Evaluating GLB-001 for patients with myeloid malignancies

A Phase 1, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of GLB-001 in Patients With Myeloid Malignancies

This study is testing a new oral treatment called GLB-001 to see if it is safe and effective for people with certain types of blood cancers that have come back or didn't respond to other treatments.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 108 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Hangzhou GluBio Pharmaceutical Co., Ltd. (Industry-sponsored)
• Drugs / interventions: CAR-T, chimeric antigen receptor, chemotherapy, immunotherapy
• Locations: 14 sites (Hefei, Anhui and 13 other locations)
• Trial ID: NCT06378437 on ClinicalTrials.gov

What this trial studies

This phase 1 clinical study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of GLB-001 in patients with relapsed or refractory myeloid malignancies, including polycythemia vera, essential thrombocythemia, myelofibrosis, myelodysplastic syndromes, and acute myeloid leukemia. The study is divided into three parts: dose escalation, dose exploration, and dose expansion, where participants will receive GLB-001 orally. The goal is to determine the relationship between dose, exposure, toxicity, tolerability, and clinical activity of the treatment.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with confirmed relapsed or refractory myeloid malignancies.

Not a fit: Patients with non-relapsed myeloid malignancies or those with severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat myeloid malignancies.

How similar studies have performed: While this approach is novel, similar studies targeting myeloid malignancies have shown promise in the past.

Eligibility criteria

Inclusion Criteria:

* Study participants must understand and voluntarily sign a written informed consent form (ICF) prior to any study-related assessments/procedures being performed.
* Study participants is ≥18 years of age at the time of signing the ICF.
* Study participants with confirmed diagnosis of relapsed or refractory or intolerant myeloid malignancies including PV, ET, primary myelofibrosis (PMF), MDS and AML according to 2022 World Health Organization (WHO) criteria classification, and post-polycythemia vera myelofibrosis (post-PV MF) and post-essential thrombocythemia myelofibrosis (post-ET MF) according to the 2013 IWG-MRT criteria.
* Eastern Cooperative Oncology Group (ECOG) performance status of 0,1 or 2.
* Life expectancy \> 3 months.
* Good performance of major organs, including hematology, liver and kidney function, and coagulation. etc.
* Study participants are willing and able to adhere to the study visit schedule and other protocol requirements.

Exclusion Criteria:

* Study participants with acute promyelocytic leukemia (APL).
* Receipt of following anticancer medications/therapies prior to the first dose of GLB-001: (1) study participants with PV or ET who received treatment with hydroxyurea within 2 days prior to the first dose, or any other treatment for PV or ET within 7 days prior to first dose of GLB-001, (2) study participants with MF who received any type of treatment for MF within 14 days prior to the first dose, such as chemotherapy, immunotherapy, radiotherapy and erythropoietin, androgens, thrombopoietin or granulocyte colony-stimulating factor, (3) study participants with LR-MDS who received any type of treatment for MDS within 14 days prior to the first dose, (4) study participants with HR-MDS or AML who received chimeric antigen receptor T cell therapy (CAR-T) or other biologic therapy within 28 days prior to the first dose of GLB-001, or received any other anticancer therapies within 14 days prior to the first dose of GLB-001.
* Receipt of any other investigational drug study within 28 days or 5 half-lives of that study drug before the first dose of GLB-001.
* Study participants with unresolved clinically significant non-hematologic toxicities that were ≥ Grade 1 or failed to recover to baseline levels following prior anticancer therapies (with the exception of alopecia or skin hyperpigmentation).
* Study participants who are scheduled to receive other anticancer therapies or other investigational drugs during the study period.
* Study participants with active acute or chronic graft versus host disease (GVHD) requiring systemic immunosuppressive therapy.
* Receipt of autologous stem cell transplantation (ASCT) within the last 3 months prior to the first dose of GLB-001, or allogeneic hematopoietic stem cell transplantation (allo-HSCT) within the last 6 months prior to the first dose of GLB-001.
* Study participants with known active involvement in central nervous system (CNS).
* Study participants with peripheral neuropathy ≥ Grade 2 (Graded according to CTCAE version 5.0).
* Study participants have a history of known malignancy other than the inclusion diagnosis for the past 5 years, with the exception of curatively resected cancer in situ, including cervical carcinoma in situ, basal cell carcinoma of the skin, or prostate cancer in situ, etc.
* QT interval interval \> 450 milliseconds (ms) using electrocardiographic (ECG) at screening.
* Study participants have impaired cardiac function or clinically significant cardiac disease at current or within last 6 months.
* Study participants with known active infection of hepatitis B virus (HBV) or hepatitis C virus C (HCV).
* Study participants with known human immunodeficiency virus (HIV) infection.
* Study participants with known life-threatening or clinical significant uncontrolled active systemic infections unrelated to malignant hematologic diseases.
* Study participants with a state condition that may alter affects the absorption, distribution, metabolism and excretion of GLB-001 after judgment of the investigator.
* Medications or supplements that are known to be strong and moderate inhibitors or inducers of cytochrome P-450 isozyme 3A (CYP3A) and/or P-glycoprotein (P-gp), or strong inhibitors or inducers of CYP450 isozyme 2C8 (CYP2C8) within 7 days or 5 half-lives prior to the first dose of GLB-001, whichever is shorter prior to the first dose of GLB-001.
* Study participants who have undergone major surgery within 28 days prior to the first dose of the GLB-001, or unability to recover from effects of surgery.
* Pregnant or lactating women.
* Study participants who have cognitive impairment due to any psychiatric or neurological condition, including epilepsy and dementia, may limit their understanding, performance, and study compliance with the ICF.
* Study participants, in the opinion of the Investigator, who are unsuitable to participate in the study.

Where this trial is running

Hefei, Anhui and 13 other locations

• The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital) — Hefei, Anhui, China (Recruiting)
• China-Japan Friendship Hospital — Beijing, Beijing Municipality, China (Recruiting)
• The First Affiliated Hospital of Chongqing Medical University — Chongqing, Chongqing Municipality, China (Recruiting)
• The First Hospital of Hebei Medical Universtiy — Shijiazhuang, Hebei, China (Recruiting)
• Henan Cancer Hospital — Zhengzhou, Henan, China (Recruiting)
• Zhongnan Hospital of Wuhan University — Wuhan, Hubei, China (Recruiting)
• The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)
• The First Affiliated Hospital of Nanchang University — Nanchang, Jiangxi, China (Recruiting)
• Sheng Jing Hospital of China Medical Universtiy — Shenyang, Liaoning, China (Recruiting)
• Huashan Hospital Affiliated to Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
• Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin Municipality, China (Recruiting)
• The Second Hospital of Tianjin Medical Universtiy — Tianjin, Tianjin Municipality, China (Recruiting)
• The First Affilicated Hospital, Zhejiang University School of Medicine — Hangzhou, Zhejiang, China (Recruiting)
• The First Affiliated Hospital of Wenzhou Medical University — Wenzhou, Zhejiang, China (Recruiting)

Study contacts

• Study coordinator: Jing Liu, Ph.D.

Conditions Polycythemia Vera; Essential Thrombocythemia; Myelofibrosis; Myelodysplastic Syndromes; Acute Myeloid Leukemia; Myeloid Malignancy