Oral GB2064 for Myelofibrosis

Recruiting at 13 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of an experimental drug, GB2064, for individuals with myelofibrosis, a bone marrow disorder that can cause severe anemia and an enlarged spleen. Participants will take the drug in pill form twice daily, while researchers monitor its effects on their condition. The trial seeks individuals diagnosed with myelofibrosis who exhibit symptoms like a noticeably enlarged spleen and are not currently on certain other treatments. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

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.

Is there any evidence suggesting that GB2064 is likely to be safe for humans?

Research has shown that GB2064 is generally safe for people with myelofibrosis. Studies have found that most patients tolerate it well, with no reports of serious side effects. This treatment may also help reduce bone marrow scarring, which is promising. Overall, evidence suggests that GB2064 is reasonably safe for people with myelofibrosis. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for myelofibrosis, like JAK inhibitors, primarily focus on managing symptoms and controlling blood cell counts. But GB2064 works differently, targeting the fibrosis directly by inhibiting a protein called LOXL2 involved in tissue scarring. Researchers are excited about GB2064 because it offers a new approach that might actually address the underlying cause of the disease, rather than just the symptoms, potentially leading to better long-term outcomes for patients.

What evidence suggests that GB2064 might be an effective treatment for Myelofibrosis?

Research has shown that GB2064, the investigational treatment studied in this trial, might help treat myelofibrosis by reducing the thickening and scarring of tissue in the bone marrow. In one study, six out of ten patients taking GB2064 experienced at least a 1-grade decrease in tissue scarring after six months. Another report found that 80% of patients had improvements in a specific type of tissue change related to myelofibrosis. These findings suggest that GB2064 could effectively manage myelofibrosis by addressing tissue scarring. Overall, the treatment appears promising and could provide a new option for those affected by this condition. ^{1 3 4 6 7}

Who Is on the Research Team?

Richard F Schlenk, MD

Principal Investigator

Universitätsklinikum Heidelberg, Germany

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. ir.galecto.com ir.galecto.com/news-releases/news-release-details/topline-results-mylox-1-trial-demonstrate-reduction-fibrosis

Topline Results from MYLOX-1 Trial Demonstrate Reduction ... Six out of ten evaluable myelofibrosis patients who received GB2064 monotherapy for at least six months experienced a ≥ 1-grade reduction in collagen fibrosis ...

2. onclive.com onclive.com/view/gb2064-displays-preliminary-efficacy-tolerability-in-myelofibrosis

GB2064 Displays Preliminary Efficacy, Tolerability in ... GB2064 displayed efficacy with a generally acceptable tolerability profile in the treatment of patients with myelofibrosis, according to topline findings from ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10431423/

P1024: MYLOX-1: A PHASE II STUDY EVALUATING THE ... Of 5 evaluable pts who received GB2064 for >6 months & had complete BM biopsy data, 1 (20%) showed improved ≥1-grade reticulin fibrosis, & 4 (80 ...

4. targetedonc.com targetedonc.com/view/gb2064-reduces-fibrosis-grade-in-early-results-of-mylox1-study-of-myelofibrosis

In MYLOX-1 Study, GB2064 Reduces Fibrosis Grade ... Early results from the phase 2a MYLOX-1 study show a reduction of collagen fibrosis of the bone marrow in patients with myelofibrosis on the ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04679870?term=AREA%5BConditionSearch%5D(myelofibrosis)%20AND%20AREA%5BOverallStatus%5D(NOT_YET_RECRUITING%20OR%20RECRUITING%20OR%20ACTIVE_NOT_RECRUITING%20OR%20UNKNOWN)&rank=5

NCT04679870 | A Study to Evaluate the Safety, Tolerability ... This study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered GB2064 a LOXL-2 inhibitor over 9 ...

6. ir.galecto.com ir.galecto.com/news-releases/news-release-details/gb2064-shows-reduction-fibrosis-bone-marrow-patients

GB2064 Shows Reduction in Fibrosis of the Bone Marrow in ... GB2064, a potentially first-in-class, LOXL2 inhibitor candidate, is in development for the treatment of fibrotic diseases and cancer.

7. journals.lww.com journals.lww.com/hemasphere/fulltext/2023/08003/p1024__mylox_1__a_phase_ii_study_evaluating_the.923.aspx

P1024: MYLOX-1: A PHASE II STUDY EVALUATING THE ... Pts with MF who received GB2064 had an acceptable safety profile & improvements in BM reticulin & collagen fibrosis that indicated disease-modifying activity.