Efineptakin Alfa and Pembrolizumab for Recurrent Glioblastoma
This study is testing two immunotherapy drugs, efineptakin alfa and pembrolizumab, for people whose glioblastoma (a type of brain tumor) has returned. Efineptakin alfa helps your immune system fight cancer cells. Pembrolizumab is also an immunotherapy that helps your immune system attack cancer and may stop tumor cells from growing and spreading. Researchers want to see if giving these two drugs together can kill more tumor cells in patients with recurrent glioblastoma. You may be able to join if you are 18 or older and have glioblastoma that has come back after previous treatments. The main goal is to see how many participants are still alive after 9 months.
- Study design
- This is a Phase 2 interventional study planning to enroll 54 participants. It is testing the combination of efineptakin alfa and pembrolizumab.
- What's involved
- Participants will undergo biospecimen collection and a tumor biopsy. Efineptakin alfa is given as an injection into a muscle, and pembrolizumab is given through a vein.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will measure overall survival for up to 9 months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05465954
Efineptakin Alfa and Pembrolizumab for the Treatment of Recurrent Glioblastoma
Recruiting
PHASE2Ages 18+InterventionalTreatmentMayo ClinicInvestigator-initiated
~54 participants
Updated 2026-01-12 on ClinicalTrials.gov
What's tested:Biospecimen CollectionEfineptakin alfaPembrolizumabBiopsy
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall survival rate
Measured over Up to 9 months
Conditions
Where it's being run
1 sites across 1 statesMinnesota1
Study leadership
- Jian L. Campian, MD, PhD · PRINCIPAL_INVESTIGATOR · Mayo Clinic in Rochester
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age \>= 18 years
Disease characteristics:
Tissue-confirmed progressive or recurrent World Health Organization (WHO) Grade IV IDH wildtype glioblastoma (including molecular glioblastoma and gliosarcoma)
Previously treated with maximum feasible resection or biopsy, radiation, and temozolomide
Have an enhancing mass on magnetic resonance imaging (MRI) amenable to resection or biopsy of the tumor (as determined by the neurosurgeon pre-operatively) and histological diagnosis of glioblastoma from a prior biopsy or surgery
Willing to undergo clinically indicated biopsy and/or resection of their glioblastoma at Mayo Clinic in Rochester, Minnesota (MN).
Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 or 1 and Karnofsky Performance Scale (KPS) \>= 70 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) (without transfusion or erythropoietin \[EPO\] dependency =\< 7 days prior to assessment)
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)
Creatinine =\< 1.5 x upper limits of normal (ULN) OR measured or calculated creatinine clearance (per institutional standard) must be \>= 45 ml/min (obtained =\< 15 days prior to registration)
Total bilirubin =\<1.5 x ULN OR direct bilirubin =\< ULN for patients with total bilirubin levels \>1.5 x ULN (obtained =\< 15 days prior to registration)
Aspartate transaminase (AST) AND alanine transaminase (ALT) =\< 2.5 x ULN (obtained =\< 15 days prior to registration)
Prothrombin time (PT)/international normalized ratio (INR)/activated partial thromboplastin time (aPTT) =\< 1.5 x ULN OR if patient is receiving anticoagulant therapy then INR or aPTT is within target range of therapy (obtained =\< 15 days prior to registration)
Negative pregnancy test done =\< 7 days prior to registration, for persons of childbearing potential only (POCBP) Note: If testing done for eligibility is \> 72 hours prior to first dose, then pregnancy testing must be repeated, and result must be negative for patient to receive treatment.
POCBP or able to father a child must be willing to use adequate contraception starting with first dose through 180 days after last dose
Provide written informed consent
Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study).
Willing to provide tissue and blood samples for correlative research purposes
Exclusion
Any of the following because this study involves an investigational agent for which genotoxic, mutagenic, and teratogenic effects 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
Signs or symptoms of life-threatening raised intracranial pressure: as determined by the treating neurosurgeon, including severe headache, nausea, decreasing level of consciousness, precluding 4-7-day delay in scheduling neurosurgery (i.e., immediate surgery is indicated, and patient cannot wait).
Prior treatment
Received bevacizumab (AVASTIN) =\< 4 months prior to registration
Note: Bevacizumab is allowed for symptom control during the adjuvant phase of the study
Received a live vaccine =\< 30 days prior to registration.
Requirement for dexamethasone dose of \> 2mg/day =\< 2 days prior to registration
Failure to recover from any adverse events related to any of the following therapies received prior to registration:
Major surgery =\< 28 days prior to registration
Radiation therapy =\< 14 days prior to registration
Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment 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 prescribed regimens
Known history of human immunodeficiency virus (HIV) infection
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations (e.g., drug addiction) that would limit compliance with study requirements
Receiving any other investigational agent
Other active malignancy requiring systemic treatment =\< 1 year prior to registration
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 (i.e., with use of disease modifying agents, corticosteroids, or immunosuppressive drugs) =\< 2 years prior to registration NOTE: Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
Concurrent known active Hepatitis B (i.e., known positive hepatitis B virus \[HBV\] surface antigen \[HBsAg\] reactive) AND known active Hepatitis C (i.e., hepatitis C virus \[HCV\] ribonucleic acid \[RNA\] \[qualitative\] detected by polymerase chain reaction \[PCR\])
Note: No testing for Hepatitis B and Hepatitis C is required unless mandated by local health authority
NOTE: Patients with known Hepatitis B OR Hepatitis C may be enrolled if they meet the following criteria:
Hepatitis B: Patients who are HBsAG positive are eligible if they have received HBV antiviral therapy for at least 4 weeks and have undetectable HBV viral load prior to randomization. Patients should remain on anti-viral therapy throughout the treatment phase of the trial and should follow local guidelines for HBV anti-viral therapy after completing study treatment
Hepatitis C: Patients with history of Hepatitis C infection are eligible if HCV viral load is undetectable at screening. Patients must have completed curative anti-viral therapy at least 4 weeks prior to registration
Known history of active TB (Bacillus Tuberculosis)
History of (non-infectious) pneumonitis or interstitial lung disease that required steroids, or current pneumonitis or interstitial lung disease
Hypersensitivity to pembrolizumab or any of its excipients
Received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent within \< 12 months prior to registration
NOTE: If such therapy was given ≥ 12 months prior to registration, patient is eligible
History of allogenic tissue/solid organ transplant
What this trial measures
- Overall survival rateUp to 9 months
9-month overall survival rate (OS9) rate is defined as the number of "successes" (patients who are alive at least 9 months after beginning study therapy) divided by the total evaluable patients. All patients who have signed a consent form, are eligible, and have begun treatment in Cycle 1 (first cycle of neo-adjuvant pembrolizumab + efineptakin alfa \[NT-I7\]) will be considered evaluable for the primary endpoint.