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High Grade Astrocytic TumorPhase 2NCT05465954

Efineptakin Alfa and Pembrolizumab for the Treatment of Recurrent Glioblastoma

A Phase 2 study testing Biospecimen Collection, Efineptakin alfa and Pembrolizumab in people with High Grade Astrocytic Tumor and Recurrent Glioblastoma, IDH-Wildtype, run by Mayo Clinic at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This phase II trial tests the safety and side effects of efineptakin alfa and pembrolizumab in treating patients with glioblastoma that has come back (recurrent). Efineptakin alfa is an immunotherapy drug that works by helping the immune system fight tumor cells. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Efineptakin alfa and Pembrolizumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
53 participants
Where
1 siteUnited States
Study dates
24 Jan 2023to 2 Sep 2031
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic

    Rochester, Minnesota, 55905

Other trials in United States

About this study

Official title: Efficacy and Safety Study of Neoadjuvant Efineptakin Alfa (NT-I7) and Pembrolizumab in Recurrent Glioblastoma

This phase II trial tests the safety and side effects of efineptakin alfa and pembrolizumab in treating patients with glioblastoma that has come back (recurrent). Efineptakin alfa is an immunotherapy drug that works by helping the immune system fight tumor cells. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving efineptakin alfa and pembrolizumab may kill more tumor cells in patients with recurrent glioblastoma.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To determine the efficacy of pembrolizumab + efineptakin alfa (NT-I7) in combination with surgery in patients with recurrent glioblastoma multiforme (GBM) using the rate of overall survival at 9 months.

SECONDARY OBJECTIVES:

  1. To assess progression-free survival in recurrent GBM patients treated with pembrolizumab + NT-I7 in combination with surgery.

II. To determine the objective response rate in recurrent GBM patients treated with pembrolizumab + NT-I7 in combination with surgery.

III. To assess changes in absolute lymphocyte counts (ALC) over time in recurrent GBM patients treated with pembrolizumab + NT-I7 in combination with surgery.

TERTIARY OBJECTIVES:

  1. To assess the adverse event (AE) and toxicity profile of pembrolizumab + NT-I7 in combination with surgery in patients with recurrent GBM.

II. To assess the anti-glioma immune response in patients with recurrent GBM, before and after pembrolizumab and NT-I7 treatment, including assessment of cytokine profiling, immune cell phenotyping, function, and activation in the pre/post-treatment blood and tumor tissue when available.

III. To test tumor mutation burden on tumor/immune cells by validated comprehensive genomic profiling.

IV. To evaluate the changes for tumor microenvironment (TME) post pembrolizumab and NT-I7 and their correlations with treatment response and survival.

  1. To evaluate tumor infiltrating lymphocytes (CD4, CD8, Treg, etc) in the tumor tissue pre- and post-treatment (when available).

VI. To assess changes in repertoire breadth and clonality by T-cell receptor (TCR) sequencing.

VII. To explore potential tissue and blood biomarkers that may predict response.

OUTLINE:

BEFORE SURGERY: Patients receive pembrolizumab intravenously (IV) over 30 minutes and efineptakin alfa intramuscularly (IM) on day 1. Patients then undergo surgery 1 week later.

AFTER SURGERY: Patients receive pembrolizumab IV over 30 minutes and efineptakin alfa IM on day 1 of each cycle. Cycles repeat every 42 days for 2 years in the absence of disease progression or unacceptable toxicity.

Patients undergo magnetic resonance imaging (MRI) or computed tomography (CT) at baseline and on study. Patients also undergo tumor biopsy at baseline and blood sample collection on study.

After completion of study treatment, patients are followed up at 30 days and every 2-3 months until disease progression (if applicable), and then every 6 months for up to 5 years from study registration.

Being tested
Biospecimen Collection, Efineptakin alfa, Pembrolizumab, Biopsy
Medical subject headings
Glioblastoma, Gliosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Correlative studies

  • Efineptakin alfaBiological

    Also called GX-I7, Hyleukin-7 (TM), Il-7 Hybrid Fc, IL-7-hyFc

    Given IM

  • PembrolizumabBiological

    Also called Keytruda, Lambrolizumab, MK-3475, SCH 900475

    Given IV

  • BiopsyProcedure or surgery

    Also called BIOPSY_TYPE, Bx

    Undergo tumor biopsy

Study groups in NCT05465954
Study groupRoleWhat they receive
Treatment (efineptakin alfa, pembrolizumab)ExperimentalBEFORE SURGERY: Patients receive pembrolizumab IV over 30 minutes and efineptakin alfa IM on day 1. Patients then undergo surgery 1 week later. AFTER SURGERY: Patients receive pembrolizumab IV over 30 minutes and efineptakin alfa IM on day 1 of each cycle. Cycles repeat every 42 days for 2 years in the absence of disease progression or unacceptable toxicity. Patients undergo MRI or CT at baseline and on study. Patients also undergo tumor biopsy at baseline and blood sample collection on study.
Receives: Procedure: Biospecimen Collection; Biological: Efineptakin alfa; Biological: Pembrolizumab; Procedure: Biopsy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall survival rate

    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.

    Measured: Up to 9 months

Secondary outcomes (4)

  1. Progression free survival time

    Progression is defined according to Immunotherapy Response Assessment in Neuro-Oncology (iRANO) criteria. The median and 95% confidence intervals will be estimated using the Kaplan-Meier estimator.

    Measured: From the date of starting study treatment to the date of disease progression or death resulting from any cause, whichever comes first, assessed up to 5 years

  2. Objective response rate (ORR)

    Objective response rate (ORR) is defined as the number of responses achieved during treatment with pembrolizumab + NT-I7 in combination with surgery divided by the total number of evaluable patients. A patient will be deemed to have a response if complete response (CR) or partial response (PR) is achieved. Only patients with measurable disease documented on the post-operative baseline scan (prior to initiation of Cycle 2) will be considered evaluable for ORR. Point estimates will be generated for response rate with corresponding 95% binomial confidence intervals (Duffy and Santner).

    Measured: Up to 5 years

  3. Changes in absolute lymphocyte counts (ALC)

    Point estimates will be generated for the difference (at the magnitude of a fold-change) with corresponding 95% confidence intervals. All other changes in ALC over time will be exploratory and hypothesis generating. Frequency distributions, graphical techniques and other descriptive measures will form the basis of these analyses.

    Measured: From baseline up to 12 weeks

  4. Incidence of adverse events

    The number and severity of all adverse events will be tabulated and summarized in this patient population. Specifically, the overall percentages for Grade 3 or higher adverse events considered at least possibly related to treatment will also be calculated and reported. All other adverse event analysis will be exploratory and hypothesis generating. Frequency distributions, graphical techniques and other descriptive measures will form the basis of these analyses.

    Measured: Up to 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jul 2022
  2. Study start24 Jan 2023
  3. Primary completion2 Sep 2026
  4. Study completion2 Sep 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
53 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05465954
Sponsor's protocol ID
MC210706
Other IDs
21-011200, R01CA288862
Status last verified
Sep 2026
First submitted
18 Jul 2022
Last updated
5 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Efineptakin alfa and Pembrolizumab in people with High Grade Astrocytic Tumor and Recurrent Glioblastoma, IDH-Wildtype, run by Mayo Clinic at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall survival rate (time frame: Up to 9 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05465954.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2026.

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