This research study will test the safety and anticancer activity of the combination of three drugs (Roginolisib, Venetoclax, and Rituximab) for participants with relapsed or refractory Chronic Lymphocytic Leukemia (CLL). The names of the study drugs involved in this study are: * Roginolisib (a novel type of PI3-kinase delta inhibitor) * Venetoclax (a type of B-cell lymphoma 2 inhibitor) * Rituximab (a type of monoclonal antibody)
This is a Phase I/II, open-label, randomized, and comparator controlled study to test the safety and anticancer activity of the combination of three drugs (Roginolisib, Venetoclax, and Rituximab) for participants with relapsed or refractory Chronic Lymphocytic Leukemia (CLL). In this study, investigators are assessing whether adding the study drug, Roginolisib, will lead to deeper responses than the standard combination of Venetoclax and Rituximab alone. A phase Ib safety lead-in portion of the trial will determine the dose and tolerability of Roginolisib with the standard 2-drug combination treatment of Venetoclax + Rituximab. In the phase 2 portion of the trial, participants will be randomized into one of two treatment arms: Arm A: Roginolisib + Venetoclax and Rituximab vs. Arm B: Venetoclax and Rituximab. Randomization means a participant is placed into a treatment arm by chance. The U.S. Food and Drug Administration (FDA) has not approved Roginolisib as a treatment for relapsed or refractory CLL. The U.S. FDA has approved Venetoclax and Rituximab as a standard 2-drug combination treatment for relapsed or refractory CLL. The research study procedures include screening for eligibility in-clinic visits, blood tests, urine tests, electrocardiograms (ECGs), Computerized Tomography (CT) scans, Magnetic Resonance Imaging (MRI) scans, and bone marrow biopsy and/or aspirations. Participants will receive study treatment for approximately 12 months and will be followed for up to 5 years. It is expected about 3-12 people will take part in the Phase 1 portion of the trial, and 52 people will participate in the Phase 2 portion of the research study. iOnctura SA is supporting this research study by providing the study drug, Roginolisib. The U.S. Department of Defense (DOD) is supporting this research study by providing funding.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
64
Phosphoinositide 3-kinase delta inhibitor, 40 mg capsule, taken orally per protocol.
B-cell lymphoma 2 inhibitor, 10, 50, and 100mg tablets, taken orally per standard of care.
Chimeric anti-CD 20 monoclonal antibody, single-use 10 and 50mL vials, via intravenous (into the vein) infusion per standard of care.
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGUndetectable Minimal Residual Disease(uMRD) rate by ClonoSEQ assay
uMRD rate is defined as the proportion of participants with uMRD after treatment with either roginolisib plus venetoclax + rituximab, or venetoclax+ rituximab, as measured in bone marrow at a level of 10-4 by the clonoSEQ assay.
Time frame: 1 year
Adverse Event of Interest Rate
Adverse event of interest rate is defined as the percentage of participants who experienced an adverse event including transaminitis, rash, diarrhea, colitis and pneumonitis as well as febrile neutropenia and infection. Assessment will be based on NCI CTCAE grading for non-hematologic toxicity, and iwCLL criteria for hematologic toxicity.
Time frame: 1 year
Undetectable Minimal Residual Disease(uMRD) rate by flow cytometry
uMRD as measured in blood at same timepoint as ClonoSeq.
Time frame: 5 years
Median Progression Free Survival(PFS)
PFS based on Kaplan Meier method is defined as the duration of time from study entry to documented disease progression (PD) requiring removal from the study or death. Participants alive without PD were censored at the earliest of the date of the last disease evaluation.
Time frame: 5 years
Median Overall Survival (OS)
Overall survival based on the Kaplan-Meier method is defined as the time from randomization to death. Participants alive are censored at the last date of contact (including lost-to-follow-up) or at the date of withdrawal of consent, if relevant.
Time frame: 5 years
Overall Response Rate (ORR)
ORR was defined as the percentage of participants achieving complete response (CR) or partial response (PR) on treatment based on iwCLL criteria.
Time frame: 1 year
Duration of Response (DOR)
DOR is defined as the time from date of first documented confirmed objective response to date of first documented progressive disease (PD).
Time frame: 5 years
Complete Response Rate (CRR)
CRR was defined as the percentage of participants achieving complete response (CR) on treatment based on iwCLL criteria.
Time frame: 1 year
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