Active not recruitingPHASE1INTERVENTIONAL

Dose Optimization and Expansion Study of DFV890 in Adult Patients With Myeloid Diseases

This research is testing a new medicine called DFV890 for adults with certain blood conditions, specifically lower-risk myelodysplastic syndromes (MDS), lower-risk chronic myelomonocytic leukemia (CMML), and high-risk clonal cytopenia of undetermined significance (CCUS). The main goals are to find a safe and effective dose of DFV890, understand any side effects, and see how well it works. This is a Phase 1 study, meaning it's an early step in testing the medicine. We want to check if the medicine can help improve these conditions and how the body handles it. Participants will take the medicine for at least 24 weeks, and doctors will carefully monitor their health throughout.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

08 May 2023

Estimated completion

13 Jan 2027

Myeloid Diseases

What is this study about?

This study is about a new medicine called DFV890. It's designed for adults who have certain blood disorders known as myeloid diseases. Specifically, we're looking at patients with lower-risk myelodysplastic syndromes (MDS), lower-risk chronic myelomonocytic leukemia (CMML), and a condition called high-risk clonal cytopenia of undetermined significance (HR CCUS). These conditions affect how your body makes healthy blood cells.

The main purpose of this research is to see if DFV890 is safe to use and if it can help improve these conditions. We also want to find the best dose of the medicine that works well without too many side effects. At the moment, we don't fully know how safe or effective DFV890 is for these specific diseases, which is why we’re doing this study. This is an important step in developing new treatments that could potentially help patients like you.

We will be carefully checking how your body reacts to DFV890, looking at any changes in your disease, and understanding how the medicine moves through your body. By taking part in this study, you could help us learn more about these myeloid diseases and contribute to finding better ways to treat them in the future. We want to ensure that any new treatments are as safe and effective as possible for patients.

Key takeaways

This study evaluates a new drug, DFV890, for specific myeloid blood disorders.
It aims to find a safe and effective dose for lower-risk MDS, CMML, and high-risk CCUS.
Participation involves taking the study medicine for at least 24 weeks and regular health check-ups.
Bone marrow samples will be needed at the start and at specific times during the study.
You have the right to withdraw from the study at any time and for any reason.

Who may be eligible?

You might be able to join this study if you are 18 years old or older. You also need to be well enough to carry out most of your daily activities without too much difficulty, as determined by a doctor's assessment.

For those in the early stages of the study, we are looking for people with lower-risk myelodysplastic syndromes (MDS) or lower-risk chronic myelomonocytic leukemia (CMML) whose current treatments either haven't worked or they couldn't tolerate them. For the later parts of the study, we're also including some patients with a history of MDS with specific characteristics, or CMML patients with symptoms where standard treatments aren't suitable, or those with high-risk CCUS who haven't had prior treatment for a myeloid condition.

There are also some reasons why you might not be able to join. For example, if you've recently had certain cancer treatments within the last month, or if you have other serious health problems that could make the study unsafe for you. The study doctors will carefully review your health information to see if this study is a good fit 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 of age or older?
Do you have a diagnosis of lower-risk MDS, lower-risk CMML, or high-risk CCUS, where previous treatments haven't worked or were not tolerated (for MDS/CMML)?
Are you generally able to perform most daily activities without significant difficulty?
Are you willing and able to undergo bone marrow tests as needed for the study?
Have you not received other specific cancer treatments within the last month?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be taking the study medicine, DFV890, for at least 24 weeks, which is about six months. You will have regular check-ups with the study team to see how you're doing and if the medicine is helping. These visits will involve blood tests and physical examinations.

To help us understand your condition and how the medicine is working, you will need to provide bone marrow samples. This will happen when you first start the study and at specific times throughout. You can stop taking part in the study at any time, for any reason, without it affecting your usual medical care. The study team may also decide to stop your participation if your disease gets worse, you experience side effects, or for other medical reasons.

Potential risks and benefits

Participating in this study might offer you some potential benefits, such as receiving a new investigational treatment for your condition that is not yet widely available. You'll also be closely monitored by a team of healthcare professionals throughout the study. However, because DFV890 is a new medicine, there are potential risks. The medicine may cause side effects, some of which might be serious or currently unknown. The study doctors and nurses will explain these potential risks in detail. You are free to withdraw from the study at any time, for any reason, and this decision will not affect your future medical care.

Locations (26)

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

Stanford Cancer Center
Verified postcode
Stanford, United States
H Lee Moffitt Cancer Center and Research Institute
Verified postcode
Tampa, United States
Northwestern University
Verified postcode
Chicago, United States
Sidney Kimmel CCC At JH
Verified postcode
Baltimore, United States
Dana Farber Cancer Institute
Verified postcode
Boston, United States
Mayo Clinic Rochester
Verified postcode
Rochester, United States
Weill Cornell Medicine NY-Presb
Verified postcode
New York, United States
Memorial Sloan Kettering Cancer Ctr
Verified postcode
New York, United States
Vanderbilt University Medical Ctr
Verified postcode
Nashville, United States
Univ of TX MD Anderson Cancer Cntr
Verified postcode
Houston, United States
Huntsman Cancer Institute
Verified postcode
Salt Lake City, United States
Novartis Investigative Site
Verified postcode
Grenoble, France

Common questions

What is a myeloid disease?

Myeloid diseases are conditions that affect how your body makes healthy blood cells in the bone marrow. This study focuses on certain types, including specific forms of MDS, CMML, and CCUS.

What is DFV890?

DFV890 is the new medicine being tested in this study. It's designed to help with the bone marrow issues seen in certain myeloid diseases, but its full effects and safety are still being investigated.

How long will I be in the study?

You'll be taking the study medicine for at least 24 weeks (about six months), unless you need to stop sooner due to side effects, your condition changing, or if you choose to withdraw.

Will I have to have bone marrow tests?

Yes, you will need to provide bone marrow samples when you first join the study and at certain times during your participation. This helps us understand how the medicine is affecting your condition.

Can I stop the study at any time?

Yes, you have the right to leave the study at any point and for any reason. Your decision will not affect your medical care.