Copanlisib and Ibrutinib in Treating Patients with Recurrent or Refractory Primary Central Nervous System Lymphoma
This phase Ib/II trial studies the side effects and best dose of ibrutinib when given together with copanlisib, and to see how well they work in treating patients with primary central nervous system lymphoma that has come back (recurrent) or does not respond to treatment (refractory). Copanlisib and ibrutinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Inclusion Criteria
- Men and woman who are at least 18 years of age on the day of consenting to the study
- Histologically documented PCNSL
- Relapsed/refractory PCNSL patients or newly diagnosed PCNSL patients who are deemed medically ineligible by the treating investigator (phase II only) to receive standard first-line chemotherapy. All recurrent/refractory patients need to have received at least one prior central nervous system (CNS) directed therapy. There is no restriction on the number of recurrences
- For recurrent/refractory patients, parenchymal lesions must have unequivocal evidence of disease progression on imaging (MRI of the brain or head computed tomography [CT]) 21 days of study registration. For patients with leptomeningeal disease only, cerebrospinal fluid (CSF) cytology must document lymphoma cells and/or imaging findings consistent with CSF disease 21 days of study registration (at the discretion of the investigator)
- Eastern Cooperative Oncology Group (ECOG) performance status =< 2
- Life expectancy of > 3 months (in the opinion of the investigator)
- Absolute neutrophil count (ANC) >= 1.5 x 10^9/L
- Platelets >= 75 x 10^9/L and no platelet transfusion within the past 14 days prior to study registration
- Hemoglobin (Hgb) >= 8 g/dL and no red blood cell (RBC) transfusion within the past 14 days prior to study registration
- International normalized ratio (INR) =< 1.5 and partial thromboplastin time (PTT)(activated partial thromboplastin time [aPTT]) =< 1.5 times the upper limit of normal
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 3 times the upper limit of normal
- Serum bilirubin =< 1.5 times the upper limit of normal; or total bilirubin =< 3 times the upper limit of normal with direct bilirubin within the normal range in patients with well documented Gilbert syndrome
- Serum creatinine =< 2 times the upper limit of normal
- Creatinine clearance >= 30 mL/min
- Lipase =< 1.5 x upper limit of normal
- Women of childbearing potential (WOCBP) and men must agree to use effective contraception when sexually active. This applies for the time period between signing of the informed consent form and 30 days (for WOCBP) and 90 days (for men) after the last administration of study treatment. A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include but are not limited to hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for continuous 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. * The investigator or a designated associate is requested to advise the patient how to achieve highly effective birth control (failure rate of less than 1%), e.g. intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner and sexual abstinence * The use of condoms by male patients is required unless the female partner is permanently sterile. Female subjects of childbearing potential must have a negative plasma pregnancy test upon study entry
- Must be able to tolerate MRI/CT scans
- Must be able to tolerate lumbar puncture and/or Ommaya taps
- Must have recovered to grade 1 toxicity from prior therapy
- Able to submit up to 20 unstained formalin-fixed, paraffin-embedded (FFPE) slides from the initial or most recent tissue diagnosis for correlative studies
- NOTE: Prior autologous stem cell transplant as well as prior radiation to the CNS does NOT prevent patients from enrollment into the trial
Exclusion Criteria
- Active concurrent malignancy requiring active therapy
- Newly diagnosed PCNSL who qualify for standard methotrexate-based chemotherapy
- Clinically significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure (New York Heart Association > class 2), unstable angina, or myocardial infarction within 6 months of screening, or any class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification
- Uncontrolled hypertension despite optimal medical management (per investigator's assessment)
- Patient has poorly controlled diabetes mellitus with a glycosylated hemoglobin > 8% or poorly controlled steroid-induced diabetes mellitus with a glycosylated hemoglobin of > 8%
- Patient is known to have an uncontrolled active systemic infection (> Common Terminology Criteria for Adverse Events [CTCAE] grade 2) and recent infection requiring intravenous anti-infective treatment that was completed =< 14 days before the first dose of study drug
- Arterial or venous thrombotic or embolic events such as cerebrovascular accident, deep vein thrombosis or pulmonary embolism within 3 months before the start of study treatment
- Non-healing wound, ulcer or bone fracture
- Not recovered to a grade 1 from the toxic effects of prior therapy if clinically relevant in the opinion of the investigator (e.g. alopecia)
- Known bleeding diathesis (e.g., von Willebrand's disease) or hemophilia
- Known history of infection with human immunodeficiency virus (HIV) or history of active or chronic infection with hepatitis C virus (HCV) or hepatitis B virus (HBV) as determined by serologic tests, or any uncontrolled active systemic infection
- Patient underwent major systemic surgery =< 2 weeks prior to starting the trial treatment or who has not recovered from the side effects of such surgery, or who plan to have surgery within 2 weeks of the first dose of the study drug
- Unable to swallow capsules or disease significantly affecting gastrointestinal function, such as malabsorption syndrome, resection of the stomach or small bowel, or complete bowel obstruction
- Any life-threatening illness, medical condition including uncontrolled diabetes mellitus (DM), uncontrolled hypertension or organ system dysfunction that, in the opinion of the investigator, could compromise the subject's safety or put the study outcomes at undue risk
- Lactating or pregnant
- Any chemotherapy, external beam radiation therapy, or anticancer antibodies within 21 days of the first dose of study drug
- Prior treatment with a PI3K inhibitor, AKT inhibitor, or mTOR inhibitor (prior ibrutinib exposure is allowed)
- Any targeted anticancer therapy =< 4 weeks or 5 half-lives, whichever is shorter
- Use of radio- or toxin-immunoconjugates within 70 days of the first dose of study drug
- Concurrent use of warfarin or other vitamin K antagonists (need to be stopped 7 days prior to starting on trial drug)
- Concurrent use of a strong cytochrome P450 (CYP) 3A4/5 inhibitor and inducers (need to be stopped 2 weeks prior to starting on trial drug)
- Enzyme-inducing antiepileptic drugs (EIAED) need to be discontinued and switched to a non-EIAED 2 weeks prior to starting on trial drug)
- Patient requires more than 4 mg of dexamethasone daily or the equivalent
- Patient is using systemic immunosuppressant therapy, including cyclosporine A, tacrolimus, sirolimus, and other such medications, or chronic administration of > 5 mg/day or prednisone or the equivalent. Participants must be off of immunosuppressant therapy for at least 28 days prior to the first dose of the study drug
- Concurrent systemic immunosuppressant therapy (e.g., cyclosporine A, tacrolimus, etc., or chronic administration of > 5 mg/day of prednisone) within 28 days of the first dose of study drug
- Prior allogeneic stem cell transplant
Additional locations may be listed on ClinicalTrials.gov for NCT03581942.
See trial information on ClinicalTrials.gov for a list of participating sites.
PRIMARY OBJECTIVES:
I. To define the maximally tolerated dose (MTD) of copanlisib hydrochloride (copanlisib) in combination with ibrutinib in refractory/relapsed primary central nervous system lymphoma (PCNSL). (phase Ib)
II. To explore the therapeutic efficacy measured by overall response rate (ORR) of copanlisib in combination with ibrutinib in patients with refractory/relapsed PCNSL or newly diagnosed PCNSL not able to tolerate standard chemotherapy. (phase II)
SECONDARY OBJECTIVES:
I. To determine the safety and tolerability of copanlisib in combination with ibrutinib in the study population. (phase Ib and II)
II. To assess progression free survival (PFS). (phase II)
III. To assess duration of response (DOR). (phase II)
IV. To assess overall survival (OS). (phase II)
V. To evaluate cerebral spinal fluid (CSF) pharmacokinetics of copanlisib and ibrutinib and correlate with plasma pharmacokinetics. (phase Ib)
VI. To correlate differences in drug response based on CD79b, MYD88, TNFAIP3, and CARD11 mutational status through targeted mutational analysis. (phase Ib and II)
VII. To correlate differences in drug response based on immunohistochemical status of phospho-S6 and p-4EBP1. (phase Ib and II)
VIII. To correlate apparent diffusion coefficient/diffusion weighted imaging (ADC/DWI) magnetic resonance imaging (MRI) to clinical response. (phase II)
OUTLINE: This is a dose-escalation study of copanlisib and ibrutinib followed by a phase II study.
Patients are treated in 28-day cycles, and only one drug is administered at a time with a ibrutinib/copanlisib ratio of 1:2. Patients are given ibrutinib orally (PO) once daily (QD) for 28 days during cycle 1, and then patients receive copanlisib hydrochloride intravenously (IV) over 1 hour on days 1, 8, and 15 during cycles 2 and 3. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo computed tomography (CT) and may undergo magnetic resonance imaging (MRI), blood and cerebrospinal fluid (CSF) sample collection throughout the study.
After completion of study treatment, patients are followed up at 30 days.
Trial PhasePhase I/II
Trial Typetreatment
Lead OrganizationMemorial Sloan Kettering Cancer Center
Principal InvestigatorChristian Grommes
- Primary ID18-246
- Secondary IDsNCI-2018-02477
- ClinicalTrials.gov IDNCT03581942