A Study of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher Risk Myelodysplastic Syndromes
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests GLB-001, a drug taken by mouth, in people for the first time. It will check safety, look for signs that it works, and help choose doses for future studies. It is for adults with acute myeloid leukemia (AML), a blood cancer, or higher-risk myelodysplastic syndromes (MDS), serious bone marrow diseases, that came back or did not respond to treatment.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18 or older.
- Understand and sign the consent form, and be able to follow the study schedule and rules.
- Have AML or higher-risk MDS confirmed by tissue or cell tests.
- Have disease that came back or did not respond to treatment, with all other treatments that may help either unsuccessful or not suitable.
- Meet the study’s blood test limits for white blood cells, liver and kidney function, and blood clotting.
- Be expected to live at least 12 weeks.
- Be able to care for yourself and be up and about for more than half of your waking hours.
- If you can become pregnant, have negative pregnancy tests at screening and before the first dose.
Who can't join
- Have acute promyelocytic leukemia, a type of AML, or leukemia in the brain or spinal cord.
- Have another active cancer, or serious, life-threatening problems from leukemia, such as uncontrolled infection, bleeding, or blood clotting.
- Have received cancer treatment too recently, or had major surgery within 4 weeks before the first dose.
- Still have moderate or worse treatment side effects not related to blood counts, except hair loss; or have ongoing graft-versus-host disease requiring medicines that weaken the immune system. This disease occurs when donated cells attack your body.
- Have a known ongoing, active hepatitis B, hepatitis C, or HIV infection.
- Cannot swallow pills, or have serious diarrhea, vomiting, or trouble absorbing medicines.
- Have another health or mental health condition that makes the study unsafe or hard to follow, or recently used medicines or supplements that affect certain proteins that handle drugs in the body.
- Be pregnant or breastfeeding.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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 sites (8)
CA
- City of Hope Medical Center · Duarte, CA 91010Brian Ball, MD · Study contact · 626-218-4784 · brball@coh.orgMore trials in Duarte
- University of California Irvine · Irvine, CA 92697More trials in Irvine
KS
- University of Kansas Medical Center Research Institute, Inc. · Kansas City, KS 66160Jesus Gonzalez Lugo, MD · Study contact · 913-588-4138 · jgonzalezlugo@kumc.eduMore trials in Kansas City
- Alliance for Multispecialty Research, LLC · Merriam, KS 66204More trials in Merriam
NY
- Roswell Park Comprehensive Cancer Center · Buffalo, NY 14263More trials in Buffalo
- Memorial Sloan Kettering Cancer Center-David H. Koch Center · New York, NY 10021More trials in New York
- Icahn School of Medicine at Mount Sinai · New York, NY 10029Lewis Silverman, MD · Study contact · 212-241-5520 · Lewis.silverman@mssm.eduMore trials in New York
TX
- University of Texas M. D. Anderson Cancer Center · Houston, TX 77030More trials in Houston