RecruitingPhase IVInterventional

Goldilocks - determining if personalised drug monitoring of fludarabine in children and young adults with acute lymphoblastic leukaemia who are undergoing CAR-T therapy is feasible and can improve outcome whilst minimising toxicity

The 'Goldilocks' study aims to improve a major cancer treatment called CAR T-cell therapy for children and young adults with acute lymphoblastic leukaemia (ALL) in the UK. ALL is a type of blood cancer, and while many get better, some face relapse. CAR T-cell therapy is a promising option for these difficult cases, but a medicine called fludarabine, given before, needs to be at just the right level. Too little, and the cancer might come back; too much, and there could be more side effects. This study will check fludarabine levels in blood samples and adjust doses in real-time. The goal is to make sure each patient gets the 'just right' amount, helping the treatment be more effective and safer.

At a glance

Status

Recruiting

Phase

Phase IV

Sponsor

Newcastle upon Tyne Hospitals NHS Foundation Trust

Enrolment target

Start

01 Feb 2026

Estimated completion

31 Aug 2028

Acute lymphoblastic leukaemia

What is this study about?

Acute lymphoblastic leukaemia (ALL) is a type of blood cancer that mainly affects young people. While many children and young adults who get ALL get better, some sadly find their cancer comes back. When this happens, it can be very hard to treat. One of the newest and most hopeful treatments for these difficult cases is called CAR T-cell therapy. This special therapy involves taking some of the patient's own immune cells (called T-cells), modifying them in a lab to make them better at fighting cancer, and then putting them back into the body. Before CAR T-cell therapy can be given, patients need some chemotherapy to prepare their body. This usually involves medicines called fludarabine and cyclophosphamide. Research has shown that getting an 'optimal' or 'just right' amount of fludarabine is really important. If the level of fludarabine is too low, the cancer is much more likely to return within a year after CAR T-cell therapy. In fact, for many patients, the fludarabine levels aren't quite right. This study, nicknamed 'Goldilocks', aims to fix this. We want to check the fludarabine levels in each patient's blood in real-time and adjust their dose if needed. This way, we hope to ensure everyone gets the perfect amount of medicine, making their CAR T-cell therapy as effective and safe as possible. Improving this treatment could make a big difference for young patients and their families.

Key takeaways

Aims to make CAR T-cell therapy more effective for blood cancer.
Focuses on giving the 'just right' amount of a medicine called fludarabine.
Involves extra blood tests to check medicine levels.
May reduce the chance of cancer returning and limit side effects.
For children and young adults under 25 with acute lymphoblastic leukaemia.

Who may be eligible?

This study is looking for children and young adults who are under 25 years old. To join the study, you must be receiving two specific chemotherapy drugs, fludarabine and cyclophosphamide, as part of the preparation for your CAR T-cell therapy. This preparation treatment is given before the main CAR T-cell therapy.

There are a few reasons why someone might not be able to join. For example, if you are pregnant or breastfeeding, you would not be able to take part. Also, if you are already involved in another study that is testing a different CAR T-cell treatment, you wouldn't be eligible for this one. Your doctor will be able to check all these details for you.

Ultimately, if you or your legal guardian can understand and agree in writing to take part, and you meet the other criteria, you may be able to join.

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 under 25 years old?
Are you having fludarabine and cyclophosphamide before CAR T-cell therapy?
Are you able to agree to take part, or can your parent/guardian do so?
Are you not pregnant or breastfeeding?
Are you not already in another CAR T-cell study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the main thing it involves is extra blood tests. When you start your fludarabine treatment, blood samples will be taken on the first day. These samples will be sent to a special lab to measure the exact amount of fludarabine in your system. Based on these results, your doctors might adjust the dose of fludarabine you receive on the following days to make sure it's at the most effective level.

Good news is you'll already be in hospital and will have a special tube (called a central line) in place for your treatment. This means the extra blood samples can usually be taken at the same time as other routine blood tests, so it shouldn't cause much extra fuss or discomfort. The study will last for the duration of your fludarabine chemotherapy, which is typically a few days.

Potential risks and benefits

The main benefit of taking part in this study is the potential to improve your own CAR T-cell therapy. By making sure you get the 'just right' amount of fludarabine, the treatment might work better at fighting the leukaemia, reducing the chance of it coming back, and potentially leading to a cure. This could significantly improve your health and quality of life. The main risk involved is the discussions about the trial, which might cause some emotional stress for you and your family during what is already a very challenging time. There's also a very small risk to your privacy, but strict measures are in place to protect your personal information, using a study ID number instead of your name. You are always free to decide not to take part or to withdraw from the study at any time, without it affecting your medical care.

Locations (7)

Great North Children's Hospital
City only
Newcastle upon Tyne, England
Northern Centre for Cancer Care
City only
Newcastle upon Tyne, England
Royal Manchester Children's Hospital
Approximate
Manchester, England
Manchester Royal Infirmary
Approximate
Manchester, England
Uclh
Approximate
London, England
Great Ormond Street Hospital
Unverified
London, England
The Christie
Unverified
Manchester, England

Common questions

What is CAR T-cell therapy?

It's a special treatment where your own immune cells are changed to better fight cancer, then put back into your body.

What is fludarabine?

It's a strong medicine used before CAR T-cell therapy to prepare your body by reducing some of your immune cells.

Why is 'personalising' the fludarabine important?

Making sure you get the exact right amount can help the CAR T-cell therapy work better and reduce the risk of the cancer coming back or causing more side effects.

Will I have more injections?

You'll have extra blood tests, but these will often be taken at the same time as your routine blood tests and usually through a line already in place.

When does the study take place?

The study runs from September 2025 to September 2028 and mostly involves measuring medicine levels during your pre-CAR T-cell therapy chemotherapy.

How to find out more

Shelby Barnett

shelby.barnett@nhs.net +44 (0)191 208 6000 ext 4357 Official trial record

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