Study of the Safety, Tolerability and Efficacy of KPT-8602 in Participants With Relapsed/Refractory Cancer Indications

Official Title

A Phase 1/2 Open-Label Study of the Safety, Tolerability and Efficacy of the Selective Inhibitor of Nuclear Export (SINE) Compound Eltanexor (KPT-8602) in Patients With Newly Diagnosed and Relapsed/Refractory Cancer Indications

Summary:

This is a first-in-human, multi-centre, open-label clinical study with separate dose escalation (Phase 1) and expansion (Phase 2) stages to assess preliminary safety, tolerability, and efficacy of the second generation oral XPO1 inhibitor KPT-8602 in participants with relapsed/refractory multiple myeloma (MM), metastatic colorectal cancer (mCRC), metastatic castration resistant prostate cancer (mCRPC), higher risk myelodysplastic syndrome (HRMDS), Acute Myeloid Leukemia (AML) and Newly Diagnosed Intermediate/High-Risk MDS. Dose escalation and dose expansion may be included for all parts of the study as determined by ongoing study results.

Trial Description

Primary Outcome:

• Part A1, A2, B, C, D, E, F: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
• Part A1, A2, B, C, D, E, F: Overall Response Rate (ORR)
• Part A1, A2, B, C, D, E, F: Duration of Response (DOR)
• Part A1, A2, B, C, D, E, F: Progression-free survival (PFS)
• Part A1, A2, B, C, D, E, F: Overall Survival (OS)
• Part A1, A2, B, C, D, E, F: Clinical Benefit Rate (CBR)
• Part A1, A2, B, C, D, E, F: Duration of Clinical Benefit Rate (CBR)
• Part A1, A2, B, C, D, E, F: Disease control rate (DCR)
• Part A1, A2, B, C, D, E, F: Duration of DCR
• Part F Phase 2: ORR
• Part G: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
• Part H: 2- Year Progression-free Survival (PFS)

• Part A1, A2, B, C, D, E, F, H: Area Under the Plasma Concentration Time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUC0-t) of Eltanexor
• Part A1, A2, B, C, D, E, F, H: Maximum Observed Plasma Concentration (Cmax) of Eltanexor
• Part A1, A2, B, C, D, E, F, H: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Eltanexor
• Part A1, A2, B, C, D, E, F, H: Apparent Terminal Half Life (t1/2) of Eltanexor
• Part A1, A2, B, C, D, E, F, H: Apparent Total Body Clearance Eltanexor
• Part A1, A2, B, C, D, E, F, H: Apparent Volume of Distribution During Terminal Phase (VZ/f) of Eltanexor
• Part F Phase 2: Overall Survival (OS)
• Part F Phase 2: 6-Month Overall Survival (OS)
• Part F Phase 2: Progression-free survival (PFS)
• Part F Phase 2: Disease control rate (DCR)
• Part F Phase 2: Duration of Response (DOR)
• Part F Phase 2: Rate of Conversion from Red Blood Cell (RBC) Transfusion Dependence to Independence
• Part F Phase 2: Rate of Conversion from Platelet Transfusion Dependence to Independence
• Part G: Overall Response Rate (ORR)
• Part G: Duration of Response (DOR)
• Part H: Rate of Minimal Residual Disease (MRD) Conversion from Positive to Negative
• Part H: Time to Minimal Residual Disease (MRD) Negativity
• Part H: Percentage of Participants with Acute and Chronic Graft-versus-Host Disease (GVHD)
• Part H: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
• Part H: Overall Survival (OS)

View this trial on ClinicalTrials.gov