Oral GB2064 for Myelofibrosis

Recruiting at 11 trial locations

Overseen By Bertil Lindmark, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Galecto Biotech AB

Must not be taking: JAK inhibitors, Corticosteroids

Disqualifiers: HIV, Hepatitis, Cardiac diseases, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 9 months

21 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that you are not currently taking a Janus kinase (JAK) inhibitor and have not taken one within two weeks before joining. If you are on chronic corticosteroids or other immunosuppressive treatments, you must be able to stop them before starting the study drug.

How does the drug GB2064 differ from other treatments for myelofibrosis?

GB2064 is an oral treatment for myelofibrosis, which is unique because it offers a non-invasive administration route compared to the more invasive stem cell transplantation, the only current curative option. Unlike conventional drugs that mainly provide symptom relief, GB2064 may target the underlying disease mechanisms, although specific details about its mechanism of action are not provided in the available research. ^{1 2 3 4 5}

Research Team

Richard F Schlenk, MD

Principal Investigator

Universitätsklinikum Heidelberg, Germany

Eligibility Criteria

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.

Inclusion Criteria

Peripheral blood blasts <10%

You are not currently taking a medication called Janus kinase (JAK) inhibitor and cannot take it due to medical reasons.

Female participants may be of non-childbearing potential defined as permanently sterile or postmenopausal, or female participants considered to be of childbearing potential who agree to use highly effective birth control methods until 90 days after the follow-up visit.

Exclusion Criteria

Hypersensitivity to GB2064 and/or its excipients.

Current treatment with a JAK inhibitor (e.g. ruxolitinib or fedratinib) or a history of treatment with a JAK inhibitor within two weeks of enrolment.

Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral GB2064, a LOXL2 inhibitor, twice daily for Myelofibrosis

9 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• GB2064

Trial Overview 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.

Participant Groups

Group I: GB2064 Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Galecto Biotech AB

Lead Sponsor

Trials

9

Recruited

580+

OPIS s.r.l

Collaborator

Trials

1

Recruited

20+

Findings from Research

Myelofibrosis (MF) is a serious blood cancer with varying prognoses; patients with low-risk disease can survive 6-15 years, while those with high-risk disease or transformed acute myeloid leukemia (AML) have a much poorer prognosis, often less than 3 months.

The only curative treatment for MF is allogeneic stem cell transplantation, but since most patients are ineligible, they typically receive older drugs for symptom relief. However, new JAK1/JAK2 inhibitors like ruxolitinib show promise based on positive results from large phase III studies, indicating a potential shift in treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and laboratory features of myelofibrosis and limitations of current therapies. Gregory, SA., Mesa, RA., Hoffman, R., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Clinical and laboratory features of myelofibrosis and limitations of current therapies. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Conventional and experimental drug therapy in myelofibrosis with myeloid metaplasia. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Identification of patients with poorer survival in primary myelofibrosis based on the burden of JAK2V617F mutated allele. [2021]

4. France pubmed.ncbi.nlm.nih.gov

Splenic irradiation prior to allogeneic transplant conditioning in myelofibrosis: A pilot experience. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Application of five prognostic survival scores to primary myelofibrosis in 62 Brazilian patients. [2021]