Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies

This study is investigating a new drug called bexmarilimab. Researchers want to see if combining it with standard treatments is safe and effective for people with specific blood cancers, including Acute Myeloid Leukaemia (AML), Chronic Myelomonocytic Leukaemia (CMML), and Myelodysplastic Syndromes (MDS). The study has two main parts: first, finding the safest and most suitable dose of bexmarilimab, and then, in the second part, checking how well this combination treatment works to battle the cancer in patients. The goal is to find new and better ways to treat these conditions.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Faron Pharmaceuticals Ltd

Enrolment target

181

Start

02 Jun 2022

Estimated completion

01 Mar 2027

Acute Myeloid Leukemia Chronic Myelomonocytic Leukemia Myelodysplastic Syndromes Relapsed/Refractory AML

What is this study about?

This research study is all about a new treatment called bexmarilimab. Doctors are carefully testing it to see if it can be a helpful addition to the treatments already available for certain types of blood cancer. These cancers include Acute Myeloid Leukaemia (AML), Chronic Myelomonocytic Leukaemia (CMML), and Myelodysplastic Syndromes (MDS).

The study is happening in two main stages. The first stage is focused on safety. Researchers will give different small doses of bexmarilimab, combined with standard treatments, to a small group of patients. Their main aim here is to find the dose that is safest and best tolerated, while also looking to see if there are any signs that it's starting to work. Once that ideal dose is found, the second stage begins.

In the second stage, more patients will receive the identified safe dose. The main goal then shifts to seeing how effective this combination treatment is in helping patients with these specific blood cancers. They'll be looking to see if the treatment can help slow down, stop, or even improve the cancer. This type of study is crucial for learning about new medicines and hopefully bringing new options to people who need them.

Key takeaways

This study is for adults with specific types of blood cancer: AML, CMML, or MDS.
It's testing a new drug, bexmarilimab, combined with standard treatments.
The first part finds a safe dose; the second investigates how well it works.
You'll get close medical monitoring throughout the study.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for adults aged 18 or over who have been diagnosed with certain blood conditions. These include specific types of Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukaemia (CMML-2 or CMML that hasn't responded to previous treatments), or Acute Myeloid Leukaemia (AML) – either if it's new and other strong treatments aren't suitable, or if it has come back despite previous treatments.

There are also some general health checks. For example, your blood counts, kidney, and liver functions need to be at certain levels. Your doctor will carefully review your full medical history to make sure the study is a safe option for you.

However, there are reasons why you might not be able to join. These include having a different type of leukaemia called acute promyelocytic leukaemia, if you've recently had a bone marrow transplant, or if you have certain active autoimmune conditions. Also, if you are pregnant or breastfeeding, or if you've recently had other specific anti-cancer treatments or immunotherapy, you wouldn't be able to take part. Your study doctor will explain all the details.

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?
Do you have a diagnosis of MDS, CMML (specific types), or AML (newly diagnosed and unfit for strong treatment, or relapsed/refractory)?
Are your kidney and liver functions generally healthy?
Are you not pregnant or breastfeeding?
Have you not had a bone marrow transplant in the last 6 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would first go through a 'screening period' where doctors do tests to make sure the study is right for you. If you qualify, you'll then begin the 'treatment period'. This will involve taking the study medicine (bexmarilimab) along with other standard treatments, as recommended by the study team. You'll have regular visits to the clinic for medical check-ups, blood tests, and to make sure you're feeling okay. These visits are important for the team to monitor your health and how the treatment is working. After your treatment period ends, you'll have an 'end of treatment' follow-up to check your safety. There will also be ongoing follow-up appointments to track your health and how your cancer progresses over time. The total duration of your involvement could vary, depending on how you respond to the treatment and how long you remain on the study.

Potential risks and benefits

Taking part in this study may offer potential benefits, such as receiving a new treatment that might help your condition, which isn't yet widely available. You'll also receive close medical attention and monitoring. However, as with any new medicine, there are potential risks and side effects. The study drug, bexmarilimab, and the combination treatments might cause reactions, and these will be carefully monitored by the study team. They will explain all known or expected side effects to you. It's important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

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

City of Hope National Medical Center
Verified postcode
Duarte, United States
Yale Cancer Center
Verified postcode
New Haven, United States
UNC Lineberger Comprehensive Cancer Center
Verified postcode
Chapel Hill, United States
University of Texas, MD Anderson Cancer Center
Verified postcode
Houston, United States
Helsinki University Hospital
Verified postcode
Helsinki, Finland
Kuopio University Hospital
Verified postcode
Kuopio, Finland
Oulu University Hospital
Verified postcode
Oulu, Finland
Tampere University Hospital
Verified postcode
Tampere, Finland
The Christie NHS Foundation Trust
Verified postcode
Manchester, United Kingdom
Royal Cornwall Hospitals NHS Trust
Verified postcode
Truro, United Kingdom

Common questions

What is bexmarilimab?

Bexmarilimab is a new drug being tested to see if it can help treat certain blood cancers by working alongside standard treatments.

What are the main conditions this study is looking at?

The study focuses on Acute Myeloid Leukaemia (AML), Chronic Myelomonocytic Leukaemia (CMML), and Myelodysplastic Syndromes (MDS).

Why are there two phases to the study?

The first phase is to find a safe and suitable dose of bexmarilimab. The second phase uses that safe dose to see how well the treatment works.

Will I get 'standard treatment' even if I join the study?

Yes, bexmarilimab is being tested in combination with 'standard of care' treatments, meaning you would receive both.

Can I stop being part of the study at any time?

Yes, you can choose to leave the study at any point without it affecting your future medical care.