RecruitingPHASE2, PHASE3INTERVENTIONAL

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

This research, called the AZTOUND study, is exploring new treatment options for specific blood cancers, including myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML). The main focus is on a new oral medication called ASTX030, which combines two drugs: azacitidine and cedazuridine. Researchers are comparing ASTX030 to the standard azacitidine injection to see if the oral form is as effective and easier for patients. They are also investigating ASTX030 in combination with another drug, venetoclax, for AML patients who haven't had treatment before. The study aims to find safe and effective ways to help people living with these conditions, potentially offering an oral treatment option instead of injections.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Taiho Oncology, Inc.

Enrolment target

316

Start

21 May 2020

Estimated completion

01 Nov 2028

Myelodysplastic Syndromes Acute Myeloid Leukemia Myelodysplastic Syndrome/Neoplasm Chronic Myelomonocytic Leukemia

What is this study about?

This study, called AZTOUND, is looking into new treatments for certain blood conditions like Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukaemia (AML). Think of these as conditions where the blood-forming cells in your bone marrow don't work quite right. Researchers are testing a new oral drug called ASTX030, which is actually two drugs (azacitidine and cedazuridine) combined into one pill. Currently, many people with these conditions receive azacitidine as an injection under the skin.

The main goal of this study is to see if taking ASTX030 as a pill is as safe and effective as receiving the standard azacitidine injection. This could be a big step forward, as taking a pill at home might be more convenient and comfortable for patients than regular injections. For some patients with newly diagnosed AML, the study is also looking at whether ASTX030, when given together with another drug called venetoclax, works well as a first treatment. The study has different stages, and some parts are still looking for new participants to join.

Key takeaways

This study is testing new treatments for certain blood cancers.
It aims to find out if an oral drug (ASTX030) works as well as injections.
Some parts are looking at ASTX030 with another drug (venetoclax) for AML.
It's for adults with specific diagnoses like MDS or AML.
Participation involves taking study medication and regular clinic visits.
You can stop participating at any time if you wish.

Who may be eligible?

Generally, this study is for adults over 18 who have been diagnosed with certain blood conditions like Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukaemia (CMML), or Acute Myeloid Leukaemia (AML). For the MDS and CMML parts of the study, you would need to be suitable for treatment with a drug like azacitidine according to your doctor. You'd also need to be well enough to take part, meaning you can look after yourself and have healthy enough organs.

For the AML part of the study, it's for people newly diagnosed with AML who haven't had treatment for it yet. This part is specifically for those who might not be able to have very strong chemotherapy, perhaps because they are over 75 years old. In all areas of the study, you would need to be generally stable and expected to live for at least 12 weeks. You should also be able to swallow pills and follow fasting instructions if needed.

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 or older?
Do you have a diagnosis of MDS, CMML, or newly diagnosed AML?
Are you generally well enough to take part and can look after yourself?
Can you swallow pills without difficulty?
If you have AML, are you unable to have very strong chemotherapy (e.g., over 75 years old)?
Have you had major surgery or strong chemotherapy recently? (This might affect eligibility).

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last about 8 years in total, though your personal involvement might be shorter. Some parts of the study compare the new oral drug to the standard injected drug. This means you might be randomly assigned to receive either the new oral medication (ASTX030) or the standard azacitidine injection. In other parts, you might receive ASTX030 with venetoclax. You'll have regular visits to the clinic for check-ups, blood tests, and to make sure the treatment is safe and working well. These visits will likely involve physical exams and discussions about how you're feeling. You'll receive the study medication as prescribed, and the team will monitor you closely throughout your time in the study.

Potential risks and benefits

Taking part in a research study means there's a chance you might benefit from the new treatment, or it might help scientists learn more for future patients. However, there's no guarantee the treatment will work for you, and it could have side effects, some of which might be serious. The study team will explain all known risks. Your safety is a priority, and you'll be closely monitored. Remember, joining is entirely your choice, and you can withdraw from the study at any time without affecting your usual medical care.

Locations (71)

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

Keck School of Medicine of USC
Verified postcode
Los Angeles, United States· Recruiting
UC Irvine Health - Chao Family Comprehensive Cancer Center
Verified postcode
Orange, United States· Recruiting
Yale University
Verified postcode
New Haven, United States· Recruiting
University of Miami - Sylvester Comprehensive Cancer Center
Verified postcode
Miami, United States· Recruiting
University of Emory - Winship Cancer Institute
Verified postcode
Atlanta, United States· Recruiting
Dana-Farber Cancer Institute
Verified postcode
Boston, United States· Recruiting
John Theurer Cancer Center / Hackensack University
Verified postcode
Hackensack, United States· Recruiting
Roswell Park Comprehensive Cancer Center
Verified postcode
Buffalo, United States· Recruiting
New York University Langone Hospital - Long Island
Verified postcode
Mineola, United States· Recruiting
Perlmutter Cancer Center - 34th Street
Verified postcode
New York, United States· Recruiting
Icahn School of Medicine at Mount Sinai
Verified postcode
New York, United States· Recruiting
Weill Cornell Medical Center
Verified postcode
New York, United States· Withdrawn

Common questions

What types of conditions is this study investigating?

It's looking at Myelodysplastic Syndromes (MDS), Acute Myeloid Leukaemia (AML), and Chronic Myelomonocytic Leukaemia (CMML).

What are the main treatments being tested?

Researchers are testing a new oral drug called ASTX030, sometimes alone and sometimes with another drug called venetoclax.

Why is an oral drug being studied?

An oral drug might be more convenient and comfortable for patients to take at home compared to regular injections.

How long does the study last for participants?

Although the whole study is very long, a participant's involvement would be for a shorter period, with regular follow-ups to check on their health.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, and this will not affect your regular medical care.

How to find out more

Taiho Oncology, Inc.

medicalinformation@taihooncology.com +1 844-878-2446 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.