An Adaptive Open-label Multicentre Phase 1/2 Trial, to Determine the Recommended Phase 2 Dose of CCTx-001, and to Assess Safety, Tolerability, and Clinical Activity in Patients With Relapsed/Refractory Acute Myeloid Leukaemia
This research study is looking at a new treatment called CCTx-001 for adults who have Acute Myeloid Leukaemia (AML) that has either returned after treatment or not improved with previous treatments. This trial is divided into two main parts. The first part is to find the best dose of CCTx-001 that is safe and can be given comfortably. The second part will then look more closely at how well this dose of CCTx-001 works against AML cells. Scientists hope this new drug could target specific cancer cells and offer a new way to treat this type of leukaemia when standard options haven't worked.
At a glance
What is this study about?
This study is testing a new medicine called CCTx-001. It's for adults who have Acute Myeloid Leukaemia (AML) which is a type of blood cancer. This particular group of patients has AML that has either come back after previous treatments (relapsed) or hasn't responded well to the treatments they've already had (refractory). This means that standard treatments haven't been successful for them, and researchers are looking for new options.
The main goal of this study is to see if CCTx-001 is safe to use, how well people tolerate it, and if it can help fight the AML cells. Scientists believe CCTx-001 works by targeting something specific on leukaemia cells called IL-1RAP. In laboratory tests, this approach has shown promising results in attacking AML cells while hopefully having less effect on healthy cells. If this treatment works well, it could potentially change how relapsed or refractory AML is treated in the future, offering a new treatment choice.
This study will start by carefully finding the right dose of CCTx-001 – one that is effective but also safe and easy for patients to manage. Once this dose is established, more patients will be given this dose to understand its effects better. The researchers will be closely monitoring participants throughout the study to understand how their bodies react to the drug and if it makes a difference to their leukaemia.
Key takeaways
- This study is testing a new drug (CCTx-001) for AML that has returned or not responded to treatment.
- It aims to find a safe and effective dose, and see if it helps against leukaemia.
- CCTx-001 works by specifically targeting certain leukaemia cells.
- Participation involves regular medical checks and receiving the study drug.
- There are potential benefits and risks, like all medicines, which will be discussed fully.
- You can stop participating at any time if you wish.
Who may be eligible?
To join this study, you would need to be an adult, aged 18 or over, and have a type of blood cancer called Acute Myeloid Leukaemia (AML) that has either come back after past treatments or didn't improve with those treatments. Your doctor would need to confirm that you've tried other suitable treatments, or that those treatments aren't right for you.
There are also some health checks to make sure you're well enough to take part. For example, your organs like your kidneys would need to be working properly, and your general health should be good enough to participate in the study procedures, including a procedure called leukapheresis. If you've had a stem cell transplant in the past, there are specific timeframes and health conditions you'd need to meet, such as being at least 3 months post-transplant and not actively experiencing graft versus host disease.
Finally, you'd need to be able to come for all the study appointments and agree to follow the study plan. You would also need to be able to read and understand the study information and sign a consent form, showing you agree to take part.
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 18 years old or older?
- Do you have Acute Myeloid Leukaemia that has relapsed or not responded to previous treatments?
- Do you have good general health and organ function (e.g., kidneys)?
- Are you able to attend all study appointments and follow the study rules?
- Have you understood and are willing to sign the informed consent form?
What does participation involve?
If you decide to take part in this study, you would first have a full medical check-up to make sure it's suitable for you. This would include blood tests, health assessments, and possibly other scans.
You would receive the new study drug, CCTx-001. This might involve regular visits to the clinic for treatments, as well as ongoing check-ups, blood tests, and possibly bone marrow biopsies to see how the treatment is affecting your leukaemia. The doctors and nurses will closely monitor your health and any side effects you might experience.
The study will continue for a certain period, and you will have follow-up appointments even after your main treatment phase finishes, to keep track of your health long-term. The total length of your participation will depend on how you respond to the treatment and the study's overall plan, but your treatment-related visits could be frequent at the start.
Potential risks and benefits
Locations (6)
- Besançon Regional and University HospitalVerified postcodeBesançon, France
- Hospital Saint LouisVerified postcodeParis, France
- Ludwig-Maximilians University of MunichVerified postcodeMunich, Germany
- University Hospital UlmVerified postcodeUlm, Germany
- Vall d'Hebron University HospitalVerified postcodeBarcelona, Spain
- Karolinska University HospitalVerified postcodeStockholm, Sweden
Common questions
What is CCTx-001?
CCTx-001 is a new experimental drug being tested for Acute Myeloid Leukaemia (AML) that targets specific features on leukaemia cells.
What does 'relapsed/refractory' AML mean?
It means your Acute Myeloid Leukaemia has either come back after previous treatment, or it hasn't responded well to the treatments you've had.
Will I receive the new drug, or a placebo?
This study does not involve a placebo. All participants who join the study will receive the active study drug, CCTx-001.
How long will I be in the study?
The length of your participation will depend on how you respond to the treatment and the study's overall plan. You will have regular checks during treatment and follow-up after.
Can I leave the study at any time?
Yes, you are free to withdraw from the study at any point, for any reason, without it affecting your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.