Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

A Phase 1/2A, Open Label Dose Escalation and Cohort Expansion Study of Orally Administered CA-4948 (IRAK4i) as a Monotherapy in Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

This research study is looking into a new medicine called CA-4948. It's for adults aged 18 and over who have acute myeloid leukaemia (AML) or a more serious type of myelodysplastic syndrome (MDS). This study has two main parts. The first part focuses on understanding if the medicine is safe and finding the right dose. Researchers will closely monitor for any side effects. The second part will then look at how well the medicine works in helping people with their AML or MDS. The goal is to see if CA-4948 can improve their condition. It's an important step in finding new ways to treat these blood cancers.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Curis Inc.

Enrolment target

Start

04 Sep 2024

Acute Myelogenous Leukemia (AML) or higher-risk Myelodysplastic Syndrome (MDS).

What is this study about?

This study is testing a new medicine called CA-4948 for two types of blood cancer: acute myeloid leukaemia (AML) and a more serious form of myelodysplastic syndrome (MDS). These conditions affect your blood-forming cells in the bone marrow.

The study is divided into two main parts. In the first part (called Phase 1), the main goal is to find out if the new medicine, CA-4948, is safe. The researchers will start with a very small dose and gradually increase it in different groups of people. They will carefully watch for any side effects and monitor your body's response with tests like ECGs (which check your heart) and blood tests. This helps them find the best dose that is both safe and has a good chance of working.

Once a safe dose is found, the study moves into the second part (called Phase 2a). Here, the main goal is to see how well CA-4948 works in treating AML or MDS. The researchers will be looking to see if the medicine helps improve your condition, for example, by reducing the number of cancer cells or helping your blood counts recover. They will compare the results against what they expect to see without this medicine. This is a very important step in developing new treatments that could help people with these conditions.

Key takeaways

This study is for adults with certain blood cancers (AML or hrMDS).
It's testing a new medicine called CA-4948.
The study first checks for safety and then how well the medicine works.
Participation involves regular clinic visits for checks and tests.
You will know if you are receiving the study medicine.
You can stop participating at any time.

Who may be eligible?

This study is for adults aged 18 years and older. Both men and women can take part.

You might be able to join this study if you have been diagnosed with acute myeloid leukaemia (AML) or a more serious type of myelodysplastic syndrome (MDS).

There will be other specific health requirements that you must meet to be considered for this study. Your doctor will carefully check these details to see if this study is suitable 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.

Are you 18 years old or older?
Have you been diagnosed with Acute Myeloid Leukaemia (AML) or a higher-risk Myelodysplastic Syndrome (MDS)?
Are you willing to take study medicine by mouth?
Are you able to attend regular clinic visits and tests?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be given the study medicine, CA-4948, by mouth. This is an 'open label' study, which means both you and your study doctor will know you are receiving CA-4948.

Throughout the study, you'll have regular visits to the clinic. At these visits, doctors and nurses will closely monitor your health. This will involve physical examinations, blood tests (to check your blood counts and how your organs are working), urine tests, and heart tests like ECGs. They will also ask you about any side effects you might be having.

The study involves several assessments to see how your body is reacting to the medicine and if your condition is improving. The total duration of your participation will depend on how you respond to the treatment and what the study protocol outlines, but you will be closely followed by the study team for an extended period.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. You might benefit from receiving a new medicine that could help your condition, especially if standard treatments haven't worked well for you. However, there's also the chance the medicine may not help, or you might experience side effects. The study team will explain all known side effects in detail. You have the right 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.

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Unverified
Germany
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Unverified
Spain
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Unverified
France

Common questions

What is CA-4948?

CA-4948 is the new medicine being tested in this study for people with AML or MDS.

What is AML or MDS?

AML (Acute Myeloid Leukaemia) and MDS (Myelodysplastic Syndrome) are types of blood cancers that affect how your body makes blood cells.

What does 'open label' mean?

It means both you and the study doctors will know that you are receiving the study medicine, CA-4948.

How long will I be in the study?

The exact duration can vary, but you will have ongoing follow-up visits and monitoring as long as you are benefiting from the medicine or until the study requires.

What are 'Phase 1' and 'Phase 2a'?

Phase 1 is the first stage where they check if the medicine is safe and find the right dose. Phase 2a then looks at how well the medicine works.