Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humansInterventional

Safety, pharmacokinetics, and preliminary efficacy of BYON4413 in acute myeloid leukemia and myelodysplastic neoplasms.

This study is looking into a new experimental medicine, BYON4413, for adults in the UK who have acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS) that has come back or hasn't improved with other treatments. This is a very early study, often called a 'Phase 1' study, meaning it's one of the first times this medicine is being given to people. The main goals are to check if the medicine is safe, to understand how the body handles it (this is called 'pharmacokinetics'), and to see if it shows any early signs of working against these conditions. The study includes both men and women aged 18 and over.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Byondis B.V.

Enrolment target

Start

30 Apr 2024

Patients with relapsed/refractory acute myeloid leukemia or myelodysplastic neoplasms

What is this study about?

This study is for people in the UK who have specific types of blood cancers: acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS). These are conditions where there are problems with how blood cells are made in the bone marrow. The study is particularly for those whose cancer has either returned after treatment or hasn't responded well to the treatments they've already received.

The main purpose of this study is to test a new medicine called BYON4413. This medicine is still in its very early stages of development. When a new medicine is first given to people, doctors want to find out if it's safe to use and what doses can be given without causing too many problems. They also want to understand how the medicine moves through the body, how long it stays there, and how it's eventually removed.

Because this is an early-stage (Phase 1) study, it's not primarily designed to cure the conditions, but rather to gather important information that will help in developing the medicine further. If the medicine shows promising results in this initial study, it may then be tested in larger studies to see if it can be an effective treatment option in the future.

Key takeaways

This study is for adults with certain blood cancers (AML or MDS) that haven't responded to other treatments.
It's testing a new medicine called BYON4413 for safety and how it works in the body.
This is an early-stage study, so new risks are possible, but you'll be closely monitored.
Participation is voluntary, and you can leave the study at any time.
The study aims to gather important information for future treatment development.

Who may be eligible?

This study is looking for volunteers who are 18 years old or older. There is no upper age limit, meaning even older adults are welcome to take part.

Both men and women can join this study. You would be considered if you have a diagnosis of acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS).

Crucially, your AML or MDS must have either returned after you've had previous treatment, or it must not have responded well to the treatments you've tried so far. This study is not for people whose condition is currently well-controlled or responding to standard treatments.

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 (AML) or myelodysplastic syndromes (MDS)?
Has your AML or MDS returned after treatment or not responded well to previous treatments?
Are you able to attend regular hospital or clinic visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given the new medicine, BYON4413. This is an early study, so the doctors will carefully monitor you. You will have regular visits to the hospital or clinic. These visits will involve blood tests to check how your body is handling the medicine and to see how the medicine levels change over time. You may also have other tests, like physical exams and possibly bone marrow biopsies, to see how your condition is responding.

The specific number of visits and the total duration of your participation will depend on how you respond to the medicine and what the study doctors decide is best. There will be a follow-up period after you stop taking the medicine to ensure your safety and monitor any lasting effects. All procedures, schedules, and expectations will be clearly explained to you before you agree to join.

Potential risks and benefits

Taking part in this early-stage study means you might be among the first to receive a new medicine, which could potentially offer some benefit if your current treatments haven't been successful. However, as it's a new medicine, there are also potential risks and side effects that are not yet fully known. The study team will carefully monitor you for any problems. You will be told about all known possible side effects before you decide to take part, and new risks that become known during the study will be shared with you immediately. Remember, deciding to participate is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
Spain
—
Unverified
Netherlands
—
Unverified
Belgium

Common questions

What is BYON4413?

BYON4413 is a new, experimental medicine being tested for specific blood cancers.

What does 'relapsed/refractory' mean?

It means your condition has either returned after treatment or hasn't improved with previous treatments.

Is this a cure?

This is an early study primarily focused on understanding the medicine's safety, not immediately curing the condition.

Who can join the study?

Adults aged 18 and over, of any gender, with relapsed or refractory AML or MDS.

What is a 'Phase 1' study?

It's one of the first times a new medicine is given to people, mainly to check safety and how the body handles it.