Efineptakin alfa (NT-I7) Plus Pembrolizumab for the Treatment of Recurrent Glioblastoma


About this study

The purpose of this study is to determine the response rate to the combination of pembrolizumab and NT-I7 in patients with recurrent glioblastoma.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

 Inclusion Criteria:

  • Age ≥ 18 years.
  • Progressive or recurrent WHO Grade IV IDH wildtype glioblastoma (including molecular glioblastoma and gliosarcoma).
  • Have an enhancing mass on 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 resection or biopsy of their glioblastoma at Mayo Clinic in Rochester, MN.
  • ECOG Performance Status (PS) of 0 or 1 and KPS ≥ 70.
    • Note: PS must be assessed again within 7 days prior to first dose of study drug.
  • The following laboratory values obtained ≤ 15 days prior to registration:
    • Absolute neutrophil count (ANC) ≥ 1500/mm^3;
    • Platelet count ≥ 100,000/mm^3;
    • Hemoglobin ≥ 9.0 g/dL without transfusion or EPO dependency (≤ 7 days prior to assessment);
    • Creatinine ≤ 2.0 x ULN OR measured or calculated creatinine clearance (per institutional standard) must be ≥ 45 ml/min;
    • Total bilirubin ≤ 1.5 x ULN OR Direct bilirubin ≤ULN for patients with total bilirubin levels > 1.5 x ULN;
    • Aspartate transaminase (AST) AND alanine transaminase (ALT) ≤ 2.5 x ULN;
    • INR/PT/aPTT ≤ 1.5 × ULN OR if patient is receiving anticoagulant therapy then INR or aPTT is within target range of therapy.
  • 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 120 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 Criteria:

Any of the following because this study involves an investigational agent whose 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 defined by the treating neurosurgeon, including severe headache, nausea, decreasing level of consciousness, precluding 4-7 day delay in scheduling neurosurgery.
  • Prior treatment.
  • Received bevacizumab (AVASTIN) ≤ 28 days 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.
  • Major surgery ≤ 28 days prior to registration.
  • Requirement for dexamethasone dose of > 2mg/day ≤ 2 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.
  • Immunocompromised patients and patients known to be HIV positive and currently receiving antiretroviral therapy.
  • 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 that would limit compliance with study requirements.
  • Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm.
  • 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.
  • Known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
  • Known history of active TB (Bacillus Tuberculosis).
  • History of (non-infectious) pneumonitis that required steroids or current pneumonitis.
  • Hypersensitivity to pembrolizumab or any of its excipients.
  • Received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Jian Campian, M.D., Ph.D.

Open for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information


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