Phase I, open-label study to assess the safety, feasibility, pharmacokinetics, and preliminary efficacy of CART123 cells given in combination with ruxolitinib in patients with relapsed or refractory acute myeloid leukemia (AML). All subjects will receive a single infusion of CART123 cells following ruxolitinib administration and lymphodepletion. Ruxolitinib dosing will begin at initiation of lymphodepleting chemotherapy (Day -6 ±1d) and continue for up to 14 days post CART123 administration.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Evaluate the safety of CART123 cells when given in combination with ruxolitinib
Type, frequency, severity, and attribution of AEs/SAEs, including the incidence and severity of IEC-associated adverse events.
Time frame: 15 Years
Evaluate the safety of CART123 cells when given in combination with ruxolitinib
Occurrence of treatment-limiting toxicities (TLTs)
Time frame: 3 months
Evaluate the safety of CART123 cells when given in combination with ruxolitinib
The proportion of subjects requiring ruxolitinib dose modifications by assigned dose.
Time frame: 3 months
Evaluate study feasibility
The proportion of eligible subjects who receive both investigational products as per protocol.
Time frame: 3 months
Evaluate study feasibility
The proportion of eligible subjects who receive all planned doses of ruxolitinib.
Time frame: 3 months
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
Objective Response Rate (ORR) at Day 28
Time frame: From enrollment to Day 28, post treatment.
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
Duration of Response (DOR)
Time frame: 15 years
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
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Progression-free survival (PFS)
Time frame: 15 Years
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
Overall Survival (OS)
Time frame: 15 Years
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
Reduction of blast count in the peripheral blood and marrow using standard clinical criteria (CBC with differential count, marrow aspirate with differential count) and flow cytometry at Day 28
Time frame: From enrollment to Day 28, post treatment.
Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib
Percentage of subjects undergoing alloHCT
Time frame: 15 Years