Phase I/II Trial to Determine Safety and Efficacy of non-Viral Allogeneic Dual CD123/33-Chimeric Antigen Receptor CIK (CARCIK-CD123/33) Cells in Adult and Pediatric Patients with Relapse/Refractory Acute Myeloid Leukemia (AML) and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This clinical trial is testing a new immune therapy for adults with acute myeloid leukaemia (AML) or blastic plasmacytoid dendritic cell neoplasm (BPDCN). It is designed for patients whose cancer has come back (relapsed) or has not responded to standard treatments (refractory). The trial uses specialised immune cells collected from a donor. In a laboratory, scientists train these cells to spot two specific flags (called CD123 and CD33) found on the surface of the leukaemia cells. The aim is to help the body's immune defences track down and destroy the cancer. Because this is an early-stage study, the main goal is to find out if the treatment is safe, discover any side effects, and determine the best dose. Doctors will also monitor how long the cells survive in the body and whether they help clear the cancer.
At a glance
What is this study about?
Acute myeloid leukaemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) are rare and fast-growing cancers that affect blood cells. Although standard treatments like chemotherapy can be very effective, the cancer can sometimes return or stop responding to treatment. When this happens, doctors need new, targeted ways to tackle the disease.
This trial investigates an experimental treatment known as CARCIK-CD123/33. It is a type of cell therapy that uses donor immune cells called cytokine-induced killer (CIK) cells. Scientists modify these cells without using viruses so that they carry special search tools on their surface. These tools lock onto two markers, CD123 and CD33, which are commonly present on AML and BPDCN cancer cells, allowing the modified cells to attack the cancer directly.
Because this is one of the first times this therapy is being given to people, doctors are focusing closely on safety. They want to ensure the treatment is well-tolerated and to find the most effective dose. The study will also track whether the modified cells successfully reduce cancer in the body and help patients stay in remission for longer.
Key takeaways
- Tests a new donor-cell immune therapy for adults with hard-to-treat blood cancers.
- Aimed at acute myeloid leukaemia (AML) and BPDCN that has returned or resisted other treatments.
- Uses laboratory-modified cells designed to hunt down two specific cancer targets.
- Main focus is to check safety, monitor side effects, and find the most suitable dose.
- Taking part is completely voluntary and involves close hospital monitoring.
Who may be eligible?
This trial is open to adults aged 18 and over who have been diagnosed with either acute myeloid leukaemia (AML) or blastic plasmacytoid dendritic cell neoplasm (BPDCN). It is specifically intended for individuals whose cancer has returned after previous treatment or has not cleared with standard therapies.
Because this is an intensive treatment, participants must be well enough overall to cope with the therapy and potential side effects. The clinical team will carry out standard health checks, including blood tests and heart and organ checks, to make sure taking part is as safe as possible for you.
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.
- You are aged 18 or older.
- You have a confirmed diagnosis of AML or BPDCN.
- Your cancer has come back (relapsed) or did not respond to standard treatment (refractory).
- You are well enough to attend regular hospital appointments and have tests.
What does participation involve?
If you decide to take part, the trial will begin with health checks and blood tests to make sure the treatment is safe for you. You will then receive the modified donor cells through a drip into a vein (an infusion). You will likely need to stay in the hospital or visit a specialist clinic frequently during the first month, particularly during the first 28 days, so doctors can monitor you closely for any reactions. Over the following months and years, you will have regular clinic visits for blood tests, bone marrow checks, and scans to see how well the therapy is working and to monitor your recovery.
Potential risks and benefits
Locations (1)
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Common questions
What makes this therapy different from standard chemotherapy?
Chemotherapy uses chemicals to attack rapidly dividing cells throughout the body. This therapy uses living immune cells that have been specially trained to recognise and attack specific targets on the cancer cells.
Where do the treatment cells come from?
The cells are collected from a healthy donor and modified in a specialised laboratory before being given to you through a drip.
Will taking part guarantee that my cancer goes away?
No. Because this is an early-stage clinical trial, researchers do not yet know how effective it will be. The main goal is to test safety and find the right dose.
Do I have to pay to take part in this trial?
No. In the UK, clinical trial medications and study-related tests are provided free of charge, and some travel expenses may be covered.
What happens if I decide I no longer want to participate?
You are free to leave the trial at any point. Deciding to withdraw will never affect your relationship with your medical team or the quality of your standard NHS care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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