Retifanlimab With or Without Difluoromethylornithine for the Treatment of Progressive High Grade Gliomas
This phase I/II trial tests the safety, side effects best dose and effect of retifanlimab with or without difluoromethylornithine (DFMO) for the treatment of high grade gliomas that are growing, spreading, or getting worse (progressive). Immunotherapy with monoclonal antibodies, such as retifanlimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. DFMO is in a class of medications called ornithine decarboxylase (ODC) inhibitors. It works by blocking the action of a substance that signals tumor cells to multiply. This helps stop or slow the spread of tumor cells. Giving retifanlimab with or without DFMO mat be safe, tolerable and/or effective in treating patients with progressive high grade glioma.
Inclusion Criteria
- Age ≥ 18 years
- Diagnosis of high-grade glioma, including any of the following: * Glioblastoma, IDH-wild type (WT) * Grade 3 or 4 IDH1/2 mutant astrocytoma or * Grade 3 oligodendroglioma * Any prior grade 2 astrocytoma or oligodendroglioma that is suspected to have recurred at a higher grade * Other high-grade glioma
- Plan for surgical resection as part of routine clinical care
- Radiographic disease progression, with or without tissue confirmation
- Measurable disease
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1 or 2 and Karnofsky Performance Status (KPS) ≥ 60 * NOTE: PS must be assessed (again) within 7 days prior to first dose of study drug
- Hemoglobin ≥ 9.0 g/dL (obtained ≤ 15 days prior to registration)
- Absolute neutrophil count (ANC) ≥ 1500/mm^3 (obtained ≤ 15 days prior to registration)
- Platelet count ≥ 100,000/mm^3 (obtained ≤ 15 days prior to registration)
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (obtained ≤ 15 days prior to registration)
- Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤ 3 x ULN (≤ 5 x ULN for patients with liver involvement) (obtained ≤ 15 days prior to registration)
- Calculated creatinine clearance ≥ 45 ml/min using the Cockcroft-Gault formula (obtained ≤ 15 days prior to registration)
- Negative pregnancy test done ≤ 7 days prior to registration, for persons of childbearing potential only
- Provide written informed consent for the current study
- Willing to provide consent for the Neuro-oncology biorepository (IRB 12-003458) for archiving of tissue, cerebrospinal fluid (CSF), and/or blood samples
- Ability to complete forms by themselves or with assistance
- Willing to return to enrolling institution for follow-up (during the Active Monitoring Phase of the study)
Exclusion Criteria
- Any of the following because this study involves an investigational agent, the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown: * Pregnant persons * Nursing persons * Persons of childbearing potential or able to father a child who are unwilling to employ adequate contraception
- Uncontrolled intercurrent illness that by the judgement of the investigator would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the regimens including, but not limited to: * ongoing or active infection (e.g., pneumonia, sepsis, etc.) requiring systemic therapy * current diagnosis or previous history of immune-related (non-infectious) pneumonitis or interstitial lung disease that requires or required steroids * active autoimmune disease that required systemic treatment other than replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroids) ≤ 2 years prior to registration * symptomatic congestive heart failure * unstable angina pectoris * psychiatric illness/social situations that would limit compliance with study requirements (e.g., drug addiction) * concurrent active Hepatitis B (defined as hepatitis B surface antigen [HBsAg] positive and/or detectable hepatitis B virus [HBV] deoxyribonucleic acid [DNA]) and Hepatitis C virus (defined as anti-hepatitis C virus [HCV] antibody [Ab] positive and detectable HCV ribonucleic acid [RNA]) infection EXCEPTIONS: * Patients with evidence of hepatitis B virus (HBV) infection (HBsAg positive) must have completed at least 4 weeks of HBV antiviral therapy, and the HBV viral load must be undetectable at the time of registration * Patients with a history of hepatitis C virus (HCV) are eligible if they have an undetectable HCV viral load. Patients must have completed curative anti-viral treatment ≥ 4 weeks prior to registration. ** NOTE: Patients without symptoms or prior history do not require testing prior to registration
- Co-morbid systemic illnesses or other severe concurrent disease that would make the patient inappropriate for entry into the study or interfere with proper assessment of safety and toxicity
- History of myocardial infarction ≤ 6 months prior to registration or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias
- Active autoimmune disease that has required systemic treatment (other than replacement therapy) ≤ 1 year prior to registration
- History of allogeneic stem cell transplant
- Receiving any other investigational agent with therapeutic intent
- Participants who are unable to swallow the DFMO solution or who are at risk for impaired absorption of oral medication. * NOTE: This restriction includes, but is not limited to, refractory vomiting, gastric resection/bypass, and duodenal/jejunal resection
- Patients with known hypersensitivity or allergy to DFMO or retifanlimab
- Contraindication to MRI or administration of gadolinium
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT07468136.
Locations matching your search criteria
United States
Minnesota
Rochester
PRIMARY GOALS:
I. Determine the safety and tolerability of retifanlimab and eflornithine (DFMO) in patients with residual or recurrent high-grade gliomas and identify the optimal dose of DFMO in this combination based on dose-limiting toxicities and pharmacodynamic impact. (phase I)
II. Determine the impact of retifanlimab, with or without DFMO, on T cell recruitment into high-grade gliomas. (phase IIa)
SECONDARY GOALS:
I. Determine the impact of DFMO + retifanlimab on T cell recruitment and phenotypes and myeloid cell abundance. (phase I)
II. Determine the tissue drug concentration of DFMO and retifanlimab. (phase I)
III. Evaluate radiographic tumor response to retifanlimab and DFMO based on Response Assessment in Neuro-Oncology (RANO) 2.0. (phase IIa)
IV. Determine the incidence and types of adverse events related to retifanlimab with DFMO in patients with recurrent high-grade glioma. (phase IIa)
CORRELATIVE RESEARCH:
I. Evaluate the impact of retifanlimab, with or without DFMO, on extracellular cytokines/chemokines within cerebrospinal fluid (CSF) after 14 days. (phase I)
II. Determine the impact of retifanlimab and DFMO on extracellular cytokines/chemokines within CSF throughout treatment. (phase IIa)
OUTLINE: This is a phase I, dose-escalation study of DFMO in combination with fixed dose retifanlimab, followed by a phase II study. Patients are assigned to either group A or B.
GROUP A: Patients receive retifanlimab intravenously (IV), over 30 minutes on day 1 of cycle 1 and DFMO orally (PO) every 8 hours (Q8H) on days 1-14 of cycle 1 in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care resection surgery. Patients then receive retifanlimab IV, over 30 minutes, on day 1 of each cycle and DFMO PO Q8H on days 1-14 of each cycle. Cycles repeat every 28 days for 16 additional cycles in the absence of disease progression or unacceptable toxicity. Patients undergo magnetic resonance imaging (MRI) and blood sample collection throughout the study and may optionally undergo CSF fluid collection with or without lumbar puncture.
GROUP B: Patients are randomized to 1 of 2 groups (group B1 or B2).
GROUP B1: Patients receive retifanlimab IV, over 30 minutes, on day 1 of cycle 1. Patients then undergo standard of care resection surgery. Patients then receive retifanlimab IV, over 30 minutes, on day 1 of each cycle and DFMO PO Q8H on days 1-14 of each cycle. Cycles repeat every 28 days for 16 additional cycles in the absence of disease progression or unacceptable toxicity. Patients undergo MRI and blood sample collection throughout the study and may optionally undergo cerebrospinal fluid collection with or without lumbar puncture.
GROUP B2: Patients receive retifanlimab IV, over 30 minutes on day 1 of cycle 1 and DFMO PO Q8H on days 1-14 of cycle 1, in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care resection surgery. Patients then receive retifanlimab IV, over 30 minutes, on day 1 of each cycle and DFMO PO Q8H on days 1-14 of each cycle. Cycles repeat every 28 days for 16 additional cycles in the absence of disease progression or unacceptable toxicity. Patients undergo MRI and blood sample collection throughout the study and may optionally undergo cerebrospinal fluid collection with or without lumbar puncture.
After completion of study treatment, patients are followed up every 3 months until progressive disease and every 6 months after progressive disease for 5 years after registration.
Trial PhasePhase I/II
Trial Typetreatment
Lead OrganizationMayo Clinic in Rochester
Principal InvestigatorTerence Calvin Burns
- Primary IDMC230718
- Secondary IDsNCI-2026-01392, 24-000432
- ClinicalTrials.gov IDNCT07468136