Active not recruitingPHASE2INTERVENTIONAL

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis

This research study is looking at a new drug, GB2064, for people who have myelofibrosis. Myelofibrosis is a condition where scar tissue builds up in the bone marrow, affecting how blood is made. This study will give GB2064 to participants for nine months to see if it's safe, how well the body takes it in and uses it, and if it helps with the condition. It's an early-stage study, meaning researchers are learning about the drug's basic effects. The aim is to find out if GB2064 could be a helpful treatment option in the future for this serious blood disorder.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Galecto Biotech AB

Enrolment target

Start

09 Jun 2021

Estimated completion

30 Jun 2026

Myelofibrosis

What is this study about?

This study is investigating a new medicine called GB2064 for people with myelofibrosis. Myelofibrosis is a rare type of cancer that affects the bone marrow, which is the spongy tissue inside some of your bones where blood cells are made. In myelofibrosis, scar tissue builds up in the bone marrow, meaning it can't make enough healthy blood cells. This can lead to symptoms like feeling very tired, having a large spleen, and bruising easily.

Researchers want to see if GB2064 can help. This drug works by targeting something in the body called LOXL-2, which is thought to be involved in the scarring process in myelofibrosis. Before a new medicine can be widely used, it goes through several stages of testing. This particular study is in an early stage (Phase 2a), which means the main focus is on checking if GB2064 is safe and if the body handles it well. They will also look for any signs that it might be having a positive effect on the condition.

The study aims to gather important information over nine months about how GB2064 works when taken by mouth. The findings from studies like this help scientists understand if new medicines are promising enough to move on to larger studies and potentially become available as future treatments for people living with myelofibrosis.

Key takeaways

This study is testing a new, experimental medicine (GB2064) for myelofibrosis.
The main goals are to check the medicine's safety and how the body handles it.
Participation involves taking tablets twice a day for nine months.
You cannot be currently taking certain other myelofibrosis medicines (JAK inhibitors).
Regular clinic visits and blood tests will be part of the study.
Patients can leave the study at any time without affecting their normal care.

Who may be eligible?

To join this study, you need to be an adult, 18 years or older, with a diagnosis of myelofibrosis. This includes either myelofibrosis that started on its own (primary) or developed from another blood condition (secondary). Your myelofibrosis needs to be classified as 'intermediate-2' or 'high-risk', or, if low-risk, you must have a noticeably enlarged spleen that causes symptoms.

Another key requirement is that you must not be currently taking a type of medicine called a JAK inhibitor (like ruxolitinib or fedratinib). This usually means that your doctor has decided these treatments aren't suitable for you, or they haven't worked well in the past. Your overall health and how well you can perform daily activities will also be checked, along with certain blood test results, such as your platelet and white blood cell counts, and how well your liver and kidneys are working.

Both men and women can join. If you are a woman who could become pregnant, you'll need to use highly effective birth control during and for 3 months after the study. If you're a man, you'll also need to use contraception during and for 3 months after the study, and refrain from donating sperm. Women will also need to avoid donating eggs during the study period.

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 primary or secondary myelofibrosis with specific risk factors or an enlarged spleen?
Are you NOT currently taking a JAK inhibitor medicine (like ruxolitinib or fedratinib) for your myelofibrosis, or have you been told these medicines are not suitable for you?
Are you able to attend regular clinic appointments for about 9-10 months?
Are you willing and able to use contraception if you are a man or a woman who could become pregnant?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the study medicine, GB2064, twice a day by mouth. The study will last for nine months. Throughout this time, you would have regular visits to the clinic for various checks. These check-ups will likely include physical examinations, blood tests, and other assessments to monitor your health and see how your body is reacting to the medicine.

The research team will closely watch for any side effects and how your body processes the drug. After the nine months of treatment, there will be a follow-up visit to ensure your well-being. The total length of your participation, including follow-up, will be around ten months.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. A potential benefit of joining this study is that you would receive a new medicine, GB2064, that is not yet widely available, which might help your myelofibrosis. However, as with any new medicine, there could be unknown side effects or the medicine might not work for you. The research team will carefully monitor your health throughout the study for any issues. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (11)

MD Andersson Cancer Hospital
Verified postcode
Houston, United States
Woden Dermatology
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Canberra, Australia
Heinrich-Heine-University Dusseldorf
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Düsseldorf, Germany
Universitätsklinikum Heidelberg
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Heidelberg, Germany
Universität Leipzig
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Leipzig, Germany
Klinikum rechts der Isar der Technischen Universitaet Munchen
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München, Germany
University of Bologna Sant Orsola Malpighi
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Bologna, Italy
Azienda Ospedaliero-Universitaria Careggi
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Florence, Italy
ASST Grande Ospedale Metropolitano Niguarda
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Milan, Italy
Azienda Ospedaliero-Universitaria San Luigi Gonzaga di Orbassano
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Orbassano, Italy
Azienda Socio-Sanitaria Territoriale dei Sette Laghi
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Varese, Italy

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where scar tissue builds up in your bone marrow, affecting how your body makes healthy blood cells.

What is GB2064?

GB2064 is a new medicine being tested in this study to see if it can help with myelofibrosis by targeting a specific process in the body.

How long does the study last?

The study involves taking the medicine for nine months, plus an additional follow-up period, making it about 10 months in total.

Are there other treatment options for myelofibrosis?

Yes, there are other treatments available or being tested for myelofibrosis. Your doctor can discuss these with you.

What does 'Phase 2a trial' mean?

It means this is an early stage of testing for a new medicine, mainly focused on checking its safety, how the body handles it, and if there are any early signs it could be helpful.