This is an open-label, Phase 1 study to determine the safety, tolerability, and efficacy of APL-4098 alone, and in combination with azacitidine, and in combination with azacitidine plus venetoclax for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
100
APL-4098 is administered orally in 28-day cycles
Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; APL-4098 is administered orally.
Azacitidine is administered at the standard dose on Day 1 - Day 7 of each 28-day cycle; Venetoclax is administered orally; APL-4098 is administered orally.
Monash Health
Clayton, Victoria, Australia
RECRUITINGSt. Vincent's Hospital Melbourne
Fitzroy, Victoria, Australia
RECRUITINGThe Alfred Hospital
Melbourne, Victoria, Australia
RECRUITINGHollywood Private Hospital
Nedlands, Western Australia, Australia
RECRUITINGRoyal Perth Hospital
Perth, Western Australia, Australia
RECRUITINGThe Royal Marsden Hospital
London, United Kingdom
RECRUITINGSarah Cannon Research Institute UK
London, United Kingdom
RECRUITINGIncidence of Treatment Emergent Adverse Events [Safety]
Evaluation of safety parameters including treatment emergent adverse events as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, Electrocardiogram results
Time frame: Through study completion, approximately one year
Incidence of Dose Limiting Toxicities [Tolerability]
Evaluation of tolerability parameters including dose limiting toxicities as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, and Electrocardiogram results
Time frame: Cycle 1 Day 1 to Cycle 2 Day 1 (a cycle is 28 days)
Determine Recommended Phase 2 Dose (RP2D)/Recommended dose range (RDR) levels of APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
Time frame: Approximately one year
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: Maximum plasma concentration (Cmax)
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: area under the curve (AUC)
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Assess the Pharmacokinetics of APL-4098
Evaluate PK parameters: Time to peak concentration (Tmax)
Time frame: On Days 1, 2, 8, and 15 of Cycle 1, and on Day 1 of Cycle 2 (each cycle is 28 days)
Assess response to disease with APL-4098 alone, in combination with azacitidine, and in combination with azacitidine plus venetoclax.
R/R AML and MDS/AML participants: response is assessed per European LeukemiaNet (ELN) 2022 criteria MDS-EB participants: response is assessed per revised International Working Group 2023 response criteria
Time frame: Response is assessed at Cycle 2 Day 1, Cycle 3 Day 1, Cycle 4 Day 1, Cycle 7 Day 1 (each cycle is 28 days long), then every three cycles thereafter (assessed for up to 2 years)
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