This is a multi-center, open-label study to evaluate the safety, pharmacokinetics (PK), and anti-cancer activity of oral administration of emavusertib alone or in combination with ibrutinib in adult participants with relapsed or refractory (R/R) hematologic malignancies. This trial will be completed in four parts. In Part A1, emavusertib will be evaluated first in a dose escalating monotherapy setting to establish the safety and tolerability (complete). In Part A2, emavusertib will be evaluated in combination with ibrutinib at 560 milligrams (mg) once daily (QD) or 420 mg QD as indicated by disease (Part A2 complete). Part B will comprise 2 cohorts to assess safety and efficacy of emavusertib in combination with ibrutinib in participants with R/R primary central nervous system lymphoma (PCNSL) who have directly progressed on a bruton tyrosine kinase inhibitor (BTKi). In this part of the study, emavusertib will be dosed at 100 mg or 200 mg twice daily (BID) in combination with ibrutinib in 28-day treatment cycles. Part C will comprise 3 treatment arms in the second-line setting to assess the efficacy and safety of emavusertib monotherapy, ibrutinib monotherapy, and emavusertib in combination with ibrutinib in participants with R/R PCNSL who are naïve to BTKi treatment. In this part of the study, eligible second-line participants with R/R PCNSL who are naïve to BTKi treatment will be randomized 1:1:1 to 1 of 3 treatment arms: (1) emavusertib 200 mg BID, (2) ibrutinib 560 mg QD, or (3) emavusertib 200 mg BID in combination with ibrutinib 560 mg QD.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
152
Emavusertib will be provided as a tablet dosage form to be taken BID.
Ibrutinib will be provided as a tablet or capsule dosage form to be taken QD.
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States
RECRUITINGMayo Clinic
Phoenix, Arizona, United States
COMPLETEDCity of Hope
Duarte, California, United States
RECRUITINGProvidence St. John's Health Center
Santa Monica, California, United States
Part A: To determine the safety and tolerability of emavusertib as a monotherapy and in combination with ibrutinib: dose-limiting toxicity (DLT)
The number of participants with a dose-limiting toxicity (DLT) in the first treatment cycle
Time frame: 12 months
Part A: Maximum tolerated dose (MTD) of emavusertib as a monotherapy and in combination with ibrutinib measured by dose-limiting toxicities (DLTs)
MTD determined by the highest dose level studied at which fewer than 2 out of 6 subjects (\<33%) experience a dose limiting toxicity.
Time frame: 12 months
Part A: Recommended Phase 2 Dose (RP2D) of emavusertib as a monotherapy and in combination with ibrutinib based on overall tolerability data
RP2D selected based on overall tolerability data from all participants treated at different dose levels and will not exceed the MTD.
Time frame: 12 months
Part B: Overall Response Rate (ORR) in participants with R/R PCNSL
Time frame: 18 months
Part C: ORR in participants with R/R PCNSL
Time frame: 18 months
Parts A, B and C: Pharmacokinetic (PK) profile of emavusertib and ibrutinib measured by AUC
Area Under the concentration-time curve (AUC)
Time frame: 24- 66 months
Parts A, B and C: Pharmacokinetic (PK) profile of emavusertib and ibrutinib measured by Cmax
Maximum plasma concentration (Cmax)
Time frame: 24- 66 months
Parts A, B and C: Pharmacokinetic (PK) profile of emavusertib and ibrutinib measured by Cmin
Minimum plasma concentration (Cmin)
Time frame: 24- 66 months
Parts A, B and C: Pharmacokinetic (PK) profile of emavusertib and ibrutinib measured by Tmax
Time to maximum plasma concentration (Tmax)
Time frame: 24- 66 months
Parts A, B and C: Pharmacokinetic (PK) profile of emavusertib and ibrutinib measured by plasma terminal half-life
Plasma terminal elimination half-life (T 1/2)
Time frame: 24- 66 months
Part A: To assess efficacy of emavusertib as a monotherapy and in combination with ibrutinib measured by ORR
Assessed by ORR
Time frame: 24- 36 months
Parts A, B and C: To assess efficacy of emavusertib as a monotherapy and in combination with ibrutinib and ibrutinib as monotherapy measured by duration of response (DOR)
Assessed by DOR
Time frame: 24- 66 months
Part A: To assess efficacy of emavusertib as a monotherapy and in combination with ibrutinib measured by disease control rate (DCR)
Assessed by DCR
Time frame: 24- 36 months
Parts A, B and C: To assess efficacy of emavusertib as a monotherapy and in combination with ibrutinib and ibrutinib as monotherapy measured by progression free survival (PFS)
Assessed by PFS
Time frame: 24- 66 months
Parts A, B and C: To assess efficacy of emavusertib as a monotherapy, in combination with ibrutinib and ibrutinib as monotherapy measured by overall survival (OS)
Assessed by OS
Time frame: 24 - 66 months
Parts B and C: To assess the safety and tolerability of emavusertib as monotherapy, ibrutinib as monotherapy and emavusertib in combination with ibrutinib in participants with R/R PCNSL
Measured by the number of participants with treatment-emergent adverse events (TEAEs) and treatment-related adverse events
Time frame: up to 66 months
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UCLA Department of Medicine - Hematology/Oncology
Santa Monica, California, United States
WITHDRAWNSmilow Cancer Hospital at Yale-New Haven
New Haven, Connecticut, United States
COMPLETEDMayo Clinic
Jacksonville, Florida, United States
RECRUITINGNorthwestern Memorial Hospital
Chicago, Illinois, United States
RECRUITINGDana Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITING...and 35 more locations