48 Participants Needed

GLB-001 for Acute Myeloid Leukemia

Recruiting at 7 trial locations
HZ
Overseen ByHongying Zhang, MD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, GLB-001, in patients with certain types of blood cancer that haven't responded to other treatments. The goal is to find out how safe the drug is and what dose works best.

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial, especially if they are strong or moderate inhibitors or inducers of specific enzymes (CYP3A4, CYP2C8) or P-glycoprotein. This should be done within 14 days or 5 half-lives of the medication, whichever is shorter, before starting the study drug.

What safety data exists for GLB-001 (glasdegib) in humans?

Glasdegib has been studied in combination with low-dose cytarabine for acute myeloid leukemia, and it was generally well-tolerated in clinical trials, leading to its FDA approval for certain patients. However, there are concerns about side effects when combined with other treatments, as it may affect blood-forming cells.12345

How does the drug GLB-001 differ from other treatments for acute myeloid leukemia?

GLB-001 may involve targeting the Hedgehog signaling pathway, similar to glasdegib, which is a known treatment for AML that works by inhibiting this pathway to improve survival in patients unsuitable for intensive chemotherapy. This approach is unique as it focuses on a specific molecular target, potentially offering a novel strategy for enhancing drug sensitivity in AML treatment.12678

What data supports the effectiveness of the drug GLB-001 for treating acute myeloid leukemia?

Research shows that inhibiting GLI1, a gene involved in the Hedgehog signaling pathway, can make acute myeloid leukemia cells more sensitive to drugs. This suggests that targeting GLI1, which GLB-001 may do, could be a promising strategy for treating this type of leukemia.156910

Who Is on the Research Team?

GL

Gang Lu, Ph.D.

Principal Investigator

GluBio Therapeutics Inc.

Are You a Good Fit for This Trial?

This trial is for adults over 18 with relapsed or refractory acute myeloid leukemia (R/R AML) or higher-risk myelodysplastic syndromes (HR-MDS). They must have failed or be ineligible for other treatments and meet specific health criteria like blood counts, liver and kidney function. Pregnant women are excluded.

Inclusion Criteria

My blood tests and health status meet the study's requirements.
You need to understand and agree to sign a document before any study-related tests or procedures are done.
You can commit to attending all the study visits and follow the study rules.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Phase 1a: Evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of GLB-001 to determine the maximum tolerated dose or maximum administered dose.

Up to 28 days after first dose

Dose Expansion

Phase 1b: Confirm tolerability of selected doses and evaluate efficacy to identify minimally active dose and select recommended dose for phase 2.

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • GLB-001
Trial Overview GLB-001, an oral medication, is being tested in this Phase 1 study on patients with certain types of blood cancers. The study has two parts: dose escalation to assess safety and dosage levels, followed by dose expansion to find the most effective yet tolerable dose for future studies.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Dose Expansion of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1bExperimental Treatment1 Intervention
Group II: Dose Escalation of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1aExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

GluBio Therapeutics Inc.

Lead Sponsor

Trials
1
Recruited
50+

Published Research Related to This Trial

In a study of 42 adult AML patients, researchers found that the Hedgehog (Hh) pathway, particularly the GLI1 protein, is overexpressed in patients with refractory/relapse AML, leading to decreased drug sensitivity.
Inhibiting GLI1 alone was shown to enhance the sensitivity of AML cells to treatment, suggesting that targeting GLI1 could be a promising strategy for improving outcomes in AML without the added risks associated with combination therapies.
Targeting the PI3K/AKT pathway via GLI1 inhibition enhanced the drug sensitivity of acute myeloid leukemia cells.Liang, H., Zheng, QL., Fang, P., et al.[2022]
In a study of 46 newly diagnosed acute myeloid leukemia (AML) patients, GLI-1 was found to be overexpressed in bone marrow samples, particularly in patients with poor risk categories, indicating its potential role as a prognostic marker.
Higher levels of GLI-1 mRNA were associated with a lack of complete remission after chemotherapy, suggesting that GLI-1 overexpression could be a novel therapeutic target for improving outcomes in AML.
High GLI-1 Expression is a Reliable Indicator of Bad Prognosis in Newly Diagnosed Acute Leukemia Patients.El Zaiat, RS., Nabil, R., Khalifa, KA., et al.[2023]
Overexpression of the GLI1 gene in acute myeloid leukemia (AML) cells promotes cell growth and reduces sensitivity to chemotherapy, indicating that targeting GLI1 could improve treatment outcomes.
Inhibiting GLI1 with GANT61, along with using the CDK4/6 inhibitor PD 0332991, showed synergistic effects in enhancing chemotherapy sensitivity, suggesting a potential new therapeutic strategy for patients with relapsed or refractory AML.
GLI1 reduces drug sensitivity by regulating cell cycle through PI3K/AKT/GSK3/CDK pathway in acute myeloid leukemia.Zhou, C., Du, J., Zhao, L., et al.[2021]

Citations

Targeting the PI3K/AKT pathway via GLI1 inhibition enhanced the drug sensitivity of acute myeloid leukemia cells. [2022]
High GLI-1 Expression is a Reliable Indicator of Bad Prognosis in Newly Diagnosed Acute Leukemia Patients. [2023]
GLI1 reduces drug sensitivity by regulating cell cycle through PI3K/AKT/GSK3/CDK pathway in acute myeloid leukemia. [2021]
Survival outcomes and clinical benefit in patients with acute myeloid leukemia treated with glasdegib and low-dose cytarabine according to response to therapy. [2021]
Is targeted therapy feasible in acute myelogenous leukemia? [2021]
Glasdegib plus intensive/nonintensive chemotherapy in untreated acute myeloid leukemia: BRIGHT AML 1019 Phase III trials. [2020]
Osteolytic Bone Lesions - A Rare Presentation of AML M6. [2020]
Glasdegib for the treatment of adult patients with newly diagnosed acute myeloid leukemia or high-grade myelodysplastic syndrome who are elderly or otherwise unfit for standard induction chemotherapy. [2019]
An evaluation of glasdegib for the treatment of acute myelogenous leukemia. [2020]
Interpreting data in AML. [2013]
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