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

NCT06146257 | Recruiting Phase 1 FDA Drug

• 1 other identifier: GLB-001-01
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 8

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

Outcome Measures

Primary Outcomes (4)

• 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.

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

• 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.

  Up to 2 years

• 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.

  Up to 2 years

• 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.

  Up to 2 years

Secondary Outcomes (34)

• GLB-001 Pharmacokinetics-AUC0-last

  Up to 2 years

• GLB-001 Pharmacokinetics-AUC0-24

  Up to 2 years

• GLB-001 Pharmacokinetics-AUC0-∞

  Up to 2 years

• GLB-001 Pharmacokinetics-Cmax

  Up to 2 years

• GLB-001 Pharmacokinetics-Tmax

  Up to 2 years

Study Arms (2)

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

EXPERIMENTAL

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.

Drug: GLB-001

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

EXPERIMENTAL

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.

Drug: GLB-001

Interventions

GLB-001 DRUG

Administered orally according to the assigned treatment schedule

Also known as: GLB-C183-A-2

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• GluBio Therapeutics Inc.: lead

Study Sites (8)

City of Hope Medical Center

Duarte, California, 91010, United States

RECRUITING

University of California Irvine

Irvine, California, 92697, United States

TERMINATED

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, 66160, United States

RECRUITING

Alliance for Multispecialty Research, LLC

Merriam, Kansas, 66204, United States

TERMINATED

Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14263, United States

TERMINATED

Memorial Sloan Kettering Cancer Center-David H. Koch Center

New York, New York, 10021, United States

TERMINATED

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

University of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030, United States

TERMINATED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Study Officials

• Gang Lu, Ph.D.

  GluBio Therapeutics Inc.

  STUDY DIRECTOR

Central Study Contacts

Kimberly Glen

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2023
• First Posted: November 24, 2023
• Study Start: January 11, 2024
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): October 8, 2026
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

US (8)