ENLIGHT-AML - An Open-label, Study of GB3226 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

NCT07084584 | Withdrawn Phase 1 DMC FDA Drug

• 2 other identifiers: GB3226-DEV-001ENLIGHT-AML
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The ENLIGHT-AML study is a Phase 1, open-label, dose-escalation and expansion study of GB3226 in the treatment of relapsed or refractory acute myeloid leukaemia

Conditions

Relapsed/Refractory Acute Myeloid Leukemia (AML)

Outcome Measures

Primary Outcomes (4)

• Occurrence of dose-limiting toxicities (Phase 1)

  Incidence of dose-limiting toxicities events identified

  28 Days

• To assess the Incidence of adverse and serious adverse events related to GB3226

  Incidence of adverse events as reported by investigators.

  28 Days

• To characterize the PK parameters of GB3226

  Plasma concentrations of GB3226

  28 Days

• To assess the complete remission and complete remission with partial hematologic recovery rate (Phase 2)

  Number of subjects in complete remission or complete remission with partial hematologic recovery

  28 Days

Study Arms (3)

Cohort A - GB3226 without CYP34A Inhibitor therapy

EXPERIMENTAL

Cohort A: Patients must not be receiving any weak, moderate, or strong CYP3A4 inhibitors/inducers. Patients who were receiving a CYP3A4 inhibitor/inducer must have discontinued the medication at least 7 days prior to enrolment

Drug: GB3226

Cohort B - GB3226 in combination with a strong CYP34A Inhibitor

EXPERIMENTAL

Cohort B: Patients must be receiving a strong CYP3A4 inhibitor for antifungal prophylaxis (e.g. parconazole, itraconazole, ketoconazole, or voriconazole) for at least 7 days prior to enrolment and while on GB3226 treatment. Patients must not be receiving any other strong CYP3A4 inhibitors/inducers.

Drug: GB3226

Cohort C - GB3226 in combination with a moderate CYP34A Inhibitor

EXPERIMENTAL

Cohort C: Patients must be receiving a moderate CYP3A4 inhibitor for antifungal prophylaxis (e.g., isavuconazole, fluconazole) for at least 7 days prior to enrolment and while on GB3226. Patients must not be receiving any other strong or moderate CYP3A4 inhibitors/inducers

Drug: GB3226

Interventions

GB3226 DRUG

GB3226: Dual inhibitor of ENL-YEATS and FLT3 pathways Administration: Oral, daily dosing in 28-day cycles

Cohort A - GB3226 without CYP34A Inhibitor therapy; Cohort B - GB3226 in combination with a strong CYP34A Inhibitor; Cohort C - GB3226 in combination with a moderate CYP34A Inhibitor

Eligibility Criteria

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

You may qualify if:

• Male or female patients aged ≥18 years of age
• Eastern Cooperative Oncology Group (ECOG) performance status score 0-2
• Adequate cardiac function defined as ejection fraction (EF) of ≥45% by echocardiogram or multigated acquisition (MUGA) scan.
• Prior Therapy:
• Prior treatment-related toxicities must have resolved to ≤Grade 1 before enrolment, except for ≤Grade 2 neuropathy or alopecia.
• Radiation therapy: ≥60 days since TBI, craniospinal, or ≥50% pelvic radiation; ≥14 days since local palliative (small port) radiation.
• Stem cell infusion: ≥90 days since HSCT and ≥4 weeks since DLI.
• Immunotherapy: ≥30 days since prior immunotherapy (including tumor vaccines) and CAR T-cell or other modified T/NK cell therapy, allowing for a first response evaluation.
• Antileukemia therapy: ≥14 days or 5 half-lives (whichever is shorter) since last antileukemia therapy (e.g. small molecule, cytotoxic, or myelosuppressive therapy), unless otherwise specified. Hydroxyurea for cytoreduction can be administered in Cycle 1 if warranted.
• Patients may receive intrathecal chemotherapy at the time of diagnostic lumbar puncture at least 24 hours prior to the start of GB3226 and may continue prophylactic intrathecal chemotherapy beginning in Cycle 2 at the treating physician's discretion.
• Hematopoietic growth factors: ≥7 days since short-acting and ≥14 days since long-acting growth factor therapy.
• Biologics: ≥90 days or 5 half-lives (whichever is shorter) since antineoplastic biologic therapy.
• Steroids: ≥7 days since systemic glucocorticoids, except for physiologic doses (≤10 mg prednisone daily)
• Adequate kidney and liver function as evidenced by GFR ≥ 60 mL/min, total bilirubin ≤ 2 times ULN (unless due to Gilbert's syndrome), ALT/AST ≤ 3 times ULN.
• Contraception

You may not qualify if:

• Patients meeting any of the following criteria are NOT eligible for study participation:
• Diagnosis
• Diagnosis of active acute promyelocytic leukemia. (APML).
• Diagnosis of chronic myelogenous leukemia (CML) in blast crisis.
• Detectable human immunodeficiency virus (HIV) viral load within the previous 6 months. Patients with a known history of HIV 1/2 antibodies must have viral load testing prior to study enrolment.
• Hepatitis B (defined as hepatitis B virus \[HBV\] surface antigen positive and HBV core antibody positive, or positive HBV deoxyribonucleic acid \[DNA\]).
• Hepatitis C (defined as positive hepatitis C \[HCV\] antibody with reflex to positive HCV ribonucleic acid \[RNA\]).
• NB: Patients with controlled HIV, Hep B and Hep C disease will not be excluded from study enrolment.
• Pregnancy and Breast-Feeding
• Pregnant or nursing women. Negative serum pregnancy tests are required during Screening and a negative serum or urine pregnancy test is required within 72 hours prior to receiving the first study drug administration, in females of childbearing potential. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
• Concurrent Conditions
• Cardiac Disease:
• Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class ≥III), life-threatening, uncontrolled hypertension or arrhythmia, ischemic or severe valvular heart disease, cerebrovascular accident, or transient ischemic attack.
• Mean QTcF ≥470 ms on triplicate ECG. Appropriate corrections for patients with bundle branch block and ventricular paced rythms are allowed.
• Gastrointestinal Disease:

Sponsors & Collaborators

• Galecto Biotech AB: lead

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

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

Study Design

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

Study Record Dates

• First Submitted: July 17, 2025
• First Posted: July 24, 2025
• Study Start: June 10, 2026
• Primary Completion (Estimated): June 10, 2029
• Study Completion (Estimated): October 10, 2029
• Last Updated: July 6, 2026

Record last verified: 2025-07