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

A PHASE 1/2 STUDY OF ALX148 IN COMBINATION WITH AZACITIDINE IN PATIENTS WITH HIGHER RISK MYELODYSPLASTIC SYNDROME (MDS) (ASPEN-02)

This research study, called ASPEN-02, is designed for adults who have a specific blood condition known as higher-risk myelodysplastic syndrome (MDS) and who haven't started treatment for it yet. The main goal is to see if combining a new experimental drug, ALX148, with a standard medicine, azacitidine, is safe and effective. Researchers will closely watch for any side effects and how well patients respond to the treatment, such as if their condition improves or goes into remission. The study is divided into two main parts: an early phase to find the right dose of ALX148, followed by a larger phase to carefully check its effects when combined with azacitidine.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Alx Oncology Holdings Inc.

Enrolment target

Start

18 Jun 2024

Patients with higher risk myelodysplastic syndrome (MDS) who have not yet been treated for their disease

What is this study about?

This study is named ASPEN-02 and it's looking for people with a specific type of blood problem called higher-risk myelodysplastic syndrome, or MDS. MDS is a condition where your bone marrow, which is the spongy material inside your bones, doesn't make enough healthy blood cells. Higher-risk MDS means the condition is more serious and can sometimes turn into leukaemia.

Currently, if you have higher-risk MDS and haven't been treated yet, this study might be for you. It's testing a new medicine called ALX148 alongside a medicine that's already used for MDS, called azacitidine. The researchers want to find out if using these two drugs together is safe and if it helps people with MDS more than azacitidine alone. They will be carefully checking for any side effects and seeing how well people's bodies respond to the treatment.

The study has two main parts. The first part is to safely find the best amount of the new drug (ALX148) to give. Once that's decided, the second part will involve more people, and the doctors will focus on how many people improve or go into remission, meaning their doctors no longer see signs of the disease. They will also look at other important things like how long people feel well and how their everyday life is affected. This is a common way to test new medicines before they can be made widely available.

Key takeaways

This study is for adults with higher-risk MDS who have not started treatment yet.
It tests a new drug (ALX148) with a standard medicine (azacitidine).
The main goals are to check for safety and how well the treatment works.
Participation involves regular clinic visits and close monitoring.
You can stop participating at any time.

Who may be eligible?

This study is looking for adults who are 18 years old or older. Both men and women are welcome to take part.

You must have been diagnosed with a type of blood condition called higher-risk myelodysplastic syndrome (MDS). A crucial part of joining this study is that you must not have received any treatment for your MDS before. This means if you've already started medication or other therapies for your MDS, this study wouldn't be suitable for you.

Your doctor will check other specific health details to make sure this study is the right fit for you and that taking part would be safe.

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 higher-risk myelodysplastic syndrome (MDS)?
Have you NOT received any prior treatment for your MDS?
Are you able to attend regular clinic appointments for treatment and check-ups?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given a combination of two medicines: ALX148 and azacitidine. You'll have regular visits with doctors and nurses who will closely monitor your health. These visits will include checks for any side effects, blood tests, and other assessments to see how you're responding to the treatment.

The study will include several cycles of treatment. For example, during the main part of the study, you'll receive treatment for at least six cycles, or until your doctors can properly see how you're responding. Doctors will also collect information about how you're feeling and your quality of life. The exact total length of your participation will depend on how you respond to the treatment and the study's design, but you should expect it to involve ongoing monitoring.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit by giving you access to a new treatment combination that could be more effective for your MDS than current options. However, there are also potential risks, as with any new medicine, including unknown side effects or that the treatment might not work for you. The study team will carefully monitor you for any problems. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is myelodysplastic syndrome (MDS)?

MDS is a condition where your body's bone marrow doesn't produce enough healthy, mature blood cells. This can lead to problems like anaemia, infections, or easy bruising.

What does 'higher risk' MDS mean?

Higher-risk MDS means the condition is more severe and has a greater chance of getting worse or developing into a type of leukaemia. This is why doctors look for effective treatments.

What is azacitidine?

Azacitidine is a medicine already approved and used to treat MDS. It works by helping the bone marrow produce healthier blood cells.

What is a 'Phase 1/2' study?

A Phase 1 study primarily checks if a new drug is safe and helps find the right dose. A Phase 2 study then looks at how well the drug works for the condition, and continues to monitor safety, in a larger group of people.

Will I receive the new drug or a placebo?

In this study, everyone will receive the new drug ALX148 in combination with azacitidine. The study is not comparing it to a placebo (a dummy treatment).