Recruiting
Phase 1

GLB-001

Sponsor:

GluBio Therapeutics Inc.

Code:

NCT06146257

Conditions

Acute Myeloid Leukemia

Myelodysplastic Syndromes

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

GLB-001

Study Details

Brief 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). 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.

Conditions

Acute Myeloid Leukemia

Myelodysplastic Syndromes

Study ID

NCT06146257

Start date

Jan 11, 2024

Status verified date

Apr, 2026

Completion date

Oct 8, 2026

Anticipated

Primary completion date

Jul 1, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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:

  • 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).

Exclusion Criteria:

  • 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.
  • 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.

Study Design

Enrollment

48 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Dose Escalation of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1a

Part 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.

experimental: Dose Expansion of GLB-001 as a Monotherapy in Participants with R/R AML and R/R HR-MDS-Phase 1b

Part 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.

Interventions

GLB-001

Administered orally according to the assigned treatment schedule

Primary outcome measure

  • Dose-limiting Toxicity (DLT) [ Time Frame: Up to 28 days after first dose of study treatment in Phase 1a ]
  • Maximum Tolerated Dose (MTD)/Maximum Administered Dose (MAD) [ Time Frame: Up to 2 years ]
  • Incidence of Adverse Events (AEs) [ Time Frame: Up to 2 years ]
  • Recommended Phase 2 Dose (RP2D) [ Time Frame: Up to 2 years ]

Central Contacts and Locations

Central contacts

Kimberly Glen

984-260-8186Kimberly.glen@glubiotx.com

Locations

City of Hope Medical Center

Recruiting

Duarte, California, United States, 91010

Contacts

Brian Ball, MD

626-218-4784brball@coh.org

University of Kansas Medical Center Research Institute, Inc.

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Jesus Gonzalez Lugo, MD

913-588-4138jgonzalezlugo@kumc.edu

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Lewis Silverman, MD

212-241-5520Lewis.silverman@mssm.edu

More Information

Sponsor

GluBio Therapeutics Inc.

Last update posted

Apr 13, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by GluBio Therapeutics Inc. on 2026-04-13.