Active not recruitingPHASE3INTERVENTIONAL

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

This study investigates a new treatment approach for adults recently diagnosed with higher-risk Myelodysplastic Syndrome (MDS). MDS is a condition where your bone marrow struggles to make healthy blood cells. Some people with higher-risk MDS may see their condition worsen or develop a type of leukaemia. This trial is testing whether adding an experimental drug called venetoclax to a standard treatment, azacitidine, is safer and more effective than taking azacitidine with a dummy pill (placebo). About 500 participants globally will be involved, helping researchers understand if this new combination could improve outcomes for those with this serious blood disorder.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

AbbVie

Enrolment target

531

Start

10 Sep 2020

Estimated completion

01 Jul 2027

Myelodysplastic Syndrome (MDS)

What is this study about?

Myelodysplastic Syndrome (MDS) is a group of conditions that affect your bone marrow, the soft, spongy tissue inside your bones where blood cells are made. In MDS, the bone marrow doesn't produce enough healthy blood cells. This can lead to symptoms like feeling very tired, short of breath, looking pale (due to low red blood cells), or bruising easily. For some people, especially those with a 'higher-risk' type, MDS can get worse over time or even develop into a type of blood cancer called acute myeloid leukaemia (AML). This is why finding effective treatments is so important.

This study is looking at a new treatment combination for adults who have recently been diagnosed with higher-risk MDS. The main goal is to find out if combining a new drug called venetoclax with an existing treatment called azacitidine works better and is safer than azacitidine given with a placebo (a pill that looks like the real drug but contains no medicine). Venetoclax is still being investigated, which means it's not yet approved for widespread use for MDS. By comparing these two approaches, researchers hope to improve how doctors care for people with this condition.

Understanding if venetoclax and azacitidine together are more effective is crucial because it could offer new hope for managing higher-risk MDS. The study will involve around 500 participants from many different hospitals and clinics worldwide. Their health will be closely monitored to see how the treatments affect their MDS and if there are any side effects. The information gathered will help doctors learn more about the best ways to treat adults with newly diagnosed higher-risk MDS.

Key takeaways

Targets adults with newly diagnosed higher-risk Myelodysplastic Syndrome (MDS).
Compares an experimental drug (venetoclax) with standard treatment (azacitidine) to azacitidine and a dummy pill (placebo).
Aims to understand if combining venetoclax and azacitidine is safer and more effective.
Participation involves regular hospital visits, medical checks, blood, and bone marrow tests.
This is a Phase 3 study, meaning it's a larger trial following earlier research to confirm results.

Who may be eligible?

To join this study, participants must be adults with a recent diagnosis of Myelodysplastic Syndrome (MDS) and be considered to have 'higher-risk' MDS. This 'higher-risk' classification is based on specific assessments of your condition.

There are also some important reasons why someone might not be able to join. For example, if you have already received certain past treatments for your MDS, such as specific chemotherapy drugs or a stem cell transplant, you would not be eligible. This also applies if your MDS developed from another blood condition or if it's a specific type of MDS combination disorder.

Quick self-check

Are you an adult aged 18 or over?
Have you been recently diagnosed with Myelodysplastic Syndrome (MDS)?
Has your doctor told you your MDS is considered 'higher-risk'?
Have you not had previous treatments for MDS like certain types of chemotherapy or a stem cell transplant?
Is your MDS not linked to previous cancer treatments or other specific blood disorders?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would receive treatment with either venetoclax tablets (an experimental drug) and azacitidine (given into a vein or under the skin), or a dummy tablet (placebo) and azacitidine. You would have regular visits to the hospital or clinic throughout the study. These visits will involve various medical checks, including blood tests and bone marrow tests, to see how the treatment is working. Doctors will also carefully monitor you for any side effects and you might be asked to complete questionnaires about how you are feeling. This trial might involve more hospital visits and tests than your usual care, but all of these steps are important for understanding the safety and effectiveness of the new treatment.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. The potential benefit of this study is that the combination of venetoclax and azacitidine might be more effective in controlling your MDS than azacitidine alone. However, there's no guarantee of this, and the experimental drug venetoclax may have side effects that are not yet fully known. Like all medications, azacitidine also has known side effects, which your doctor will discuss with you. All procedures, including blood draws and bone marrow tests, carry their own small risks. Your safety is a top priority, and you will be closely monitored throughout the study. You always have the right to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (220)

Duplicate_Providence Medical Foundation /ID# 222633
Fullerton, United States
University of California, Los Angeles /ID# 221760
Los Angeles, United States
Torrance Memorial Physician Network Cancer Care /ID# 222702
Torrance, United States
PIH Health Whittier Hospital /ID# 222647
Whittier, United States
Rocky Mountain Cancer Centers - Boulder /ID# 223723
Boulder, United States
Yale University School of Medicine /ID# 222764
New Haven, United States
Helen F. Graham Cancer Center & Research Institute /ID# 223731
Newark, United States
Florida Cancer Specialists - Fort Myers /ID# 221319
Fort Myers, United States
Memorial Healthcare System /ID# 222703
Hollywood, United States
Florida Cancer Specialists - North /ID# 221318
St. Petersburg, United States
Florida Cancer Specialists - Panhandle /ID# 221315
Tallahassee, United States
Florida Cancer Specialists - East /ID# 221317
West Palm Beach, United States

+208 more sites — see the official record for the full list.

Common questions

What is Myelodysplastic Syndrome (MDS)?

MDS is a disorder where your bone marrow doesn't make enough healthy blood cells, leading to problems like tiredness and easy bruising.

What is 'higher-risk' MDS?

This means your MDS condition has a greater chance of getting worse over time or developing into a type of blood cancer called acute myeloid leukaemia (AML).

What is venetoclax?

Venetoclax is a new drug being tested for MDS. It is not yet approved for general use but is being studied in this trial.

What is a placebo?

A placebo is a 'dummy' pill that looks just like the study drug but contains no medicine. It's used to fairly compare the effects of the active treatment.

Will I know which treatment I'm getting?

No, this is a 'blinded' study, meaning neither you nor your doctors will know if you are receiving venetoclax or the placebo alongside azacitidine. This helps ensure fair results.