Active not recruitingPHASE1INTERVENTIONAL

HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children With Ependymoma

This study is a Phase 1 trial for children aged 1-21 with recurrent or progressive ependymoma, a type of brain tumour. It involves a new immune therapy called HER2 CAR T cells, where a patient's own immune cells are specially trained to find and destroy cancer cells. The main goal is to check how safe this treatment is and identify any side effects. Researchers will also be looking at how well it works against the tumour. An important part of the study is to see if it's practical to offer this treatment at different hospitals. Some patients might also have surgery so researchers can examine how the new treatment affects their tumour tissue.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Children's Oncology Group

Enrolment target

Start

27 Jul 2022

Estimated completion

31 Mar 2033

Ependymoma

What is this study about?

This study is exploring a new way to treat a type of brain tumour in children called ependymoma. This treatment is being considered for children whose ependymoma has come back after previous treatments, or hasn't responded well to them. It's a 'Phase 1' study, which means it's one of the first times this treatment is being tested in children, so the main aim is to carefully check if it's safe and what side effects it might cause. Researchers will also observe if the treatment helps to shrink or control the tumour.

The new treatment uses your child's own immune cells, called T cells. These cells are taken from a blood sample and then specially changed in a lab to become 'HER2 CAR T cells'. These modified cells are like tiny, smart soldiers that are designed to find and attack specific proteins (HER2) on the surface of the ependymoma tumour cells. By targeting HER2, these engineered T cells aim to destroy the cancer while leaving healthy cells alone.

Another important aspect of this research is to figure out if it's practical to deliver this type of complex CAR T cell therapy across different hospitals. This helps to see if it could eventually become a more widely available treatment option for children if it proves to be safe and effective. Some children in the study might also have surgery to remove their tumour, and researchers will examine these samples to understand better how the HER2 CAR T cells work inside the body.

Key takeaways

This is a new immune cell therapy for children with recurrent or progressive ependymoma.
It uses your child's own T cells, specially trained to fight the tumour.
The main goal is to check how safe this new treatment is for children.
Researchers will also see if the treatment helps to control the tumour.
Your child must be between 1 and 21 years old and have specific tumour features.
Participation involves treatment, regular check-ups, and long-term follow-up.

Who may be eligible?

To be considered for this study, your child must have been diagnosed with ependymoma that has either returned after being treated or has continued to grow despite treatment. They must have already received standard treatments for their condition, such as surgery and local radiotherapy.

Your child needs to be between 1 and 21 years old. Doctors will also need to check if there's enough of their previous tumour tissue available to test for a specific protein called HER2. This protein helps researchers determine if the CAR T cell therapy would be suitable for your child.

Unfortunately, children who are known to have HIV would not be able to join the study due to concerns about the safety of this treatment process with their immune system. Also, if there isn't enough tumour tissue to test for HER2, your child wouldn't be able to participate.

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.

Is my child between 1 and 21 years old?
Has my child been diagnosed with ependymoma that has come back or grown?
Has my child already had standard treatments like surgery and radiotherapy?
Is there enough of my child's previous tumour tissue available for testing?
Does my child NOT have HIV?

Answer every question to see your result.

What does participation involve?

If your child joins the study, they will first have chemotherapy to prepare their body for the new CAR T cells. Then, they will receive the HER2 CAR T cells through a drip into a vein. After this first treatment, they will have a break for about 8 to 12 weeks. If they are recovering well, they might receive up to two more doses of the CAR T cells. You would be expected to stay in the study for about 9 months while receiving treatment.

Some children, after the safety of the initial treatments is known, might also be part of a 'surgical study'. This means they would get one dose of the CAR T cells about 4 to 6 weeks before a planned surgery to remove their tumour. During the surgery, doctors would take samples of the tumour to see how the CAR T cells have affected it. After recovering from surgery, they could also receive more doses of the CAR T cells. All participants will be followed up for a very long time, up to 15 years, to monitor their health.

Potential risks and benefits

Participating in this study might offer the potential benefit of a new treatment for your child's ependymoma that has not responded well to other therapies. As with any new treatment, there are potential risks, primarily side effects from the CAR T cells or the preparation chemotherapy, which the medical team will monitor very closely. It's important to remember that this is a Phase 1 study, so the safety of the treatment is still being established. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing care.

Locations (13)

Children's Hospital Los Angeles
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Los Angeles, United States
Children's Hospital Colorado
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Aurora, United States
Children's National Medical Center
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Washington D.C., United States
University of Florida
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Gainesville, United States
Children's Healthcare of Atlanta
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Atlanta, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
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Chicago, United States
Dana Farber Cancer Institute
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Boston, United States
Memorial Sloan Kettering Cancer Center
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New York, United States
Cincinnati Children's Hospital Medical Center
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Cincinnati, United States
Nationwide Children's Hospital
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Columbus, United States
Pittsburgh Children's Hospital of UPMC
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Pittsburgh, United States
Texas Children's Cancer Center
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Houston, United States

Common questions

What is ependymoma?

Ependymoma is a type of tumour that grows in the brain or spinal cord, most commonly affecting children.

What are CAR T cells?

CAR T cells are immune cells from a patient's own body that have been specially trained in a lab to find and destroy cancer cells.

What is 'Phase 1' of a study?

Phase 1 is the first stage where a new treatment is tested in people. The main goal is to check its safety and find the right dose.

Will my child definitely get the CAR T cells?

If your child meets all the criteria, they will receive the CAR T cells outlined in the study plan.

How long will we be involved in the study?

Your child will receive treatment for about 9 months, and then be followed up by the study team for 15 years to track their health.