Immune drug takes on recurrent brain tumors in small pilot study
NCT ID NCT02658279
First seen Jun 26, 2026 · Last updated Aug 21, 2026 · Updated 3 times
Summary
This pilot study tests whether the drug pembrolizumab can shrink or control recurrent malignant glioma, a type of brain cancer, in people whose tumors have many genetic mutations (hypermutator phenotype). The 27 participants have already had radiation and chemotherapy. The goal is to see if the drug can stop tumor growth for at least 6 months or shrink it by half.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- pembrolizumab (a drug that helps the immune system attack cancer cells)
- What this could lead to
- If it works, this could point toward a treatment option for people with recurrent malignant glioma that has a hypermutator phenotype.
- What could go wrong
- This is a very early, small pilot study with only 27 participants. The drug may not shrink or control the cancer, and it can cause side effects like immune system overactivation.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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27 people
The number who actually took part.
- Started
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Jan 2016
- Expected to finish
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Jan 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Histologically confirmed diagnosis of malignant glioma by enrolling institution: * WHO grade IV tumors (GBM or its variants) * WHO grade III anaplastic astrocytoma or oligodendroglial tumors or * WHO grade II gliomas, if MRI shows contrast enhancement * Tumor recurrence after previous treatment, which must have included at least radiation therapy and one cytotoxic chemotherapy. There is no limit on number of previous recurrences or lines of treatment. * Previously obtained tumor sample exhibits a hypermutator phenotype. For the purposes of this trial, a hypermutator phenotype is defined as tumors harboring 30 mutations (non-synonymous somatic point or indel mutations) detected by the MSK-IMPACT or comparable next generation sequencing performed in a CLIA environment. Contingent to approval by the MSK Principal Investigator, patients with less than 30 mutations may be eligible if they display a mutation in a mismatch repair gene or other mutations in genes known to be associated with hypermutator phenotypes or microsatellite instability, including but not limited to MLH1, MSH2, MSH6, PMS2, POLE, POLD as determined by validated methods, or if microsatellite instability is present, as identified by polymerase chain reaction (PCR) or other validated methods. \*Note: The MSK-IMPACT (Integrated Mutation Profiling for Actionable Cancer Targets) assay is a next generation genomic profiling performed on formalin-fixed, paraffin-embedded (FFPE) tumor tissue in a CLIA-certified Molecular Diagnostic Service laboratory. IMPACT provides full exon coverage of 410 cancer related genes and can detect base substitutions, small indels, copy number alterations and selected gene re-rearrangements. In some cases, additional assays such as Sanger Sequencing or fluorescence in situ hybridization (FISH) may be required to confirm specific results detected on IMPACT. Patients at MSK will have this assay to determine eligibility. Use of other validated next-generation sequencing techniques for eligibility may be considered, provided they are performed in a CLIA-certified laboratory and are approved by the MSK Principal Investigator. * Be willing and able to provide written informed consent/assent for the trial. * Be ≥ 18 years of age on day of signing informed consent. * An interval of ≥ 12 weeks from the end of prior radiation therapy is required unless there is either: i) histopathologic confirmation of recurrent tumor, or ii) new enhancement on MRI outside of the radiation treatment field * An interval of ≥ 4 weeks after the last administration of any investigational agent or any other treatment prior to first dose of pembrolizumab * Must have recovered (i.e., ≤ Grade 1 or at baseline) from adverse events of any previous treatment. Note: Surgical resection for recurrent tumor prior to enrollment is allowed. * Karnofsky performance status of ≥ 70 * Demonstrate adequate organ function as per below. All screening labs should be performed within 14 days of treatment initiation. * Absolute neutrophil count (ANC) ≥1,500 /mcL * Platelets ≥100,000 / mcL * Hemoglobin ≥9 g/dL or ≥ 5.6 mmol/L without transfusion or EPO dependency (within 7 days of assessment) * Serum creatinine ≤1.5 X upper limit of normal (ULN) OR measured or calculated creatinine clearance (GFR can also be used in place of creatinine or CrCl) ≥ 60 mL/min for subject with creatinine levels \> 1.5 X institutional ULN * Serum total bilirubin ≤ 1.5 X ULN OR direct bilirubin ≤ ULN for subjects with total bilirubin levels \> 1.5 ULN * AST (SGOT) and ALT (SGPT) ≤ 2.5 X ULN * Albumin \>2.5 mg/dL * International Normalized Ratio (INR) or Prothrombin Time (PT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants * Activated Partial Thromboplastin Time (aPTT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants * Female subject of childbearing potential should have a negative urine or serum pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. * Female subjects of childbearing potential should be willing to use 2 methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through 120 days after the last dose of study medication (Reference Section 9.7.2). Subjects of childbearing potential are those who have not been surgically sterilized or have not been free from menses for \> 1 year. * Male subjects should agree to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy. Exclusion Criteria: * Subjects requiring escalating or chronic supraphysiologic doses of corticosteroids (\> 10 mg/d of prednisone equivalent) for control of disease at the time of registration * Previous or current treatment with an anti-CTLA-4, anti-PD-1, anti-PD-L1, or anti-PD-L2 agent. * Hypersensitivity to pembrolizumab or any of its excipients. * Has a diagnosis of immunodeficiency, including Human Immunodeficiency Virus (HIV) or acquired immunodeficiency syndrome (AIDS) * Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA \[qualitative\] is detected). * Has a known history of active TB (Bacillus Tuberculosis) * Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. * Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment. * Has known history of, or any evidence of active, non-infectious pneumonitis. * Has an active infection requiring systemic therapy. * Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator. * Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial. * Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment. * Unable to undergo MRI of the brain (i.e. pacemaker or any other contraindication for MRIs). * Has received a live vaccine within 30 days of planned start of study therapy. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines, and are not allowed. * Has previously received treatment with bevacizumab
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Dana Farber Cancer Institute
Boston, Massachusetts, 02115, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Md Anderson Cancer Center
Houston, Texas, 77030, United States
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Memorial Sloan Kettering Bergen
Montvale, New Jersey, 07645, United States
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Memorial Sloan Kettering Cancer Center
Basking Ridge, New Jersey, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Memorial Sloan Kettering Commack
Commack, New York, 11725, United States
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Memorial Sloan Kettering Monmouth
Middletown, New Jersey, 07748, United States
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Memorial Sloan Kettering Nassau
Uniondale, New York, 11553, United States
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Memorial Sloan Kettering Westchester
East White Plains, New York, 10604, United States
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University of California San Francisco
San Francisco, California, 94143, United States
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University of California, Los Angeles
Los Angeles, California, 90095, United States
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University of Miami
Miami, Florida, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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