RecruitingPHASE1INTERVENTIONAL

Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma

This study, called MIGHTY, is exploring a new type of treatment called CAR T-cell therapy for young people aged 1 to 24. It's for those with certain sarcomas (rhabdomyosarcoma, Ewing sarcoma, or desmoplastic small round cell tumour) that have either returned or not improved with other treatments. The main goal is to see if this new cell therapy, called hBRCA84D CAR T cells, can be safely made and given to patients. Researchers will also check how well the treatment works and how patients feel after receiving it. This is a Phase 1 study, meaning it's an early step to understand its safety and how the body reacts to it.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

University College, London

Enrolment target

Start

23 Jul 2025

Estimated completion

01 Jul 2042

Sarcoma

What is this study about?

The MIGHTY study is testing a new way to fight certain types of cancer called sarcoma, specifically rhabdomyosarcoma, Ewing sarcoma, and desmoplastic small round cell tumour. These are cancers that have either come back after treatment or haven't responded to previous therapies. This study is for children, teenagers, and young adults aged between 1 and 24 years old. The new treatment involves using special cells from the patient's own body.

This new treatment is called CAR T-cell therapy. It's a type of 'advanced therapy' because it uses specially modified immune cells. In this study, doctors will take some of the patient's own white blood cells, called T cells, and modify them in a lab to recognise and attack the cancer cells. These modified cells are called hBRCA84D CAR T cells. The aim is to make the patient's immune system better at finding and destroying the cancer.

The main goals of this early-stage study (Phase 1) are to see if these modified T cells can be successfully created for each patient and safely given. The doctors will also carefully watch for any side effects and see how well these special cells grow and stay in the body after they are put back in. This will help them understand if this treatment could be a useful option for future patients.

Key takeaways

This study is testing a new cell therapy for certain sarcomas in young people.
It uses your own immune cells, specially modified to fight cancer.
The study is for ages 1-24 whose cancer has returned or didn't respond to other treatments.
It involves blood collection, chemotherapy, and then receiving the special cells.
You'll have close monitoring in hospital and long-term follow-up for up to 15 years.
This is an early-stage study to check safety and how the treatment works.

Who may be eligible?

This study is looking for children, teenagers, and young adults aged between 1 and 24 years old. You would need to have been diagnosed with rhabdomyosarcoma, Ewing sarcoma, or desmoplastic small round cell tumour, and the cancer must have come back or not responded to earlier treatments. Importantly, your tumour must also show a specific marker called B7-H3, which helps the treatment work, and the cancer needs to be visible on scans.

There are also some health requirements to make sure you can safely take part. For example, your heart, kidney, and overall health need to be good enough. Your doctor will check your medical history, including any past treatments or conditions like hepatitis or HIV. They'll also make sure you can have the necessary blood tests and procedures involved in preparing the treatment.

If you only have cancer cells found in your bone marrow without any visible tumours on scans, you wouldn't be able to join this study. Also, if you have active cancer in your brain or spinal cord, or certain other health conditions, you 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.

Are you between 1 and 24 years old?
Do you have rhabdomyosarcoma, Ewing sarcoma, or desmoplastic small round cell tumour that has returned or not responded to previous treatments?
Is your tumour visible on scans and does it have the B7-H3 marker?
Do you meet certain health requirements for your heart, kidneys, and blood counts?
Are you able to undergo a blood collection procedure?
Do you understand that this is a new, investigational treatment?

Answer every question to see your result.

What does participation involve?

If you join the study, the first step is to have a special blood collection procedure called 'leukapheresis'. This takes some of your white blood cells, which will then be sent to a lab to create the special CAR T cells. This process takes about 15 days.

Before you receive the CAR T cells, you will have a short course of chemotherapy over 4 days. This is done to prepare your body to receive the new cells. After this, you will get the hBRCA84D CAR T cells. You will stay in hospital for 2 to 4 weeks after receiving the cells so the medical team can closely monitor you.

Once you go home, you'll have regular check-ups for a year. These will be at 6 weeks after your treatment, and then every 3 months. After this first year, all patients will be followed up once a year for a longer period, up to 15 years, to see how you are doing in the long term, even if your cancer comes back.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. The potential benefit of this study is that you might receive a new treatment that could help fight your cancer, especially if other treatments haven't worked. However, because this is an early-stage study, we don't yet know how effective it will be or what all the side effects might be. There could be side effects from the chemotherapy and the CAR T cells, which your medical team will discuss with you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Great Ormond Street Hospital
Verified postcode
London, United Kingdom· Recruiting
University College London Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What kind of cancer is this study for?

This study is for specific types of sarcoma: rhabdomyosarcoma, Ewing sarcoma, and desmoplastic small round cell tumour.

Who can take part in this study?

Children, teenagers, and young adults aged 1 to 24 with these types of sarcoma that have returned or not responded to previous treatments.

What is CAR T-cell therapy?

It's a treatment where your own immune cells are specially trained in a lab to find and attack your cancer cells.

How long will I be followed up after treatment?

You'll have regular checks for one year, then annual checks for up to 15 years.

Can I stop being in the study if I want to?

Yes, you can choose to leave the study at any time, and it won't affect your regular medical care.

How to find out more

MIGHTY Trial Coordinator

ctc.mighty@ucl.ac.uk +4420 7679 9852 Official trial record

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