A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)
This study is testing a new cell therapy, called AUTO1, for children and teenagers under 18 with specific types of blood cancer: B-cell acute lymphoblastic leukaemia (B-ALL) and aggressive B-cell non-Hodgkin lymphoma (B-NHL). These are cancers that haven't improved with other treatments, or have come back. The therapy involves taking some of a patient's own immune cells, arming them to fight cancer, and then giving them back. This study is an early-stage trial (Phase 1b/2) to find out how safe AUTO1 is and how well it works. Researchers will carefully watch participants for any side effects and see if the treatment helps shrink or control their cancer.
At a glance
What is this study about?
This study is for children and teenagers who have particular types of blood cancer. These are B-cell acute lymphoblastic leukaemia (B-ALL) and B-cell non-Hodgkin lymphoma (B-NHL). The doctors are especially interested in patients where the cancer has come back after treatment, or hasn't responded to standard treatments. This means that current treatments haven't worked for them.
The experimental treatment being tested is called AUTO1 (also known as obe-cel). It's a type of 'CAR T-cell therapy'. This is an exciting new approach where a patient's own immune cells are collected, specially changed in a lab to recognise and attack cancer cells, and then given back to the patient. Think of it like training your body's own defence system to be super smart at finding and fighting cancer.
Because this is an early study (a 'Phase 1b/2' trial), the main goals are to carefully check how safe this new therapy is for young people and to see how effective it is in treating their cancer. Researchers will be monitoring the children very closely for any side effects and will also be looking to see if the treatment helps to control or get rid of the cancer.
Key takeaways
- This study is for children and teens with specific blood cancers that haven't responded to other treatments.
- It tests a new type of 'CAR T-cell therapy' called AUTO1.
- The main goals are to check safety and how well the treatment works.
- Participation involves several steps, from cell collection to long-term follow-up.
- There are potential benefits and risks, and joining is always voluntary.
Who may be eligible?
This study is for children and teenagers under 18 years old who weigh at least 6 kilograms. They must have been diagnosed with B-cell acute lymphoblastic leukaemia (B-ALL) or a specific type of aggressive B-cell non-Hodgkin lymphoma (B-NHL) where previous treatments haven't worked or the cancer has returned.
To be considered, their cancer cells need to show a particular marker called CD19, which is like a flag that the new treatment is designed to recognise. Participants also need to be generally well enough to take part, with their bodies (like kidneys, heart, and lungs) working adequately, and they shouldn't have any serious ongoing infections.
Patients would not be suitable if they have certain other health problems, such as a different type of leukaemia called chronic myelogenous leukaemia, or certain brain conditions not related to their cancer. Also, if they've had a recent stem cell transplant (in the last 3 months) or certain previous treatments targeting CD19 (other than a specific medicine called blinatumomab), they might not be able to join. If they've had severe nerve issues from blinatumomab previously, they also wouldn't be eligible.
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 the patient under 18 years old?
- Does the patient have B-cell acute lymphoblastic leukaemia (B-ALL) or aggressive B-cell Non-Hodgkin lymphoma (B-NHL) that hasn't responded to other treatments?
- Are the patient's major organs (heart, lungs, kidneys, liver) working well?
- Has the patient NOT had a stem cell transplant in the last 3 months?
- Does the patient NOT have a severe ongoing infection?
- Has the patient NOT had severe nerve-related issues from a previous medicine called blinatumomab?
What does participation involve?
If you or your child decides to take part, there will be several steps. First, there's a 'screening' period where doctors will do tests to make sure the study is a good fit. Then, blood will be collected in a process called 'leukapheresis' – this is where specific immune cells are taken out of the blood. If needed, there might be a short 'bridging' treatment to manage the cancer while your cells are being prepared.
Before you or your child receives the AUTO1 treatment, there's a 'lymphodepletion' phase, which involves a short course of chemotherapy to prepare the body for the new cells. Then, the AUTO1 cells will be given through a vein. After this, doctors will carefully watch and evaluate the treatment's effects. There will be regular check-ups and monitoring over time – this is the 'follow-up' period – to track progress and any side effects. The total length of time you'd be involved in the study would include these steps and the follow-up, which can last for a significant period.
Potential risks and benefits
Locations (8)
- Children's Hospital of PhiladelphiaVerified postcodePhiladelphia, United States· Recruiting
- Methodist Children's HospitalVerified postcodeSan Antonio, United States· Recruiting
- Primary Children's HospitalVerified postcodeSalt Lake City, United States· Recruiting
- Hospital Vall d'HebronVerified postcodeBarcelona, Spain· Recruiting
- Hospital Nino JesusVerified postcodeMadrid, Spain· Recruiting
- Great Ormond Street Hospital for Children NHS Foundation TrustVerified postcodeLondon, United Kingdom· Recruiting
- Royal Manchester Children's HospitalVerified postcodeManchester, United Kingdom· Recruiting
- Great North Children's HospitalVerified postcodeNewcastle upon Tyne, United Kingdom· Recruiting
Common questions
What is CAR T-cell therapy?
It's a type of treatment that uses a patient's own immune cells, specially modified to find and attack cancer.
What types of cancer is this study for?
It's for specific types of blood cancer in children and teenagers: B-cell acute lymphoblastic leukaemia (B-ALL) and aggressive B-cell Non-Hodgkin lymphoma (B-NHL) that haven't responded to other treatments.
Is this treatment available to everyone?
No, AUTO1 is an experimental treatment only available to patients who meet the specific requirements of this research study.
What does a 'Phase 1b/2' study mean?
It means it's an early-stage study primarily focused on checking the treatment's safety, finding the best dose, and seeing if it shows signs of working.
How old do you need to be to join?
Participants must be under 18 years old but at least 0 years old.
How to find out more
Autolus Ltd
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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