Active not recruitingPHASE1INTERVENTIONAL

Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

This study is for children aged 6 months to 16 years with Acute Myeloid Leukaemia (AML) that has come back. It's testing a new treatment called 'BE CAR-33', which uses special white blood cells (T cells) from a healthy donor. These T cells are altered using a new method called Base Editing to help them find and kill leukaemia cells. The goal is to safely reduce the amount of leukaemia in the body before a planned bone marrow transplant. By doing this, doctors hope to make the transplant more successful and lower the chance of the leukaemia coming back. Participants will receive a single dose of 'BE CAR-33' and be carefully checked for safety and how well the treatment is working.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Great Ormond Street Hospital for Children NHS Foundation Trust

Enrolment target

Start

21 Jul 2023

Estimated completion

01 Jun 2026

Relapsed Acute Myeloid Leukaemia

What is this study about?

This study is looking at a new treatment for children and young people aged 6 months to 16 years who have Acute Myeloid Leukaemia (AML). This is a type of blood cancer where abnormal white blood cells grow quickly in the bone marrow. In this study, we are focusing on AML that has returned after previous treatments.

The new treatment involves using special white blood cells called T cells. These T cells are taken from a healthy donor and are then modified in a lab using a new technique called 'Base Editing'. This technique changes their natural code (DNA) to make them better at finding and killing leukaemia cells. These specially prepared cells are called 'BE CAR-33' T cells. They are 'ready-made', meaning they come from a donor rather than the patient themselves.

The main aim of this study is to see if 'BE CAR-33' T cells are safe to use and if they can effectively reduce the amount of leukaemia in a child's body before they have a bone marrow transplant. A bone marrow transplant is a common treatment for AML, and if we can get rid of more leukaemia cells beforehand, it might make the transplant more successful and help prevent the cancer from coming back.

Key takeaways

This study is for children (6 months to 16 years) with relapsed AML.
It tests a new 'ready-made' cell therapy called 'BE CAR-33'.
The goal is to reduce leukaemia before a bone marrow transplant.
Participants will have chemotherapy, a single cell infusion, and close monitoring.
There are potential benefits in improving transplant success, but also risks like infection and immune reactions.
Long-term follow-up is part of the study.

Who may be eligible?

This study is for children and young people with Acute Myeloid Leukaemia (AML) that has returned. They must be between 6 months and almost 16 years old. They also need to be planning to have a bone marrow transplant and have a suitable donor available.

There are also some medical checks. For example, a certain level of leukaemia cells needs to be found in the bone marrow, and the leukaemia cells need to have a specific marker (called CD33) on their surface. Patients must also be well enough to take part, with a life expectancy of at least 12 weeks.

Some things would mean a child couldn't join. This includes not being willing to take part in a long follow-up (up to 15 years), having uncontrolled infections, or having other serious health problems that might make the treatment unsafe. Children weighing less than 6kg or those who have had certain severe reactions to donated cells in the past would also not 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 6 months and almost 16 years old?
Has my child's Acute Myeloid Leukaemia returned?
Is my child planning to have a bone marrow transplant soon, and is a suitable donor available?
Are we prepared for follow-up appointments for up to 15 years?
Does my child meet general health requirements to participate in a study?
Does my child not have any uncontrolled infections or other severe health issues currently?

Answer every question to see your result.

What does participation involve?

If you or your child are eligible and choose to take part, there will be several steps. First, there will be careful checks to make sure the treatment is right for your child. Before receiving the 'BE CAR-33' cells, your child will have some chemotherapy. This helps prepare their body for the new cells.

Then, your child will receive a single infusion (like a drip) of the 'BE CAR-33' cells. They will then stay in the hospital for about 5 weeks. During this time, the medical team will closely monitor them using blood and bone marrow tests. These tests help ensure their safety, check how well the 'BE CAR-33' cells are working, and see how the leukaemia is responding. The planned bone marrow transplant will happen about 4 weeks after your child finishes the 'BE CAR-33' treatment. After going home, doctors will continue to monitor your child's health with monthly check-ups for the first three months, then every 6 months for a longer period.

Potential risks and benefits

Taking part in this study could offer some benefits. By trying to reduce the amount of leukaemia in the body before a bone marrow transplant, there's a chance the transplant could be more successful and prevent the cancer from returning. However, there are also potential risks and side effects. These can include a drop in healthy blood cells, a higher risk of infections, and a severe reaction where the body's immune system overreacts (called 'cytokine storm'). There's also a risk called 'graft versus host disease', where the donated cells might attack your child's healthy tissues. You have the right to withdraw from the study at any time, even if you initially agree to participate.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Great Ormond Street Hospital for Children
Verified postcode
London, United Kingdom

Common questions

What is AML?

AML stands for Acute Myeloid Leukaemia. It's a type of cancer that starts in the bone marrow, where new blood cells are made.

What are 'BE CAR-33' cells?

These are special white blood cells from a healthy donor that have been changed in the lab to target and kill leukaemia cells more effectively.

Why is this treatment given before a bone marrow transplant?

The hope is that by getting rid of more leukaemia cells beforehand, the bone marrow transplant will have a better chance of success and keep the leukaemia away.

How long will my child be in the hospital?

Your child is expected to be in the hospital for about 5 weeks for the 'BE CAR-33' treatment, with the bone marrow transplant scheduled about 4 weeks after that.

Can we stop participating at any time?

Yes, you have the right to withdraw your child from the study at any time, for any reason.