at diagnosis?
Has medulloblastoma been diagnosed and confirmed with specific molecular tests?
Is the medulloblastoma considered high-risk, very high-risk, or has it returned?
Have brain and spine scans confirmed the tumour is gone or very small and stable?
Have you/the patient finished previous chemotherapy treatments within the last 60 days (or specific time after stem cell transplant)?
Are you/the patient generally well enough to participate in the study activities?

Answer every question to see your result.

What does participation involve?

If you join this study, you would take the study drug, DFMO, twice a day by mouth for a total of 730 days, which is about two years. The dose of the drug will be calculated based on your body size.

Throughout the study, you will have regular check-ups to see how you are doing and to monitor your health. This will involve appointments where doctors will assess your condition. You will also have scans and other tests to keep an eye on the medulloblastoma. The study aims to enrol 118 patients in total, across the three different patient groups. Once the treatment period is over, there will likely be follow-up appointments to continue monitoring your health and the long-term effects.

Potential risks and benefits

Participating in any clinical study comes with potential benefits and risks. A potential benefit of this study is that DFMO might help to prevent medulloblastoma from recurring, especially for those at higher risk. This could offer a new therapy option. However, there are also potential risks, as DFMO, like all medicines, can have side effects. Doctors will discuss these with you, and your health will be closely monitored throughout the study to manage any side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (24)

Arkansas Children's Hospital
Verified postcode
Little Rock, United States· Recruiting
UCSF Benioff Children's Hospital Oakland
Verified postcode
Oakland, United States· Recruiting
Rady Children's Hospital
Verified postcode
San Diego, United States· Recruiting
Stanford University
Verified postcode
Stanford, United States· Recruiting
Connecticut Children's Hospital
Verified postcode
Hartford, United States· Recruiting
Nicklaus Children's Hospital
Verified postcode
Miami, United States· Recruiting
Arnold Palmer Hospital for Children
Verified postcode
Orlando, United States· Recruiting
All Children's Hospital Johns Hopkins Medicine
Verified postcode
St. Petersburg, United States· Recruiting
St. Joseph's Children's Hospital
Verified postcode
Tampa, United States· Recruiting
Kapiolani Medical Center for Women and Children
Verified postcode
Honolulu, United States· Recruiting
Advocate Aurora Research Institute
Verified postcode
Chicago, United States· Recruiting
Kentucky Children's Hospital
Verified postcode
Lexington, United States· Recruiting

Common questions

What is medulloblastoma?

Medulloblastoma is a type of cancerous brain tumour that usually starts in the back lower part of the brain called the cerebellum, which controls balance and coordination.

What does 'maintenance therapy' mean?

Maintenance therapy is a treatment given for an extended period after initial intensive treatments have successfully treated the main cancer. The goal is to keep the cancer from coming back.

What is DFMO?

DFMO is a drug being studied to see if it can help stop cancer cells from growing. It is not currently a standard treatment for medulloblastoma but is being investigated in this trial.

Will I know if I'm getting the drug?

Yes, this is an 'open-label' study, which means everyone involved – patients, families, and doctors – will know that DFMO is being given.

How long will I take the DFMO?

If you join this study, you would take DFMO by mouth twice a day for approximately two years (730 days).

How to find out more

BCC Enroll

bccenroll@pennstatehealth.psu.edu 7175310003 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.