Active not recruitingPHASE1INTERVENTIONAL

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

This research study is open to adults in the UK diagnosed with a higher-risk form of Myelodysplastic Syndromes (MDS) who have not yet received treatment for their condition. MDS is a type of blood cancer. The study is testing a new combination of two medicines, venetoclax and azacitidine, which are given together. This is a Phase 1 study, meaning it's an early stage of research. The main aim is to find the safest and most effective dose of venetoclax when used with azacitidine. Researchers will also be checking how well this combination works and if there are any side effects. The study includes different stages to determine the best dose and then to further check safety.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

AbbVie

Enrolment target

129

Start

12 Jan 2017

Estimated completion

01 Jan 2027

Myelodysplastic Syndromes (MDS)

What is this study about?

This study is investigating a new treatment approach for a type of blood cancer called Myelodysplastic Syndromes (MDS). MDS affects your bone marrow, which is the spongy material inside your bones where blood cells are made. In MDS, the bone marrow doesn't produce enough healthy blood cells, leading to problems like anaemia, infections, and bruising. This particular study is for people who have a 'higher-risk' form of MDS, meaning their condition is more likely to get worse quickly, and who have not yet had any treatment for their MDS.

The research is looking at two medicines used together: venetoclax and azacitidine. Azacitidine is a standard treatment for MDS, but venetoclax is a newer drug that works in a different way, targeting certain proteins in cancer cells. By combining them, doctors hope to get a more effective treatment. Because this is an early-stage study (called Phase 1), the main goal is to find the right dose of venetoclax that can be safely given with azacitidine, and to see if this combination causes any side effects. It will also give us an early idea of how well this combination might work.

Understanding and finding new treatments for MDS is very important. Studies like this help us move forward in improving care for people with conditions like MDS. While this is an early study, it's a crucial step in potentially developing new and better options for patients.

Key takeaways

This study is testing a new combination of drugs (venetoclax and azacitidine) for higher-risk MDS.
It's for people who haven't had previous treatment for their MDS.
The main aim is to find the safest and best dose of the drug combination.
It's an early-stage study (Phase 1) contributing to future MDS treatments.
Participation involves regular hospital visits for treatment and monitoring.
You can withdraw from the study at any time without affecting your care.

Who may be eligible?

This study is looking for adults aged 18 or over in the UK who have recently been diagnosed with a higher-risk type of Myelodysplastic Syndromes (MDS) and have not yet started treatment for it. Your doctor will have classified your MDS as either 'Intermediate-2' or 'High' risk under an older system (IPSS), or 'intermediate', 'high', or 'very high' risk under a newer system (IPSS-R). You also need to have less than 20% abnormal cells (blasts) in your bone marrow.

To be considered for the study, you also need to be generally well enough to take part. This means your daily activities shouldn't be too restricted by your illness, as assessed by a common scoring system called ECOG, where a score of 2 or less is needed.

You would not be able to join if you've already had treatment for your MDS (though supportive care like blood transfusions is fine). Also, if you've had a bone marrow transplant, or if your MDS is linked to previous cancer treatment, or if you have certain other blood disorders like chronic myelomonocytic leukemia (CMML), you wouldn't be eligible. You also can't have received a specific type of drug called a BCL-2 Homology 3 (BH3) mimetic before.

Quick self-check

Are you 18 years old or over?
Do you have a new diagnosis of Myelodysplastic Syndromes (MDS)?
Has your doctor classified your MDS as 'higher-risk'?
Have you NOT received any specific treatment for your MDS yet?
Are you generally well enough for daily activities (ECOG score of 2 or less)?
Have you NOT had a bone marrow transplant or specific other blood disorders?

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

What does participation involve?

If you join this study, you will receive a combination of two medications: azacitidine and venetoclax. Azacitidine is usually given as an injection under the skin, and venetoclax is a tablet you take by mouth. The study will involve regular visits to the hospital for assessments, which will include blood tests, physical examinations, and possibly bone marrow biopsies, to check how you are responding to the treatment and to monitor for any side effects. The exact number and frequency of visits will be explained by the study team. You will be closely monitored throughout the treatment period. The total duration of your participation in the study, including follow-up after the treatment phase, will also be discussed with you.

Potential risks and benefits

Taking part in any clinical study carries both potential benefits and risks. You might benefit from receiving a new treatment that isn't widely available yet, which could be more effective for your MDS. However, there's no guarantee the treatment will work for you, and you might experience side effects from the medications. All potential risks and side effects will be fully explained by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (37)

Duplicate_University of Arizona Cancer Center - North Campus /ID# 154155
Tucson, United States
The University of Chicago Medical Center /ID# 153673
Chicago, United States
University of Maryland, Baltimore /ID# 153669
Baltimore, United States
Tufts Medical Center /ID# 153672
Boston, United States
Dana-Farber Cancer Institute /ID# 152735
Boston, United States
Washington University-School of Medicine /ID# 153671
St Louis, United States
Columbia University Medical Center /ID# 153661
New York, United States
Weill Cornell Medical College /ID# 155524
New York, United States
Oregon Medical Research Center /ID# 152734
Portland, United States
University of Pittsburgh MC /ID# 153662
Pittsburgh, United States
Tennessee Oncology-Nashville Centennial /ID# 222769
Nashville, United States
Vanderbilt University Medical Center /ID# 152738
Nashville, United States

+25 more sites — see the official record for the full list.

Common questions

What is Myelodysplastic Syndromes (MDS)?

MDS is a group of blood cancers where your bone marrow doesn't produce enough healthy blood cells. It can lead to problems like fatigue, infections, and bleeding.

What does 'Phase 1 study' mean?

Phase 1 is an early stage of clinical research. Its main goal is to find the safest dose of a new treatment or combination of treatments and to identify any early side effects.

What are venetoclax and azacitidine?

Azacitidine is a standard medication used for MDS. Venetoclax is a newer drug that works differently to target cancer cells. This study is testing them together.

Will I know which dose I'm getting?

Yes, this study is 'open-label,' meaning both you and the study team will know which dose of the medication you are receiving.

What does 'treatment-naïve' mean?

This means you have not had any previous treatments specifically for your MDS. Supportive care like blood transfusions is generally allowed.