What side effects are associated with this type of intervention?

The most common treatments for Myelofibrosis, particularly JAK inhibitors like ruxolitinib and fedratinib, are associated with several side effects. Ruxolitinib can cause anemia, thrombocytopenia, and has been linked to a withdrawal syndrome characterized by fever, hypotension, and hypoxia. Fedratinib also reports similar hematologic side effects, including grade ≥3 anemia and thrombocytopenia. General adverse effects of JAK inhibitors include nausea, headache, and an increased risk of serious infections, major cardiovascular events, cancer, thrombosis, and death.

What prior FDA approvals exist?

The FDA has approved several JAK inhibitors for the treatment of Myelofibrosis. Ruxolitinib, a JAK1 and JAK2 inhibitor, was one of the first to be approved and has shown efficacy in reducing spleen size and alleviating symptoms in patients with Myelofibrosis. Another JAK inhibitor, Fedratinib, has also been approved for patients who are resistant or intolerant to Ruxolitinib. These treatments work by inhibiting the Janus kinase pathways, which helps to reduce abnormal blood cell production and alleviate symptoms associated with Myelofibrosis.

What other types of research exist?

A promising novel alternative to JAK inhibitors for Myelofibrosis patients is GB2064, which is currently being evaluated in an open-label, phase IIa trial. This treatment is being studied for its safety, tolerability, pharmacokinetics, and pharmacodynamics in Myelofibrosis patients.

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Oral GB2064 for Myelofibrosis

Phase 2

Waitlist Available

Led By Srdan Verstovsek, MD, PhD

Research Sponsored by Galecto Biotech AB

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 9 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new medication called GB2064, which blocks a protein known as LOXL-2. It is aimed at patients with Myelofibrosis, a condition where the bone marrow becomes scarred. By blocking LOXL-2, the medication hopes to reduce this scarring and improve symptoms.

Who is the study for?

Adults over 18 with Myelofibrosis who haven't responded well to, or can't take, JAK inhibitors. They should have a low blast count in blood, be able to perform daily activities (ECOG 0-2), and have proper organ function. Women must use effective birth control or be non-childbearing; men must also agree to contraception.

What is being tested?

The trial is testing GB2064, an oral medication for Myelofibrosis patients. It's an open-label phase IIa study which means everyone gets the drug and both the researchers and participants know what's being given.

What are the potential side effects?

Specific side effects of GB2064 aren't listed but may include typical reactions like nausea, fatigue, liver issues based on its pharmacological class. Participants will be monitored for any adverse effects throughout the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 9 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~9 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 9 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and tolerability of GB2064: AE

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: GB2064Experimental Treatment1 Intervention

GB2064 will be administered orally as 4 x 250 mg tablets twice a day.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

JAK inhibitors, such as ruxolitinib, work by targeting the Janus kinase (JAK) pathways, which are involved in the signaling processes that lead to abnormal blood cell production in Myelofibrosis. By inhibiting these pathways, these treatments can reduce the overproduction of blood cells and alleviate symptoms like splenomegaly and systemic inflammation. This mechanism is crucial for Myelofibrosis patients as it directly addresses the disease's underlying causes, potentially improving both quality of life and overall disease outcomes.