AzTrials
AzTrials

Acute Myeloid LeukemiaPhase 1NCT06146257

A Study of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher Risk Myelodysplastic Syndromes

A Phase 1 study testing GLB-001 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by GluBio Therapeutics Inc. at 8 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

Study GLB-001-01 is a first-in-human (FIH), Phase 1, open-label, dose escalation and expansion clinical study of GLB-001 in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in participants with relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
GLB-001
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
48 participants
Where
8 sitesUnited States
Study dates
11 Jan 2024to 8 Oct 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants is ≥ 18 years of age at the time of signing the Informed Consent Form (ICF).
  • Participants must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
  • Participants are willing and able to adhere to the study visit schedule and other protocol requirements.
  • Participants with histologically or cytologically confirmed AML including de novo AML or secondary AML transformed from MDS according to 2022 World Health Organization (WHO) criteria classification, or with histologically or cytologically confirmed HR-MDS.
  • R/R AML and R/R HR-MDS who have failed or are ineligible for all available therapies which may provide clinical benefit.
  • Participants must have the following screening laboratory values:
Show all 14Show fewer
  • Total white blood cell count (WBC) < 25 x 10^9/L prior to the first dose of the study drug.
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 × upper limit of normal (ULN), unless considered due to extensive leukemic liver involvement, in which case AST and ALT can be ≤ 5.0 x ULN.
  • Serum total bilirubin ≤ 1.5 x ULN, unless considered due to Gilbert's syndrome, in which case serum total bilirubin < 3 x ULN.
  • Estimated serum creatinine clearance of ≥ 60 mL/min using the Cockcroft-Gault equation. Measured creatinine clearance from a 24-hour urine collection is acceptable if clinically indicated.
  • International normalized ratio (INR) ≤ 1.5 x ULN and active partial thromboplastin time (aPTT) ≤ 1.5 x ULN.
  • Life expectancy ≥ 12 weeks.
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2.
  • Female Participants of child-bearing potential must have a negative serum or urine pregnancy test at screening and at pre-dose on Cycle 1 Day 1 (C1D1).

Who cannot join

  • Participants with acute promyelocytic leukemia (APML).
  • Participants with known leukemic involvement in central nervous system (CNS).
  • Receipt of anticancer medications/therapies within 5 half-lives or 28 days before the first administration of the study drug.
  • Participants with unresolved clinically significant non-hematologic toxicities of ≥ Grade 2 AE from prior therapies with exception of residual alopecia.
  • Participants with chronic graft versus host disease (GVHD) requiring systemic immunosuppressive therapy.
  • Participants with active malignancies other than AML or MDS.
Show all 13Show fewer
  • Participants who have undergone major surgery ≤ 4 weeks prior to the first dose of the study drug.
  • Participants with immediately life-threatening, severe complications of leukemia such as disseminated/uncontrolled infection (bacterial and/or fungal), uncontrolled bleeding, and/or uncontrolled disseminated intravascular coagulation.
  • Participants with known chronic, active infection of hepatitis B virus (HBV), hepatitis C virus C (HCV), human immunodeficiency virus (HIV).
  • Participants unable to swallow oral medications, or Participants with clinically significant diarrhea, vomiting or malabsorption felt limited absorption of orally administered medications.
  • Participants with any other significant medical conditions, any other conditions, laboratory abnormality, or psychiatric illness which place the Participants at unacceptable risk if he/she were to participate in the study or that would hamper the Participants understanding of the study, or would prevent the Participant from complying with the study.
  • Medications or supplements that are known to be strong and moderate inhibitors or inducers of CYP450 isozyme 3A4 (CYP3A4) and/or P-glycoprotein (P-gp), or strong inhibitors or inducers of CYP450 isozyme 2C8 (CYP2C8) within 14 days or 5 half-lives, whichever is shorter, before the first dose of study drug.
  • Pregnant or lactating women.

Trial locations and contacts

8 sites in 1 country. Contact the study team or a site near you to ask about taking part.

8 sites in 1 country

United States8 sites, 3 recruiting
  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263

    Terminated
  • City of Hope Medical Center

    Duarte, California, 91010

    Brian Ball, MD, 626-218-4784, brball@coh.org

    Recruiting
  • University of Texas M. D. Anderson Cancer Center

    Houston, Texas, 77030

    Terminated
  • University of California Irvine

    Irvine, California, 92697

    Terminated
  • University of Kansas Medical Center Research Institute, Inc.

    Kansas City, Kansas, 66160

    Jesus Gonzalez Lugo, MD, 913-588-4138, jgonzalezlugo@kumc.edu

    Recruiting
  • Alliance for Multispecialty Research, LLC

    Merriam, Kansas, 66204

    Terminated
  • Memorial Sloan Kettering Cancer Center-David H. Koch Center

    New York, New York, 10021

    Terminated
  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

    Lewis Silverman, MD, 212-241-5520, Lewis.silverman@mssm.edu

    Recruiting

Other trials in United States

About this study

Official title: A First-in-human, Phase 1, Dose Escalation and Expansion Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes

Study GLB-001-01 is a first-in-human (FIH), Phase 1, open-label, dose escalation and expansion clinical study of GLB-001 in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in participants with relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS). The dose escalation part (Phase 1a) of the study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of GLB-001 administered orally. Approximately 24 participants (up to 42 participants) may be enrolled in Phase 1a of the study.

The dose expansion part (Phase 1b) will be followed to understand the relationships among dose, exposure, toxicity, tolerability and clinical activity, to identify minimally active dose, and to select the recommended dose(s) for phase 2 study. Up to 24 participants (12 participants per dose level) may be enrolled in Phase 1b of the study.

Read the detailed description

A standard 3+3 dose-escalation design will be applied to evaluate a set of several dose levels to determine the maximum tolerated dose (MTD) or maximum administered dose (MAD) of GLB-001 in R/R AML or R/R HR-MDS patients who are eligible for DLT evaluation. The actual dose-escalation magnitude or dosing frequency may be adjusted based on the available PK and safety data in human.

After the MTD or MAD of GLB-001 is defined in Phase 1a, 1 or 2 dose levels will be selected for expansion per safety review committee (SRC) recommendation, approximately 12 patients will be enrolled per dose level. Recommended phase 2 dose (RP2D) will be selected based on the results of PK, PD, safety and efficacy in the dose escalation and expansion study.

Being tested
GLB-001
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GLB-001Drug

    Also called GLB-C183-A-2

    Administered orally according to the assigned treatment schedule

Study groups in NCT06146257
Study groupRoleWhat they receive
Dose Escalation of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1aExperimentalPart 1a (Dose Escalation) of the study will enroll R/R AML and R/R HR-MDS participants and will evaluate the safety, tolerability, PK, PD and preliminary efficacy of GLB-001 administered orally, and determine the maximum tolerated dose/maximum administered dose (MTD/MAD) in R/R AML or R/R HR-MDS patients who are eligible for dose limiting toxicity (DLT) evaluation.
Receives: Drug: GLB-001
Dose Expansion of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1bExperimentalPart 1b (Dose Expansion) will confirm tolerability of the selected doses and schedules and evaluate whether efficacy is in a range that warrants further clinical development for R/R AML and R/R HR-MDS participants.
Receives: Drug: GLB-001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Dose-limiting Toxicity (DLT)

    Dose-limiting toxicity is defined as the treatment emergent adverse events (TEAEs) meeting protocol specified DLT criteria and occurring within the DLT assessment period.

    Measured: Up to 28 days after first dose of study treatment in Phase 1a

  2. Maximum Tolerated Dose (MTD)/Maximum Administered Dose (MAD)

    Maximum tolerated dose is defined as the highest dose level at which no more than 1 of 6 DLT-evaluable participants experienced a DLT. If MTD is not established at the end of dose escalation phase, the maximum safety dose will be defined as Maximum administered dose.

    Measured: Up to 2 years

  3. Incidence of Adverse Events (AEs)

    Adverse Events will be graded according to the National Cancer Institute Common Terminology Criteria for AE (NCI CTCAE) version 5.0.

    Measured: Up to 2 years

  4. Recommended Phase 2 Dose (RP2D)

    Recommended phase 2 dose based on the totality of data across dosing cohorts in the dose escalation and expansion phases of the study including PK, PD, safety and efficacy outcomes.

    Measured: Up to 2 years

Secondary outcomes (34)
  1. GLB-001 Pharmacokinetics-AUC0-last

    Area under the concentration-time curve from zero to the last measurable concentration.

    Measured: Up to 2 years

  2. GLB-001 Pharmacokinetics-AUC0-24

    Area under the concentration-time curve from 0 to 24 hours.

    Measured: Up to 2 years

  3. GLB-001 Pharmacokinetics-AUC0-∞

    Area under the concentration-time curve from 0 to infinity.

    Measured: Up to 2 years

  4. GLB-001 Pharmacokinetics-Cmax

    Maximum plasma concentration.

    Measured: Up to 2 years

  5. GLB-001 Pharmacokinetics-Tmax

    The time to reach maximum concentration.

    Measured: Up to 2 years

  6. GLB-001 Pharmacokinetics-T1/2

    Terminal half-life.

    Measured: Up to 2 years

  7. GLB-001 Pharmacokinetics-Vd/F

    Apparent volume of distribution.

    Measured: Up to 2 years

  8. GLB-001 Pharmacokinetics-LI

    Linear index.

    Measured: Up to 2 years

  9. GLB-001 Pharmacokinetics-CL/F

    Apparent clearance.

    Measured: Up to 2 years

  10. GLB-C183-A-2R (Isomer of GLB-001) Pharmacokinetics-AUC0-last

    Area under the concentration-time curve from zero to the last measurable concentration.

    Measured: Up to 2 years

  11. GLB-C183-A-2R Pharmacokinetics-AUC0-24

    Area under the concentration-time curve from 0 to 24 hours.

    Measured: Up to 2 years

  12. GLB-C183-A-2R Pharmacokinetics-AUC0-∞

    Area under the concentration-time curve from 0 to infinity.

    Measured: Up to 2 years

  13. GLB-C183-A-2R Pharmacokinetics-Cmax

    Maximum plasma concentration.

    Measured: Up to 2 years

  14. GLB-C183-A-2R Pharmacokinetics-Tmax

    The time to reach maximum concentration.

    Measured: Up to 2 years

  15. GLB-C183-A-2R Pharmacokinetics-T1/2

    Terminal half-life.

    Measured: Up to 2 years

  16. GLB-C183-A-2R Pharmacokinetics-Vd/F

    Apparent volume of distribution.

    Measured: Up to 2 years

  17. GLB-C183-A-2R Pharmacokinetics-LI

    Linear index.

    Measured: Up to 2 years

  18. GLB-C183-A-2R Pharmacokinetics-CL/F

    Apparent clearance.

    Measured: Up to 2 years

  19. Complete Remission Without Minimal Residual Disease (CRMRD-) Rate in Participants with Acute Myeloid Leukemia (AML)

    CRMRD- rate is defined as the percent of participants with minimal residual disease negative complete remission. CRMRD- will be assessed by the 2022 European Leukemia Net Response Criteria (2022 ELN) for AML .

    Measured: Up to 2 years

  20. Complete Remission (CR) Rate in Participants with AML

    CR rate is defined as the percent of participants whose best response is CR. CR will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  21. CR with Incomplete Hematologic Recovery (CRi) Rate in Participants with AML

    CRi rate is defined as the percent of participants whose best response is CRi. CRi will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  22. CR with Partial Hematological Recovery (CRh) Rate in Participants with AML

    CRh rate is defined as the percent of participants whose best response is CRh. CRh will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  23. Morphologic Leukemia-free State (MLFS) Rate in Participants with AML

    MLFS rate is defined as the percent of participants with the best response of morphologic leukemia-free state. MLFS will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  24. Partial Remission (PR) Rate in Participants with AML

    PR rate is defined as the percent of participants whose best response is PR. PR will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  25. Duration of Remission or Response (DOR) in Participants with AML

    For participants with best response of any of CRMRD-, CR, CRh, CRi, PR, and MLFS, DOR is measured from the time when criteria (2022 ELN for AML) for the best response of any of CRMRD-, CR, CRh, CRi, PR, and MLFS are first met (whichever is first recorded) until the first date at which relapse, or progressive disease is objectively documented assessment.

    Measured: Up to 2 years

  26. Time to Remission or Response (TOR) in Participants with AML

    Time to onset of first remission or response is defined as the time interval from the date of first dose and the earliest date any remission or response [any CRs (including CR, CRh and CRi) or PR] is observed.

    Measured: Up to 2 years

  27. Stable Disease (SD) Rate in Participants with AML

    SD rate is defined as the percent of participants with the best response of stable disease. SD will be assessed by 2022 ELN for AML.

    Measured: Up to 2 years

  28. CR Rate in Participants with Higher Risk Myelodysplastic Syndromes (HR-MDS)

    CR rate is defined as the percent of participants whose best response is CR. CR will be assessed by 2006 Modified International Working Group (IWG) MDS response criteria.

    Measured: Up to 2 years

  29. PR Rate in Participants with HR-MDS

    PR rate is defined as the percent of participants whose best response is PR. PR will be assessed by 2006 Modified IWG MDS response criteria.

    Measured: Up to 2 years

  30. SD Rate in Participants with HR-MDS

    SD rate is defined as the percent of participants with the best response of stable disease. SD will be assessed by 2006 Modified IWG MDS response criteria.

    Measured: Up to 2 years

  31. DOR in Participants with HR-MDS

    For participants with best response of any of CR, marrow CR, or PR, DOR is measured from the time when criteria (2006 Modified IWG MDS response criteria) for the best response of any of CR, marrow CR, or PR are first met (whichever is first recorded) until the first date at which relapse or progressive disease is objectively documented assessment.

    Measured: Up to 2 years

  32. TOR in Participants with HR-MDS

    Time to onset of first remission or response is defined as the time interval from the date of first dose of GLB-001 and the earliest date any remission or response [any CRs (including CR, marrow CR or PR)] is observed.

    Measured: Up to 2 years

  33. Progression-free Survival (PFS) in Participants with AML or HR-MDS

    PFS is defined as the time from the first dose of GLB-001 to the first occurrence of relapse or progression or death from any cause.

    Measured: Up to 2 years

  34. Overall Survival (OS) in Participants with AML or HR-MDS

    OS is defined as the time from the first dose of GLB-001 to death due to any cause.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Nov 2023
  2. Study start11 Jan 2024
  3. Primary completion1 Jul 2026 (estimated)
  4. Study completion8 Oct 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
48 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06146257
Sponsor's protocol ID
GLB-001-01
Status last verified
Apr 2026
First submitted
10 Nov 2023
Last updated
13 Apr 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing GLB-001 in people with Acute Myeloid Leukemia and Myelodysplastic Syndromes, run by GluBio Therapeutics Inc. at 8 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Kimberly Glen (984-260-8186, Kimberly.glen@glubiotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose-limiting Toxicity (DLT) (time frame: Up to 28 days after first dose of study treatment in Phase 1a). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in