Immunotherapy drug nivolumab tested in young brain cancer patients
NCT ID NCT04323046
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing the immunotherapy drug nivolumab in children and young adults whose high-grade glioma has come back or is getting worse. Participants receive the drug before and after surgery to remove as much of the tumor as possible. The main goals are to check for side effects and to see if the drug changes certain genetic signals in the tumor. Only 9 people are enrolled, so this is a very small study focused on safety and biological activity, not yet on proving the drug works.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nivolumab (Opdivo), an immunotherapy drug
- What this could lead to
- If it works, this could point toward a new treatment option for young people with recurrent high-grade glioma, a type of brain cancer with few effective therapies.
- What could go wrong
- This is a very early, small pilot study with only 9 participants. It primarily looks at safety and biological changes, not yet at curing the disease. The drug may not work or could cause serious side effects.
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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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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9 people
The number who actually took part.
- Started
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Oct 2020
- Expected to finish
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Jun 2028
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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6 months to 25 years
- 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: 1. Participants with recurrent or progressive high-grade gliomas (HGG) (World Health Organization (WHO) grade III or grade IV) who are candidates for surgical tumor debulking will be enrolled in this trial 2. All assessments are to occur within 14 days of registration except where otherwise noted. The participant and their legal parent/guardian must be thoroughly informed about all aspects of the study, including the study visit schedule and required evaluations and all regulatory requirements for informed consent. The written informed consent must be obtained from the participant and legal parent/guardian prior to enrollment 3. Have a history of previously treated histologically confirmed World Health Organization grade III or IV HGG. Previous first line therapy with radiation and/or chemotherapy 4. Have evidence of recurrence or progression of disease by MRI scan 5. Participants must be adequate medical candidates for surgical resection. The intent of surgical resection is to allow both cytoreduction and tumor debulking as part of standard of care, and also collect a minimum of 100 mg of tumor tissue for the study tissue endpoints 6. A primary goal of surgery must be cytoreduction, and not solely on diagnostic biopsy 7. Age: Participants must be \> 6 months and \< 25 years of age at time of enrollment 8. Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants =\< 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score 9. Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment (on stable or tapering dosing of steroids). A baseline detailed neurological exam should clearly document the neurologic status of the patient at the time of enrollment on the study. 10. Prior Therapy: Participants must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the defined eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately * Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive. At least 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea) * An interval of at least 12 weeks from the completion of radiation therapy to registration unless there is unequivocal histologic confirmation of tumor progression * Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events (AEs) are known to occur. The duration of this interval must be discussed with the study chair. * Had their last dose of biologic (anti-neoplastic agent) ≥7 days prior to study registration, or beyond the time during which AEs are known to occur. * Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count \[ANC\] counts): At least 7 days after the last dose of agent * Interleukins, interferons and cytokines (other than hematopoietic growth factors): \>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors) * Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1 * An interval of at least 12 weeks from prior exposure to PD-1 or PD-L1 inhibitors. * Stem cell infusion (with or without total-body irradiation (TBI)): * Autologous stem cell infusion including boost infusion: \>= 42 days 11. Participants must be willing to forego cytotoxic anti-tumor therapies except study-defined therapy while being treated on study 12. Organ Function Requirements: * Peripheral absolute neutrophil count (ANC) \>= 1000/mm\^3 * Platelet count \>= 100,000/mm\^3 * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 OR a serum creatinine based on age/gender as follows: * Age: Maximum Serum Creatinine (mg/dL) * 6 months to \< 3 years: 0.6 (male and female) * 3 to \< 6 years: 0.8 (male and female) * 6 to \< 10 years: 1 (male and female) * 10 to \< 13 years: 1.2 (male and female) * 13 to \< 16 years: 1.5 (male), 1.4 (female) * \>= 16 years: 1.7 (male), 1.4 (female) * Bilirubin (sum of conjugated and unconjugated) =\< 1.5 x upper limit of normal (ULN) for age (except participants with Gilbert syndrome who must have a total bilirubin level of \< 3.0 * Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =\< 3.0 x ULN * Serum albumin \>= 2 13. Pregnancy: The effects of nivolumab on the developing human fetus are unknown. For this reason women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 5 months after completion of therapy. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. 14. MRI within 28 days prior to registration. Exclusion Criteria: 1. Current or planned participation in a study of an investigational agent or using an investigational device. 2. Has a diagnosis of immunodeficiency. 3. Has tumor primarily localized to the brainstem or spinal cord. 4. Has presence of diffuse leptomeningeal disease or or disseminated/multi-focal disease, or extracranial disease. 5. Has received systemic immunosuppressive treatments (such as methotrexate, chloroquine, azathioprine, etc.), aside from anti-neoplastic chemotherapy or systemic corticosteroids within six months of registration. 6. Participants with a concurrent condition requiring systemic treatment with either corticosteroids (\> 0.25 mg/kg daily prednisone equivalent) or other immunosuppressive medications within 14 days of start of study treatment. Inhaled or topical steroids, and adrenal replacement steroid doses \> 0.25 mg/kg daily prednisone equivalent, are permitted in the absence of active autoimmune disease. 7. Unable to taper steroids due to ongoing mass effect; a maximum dexamethasone dose of 0.1 mg/kg/day is allowed (4mg maximum), but preferably have been discontinued (inhaled or topical use of steroids is allowed). 8. Has a known history of active TB (Bacillus tuberculosis). 9. Has a known additional malignancy that is progressing or requires active treatment within 3 years of registration. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy. 10. 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 (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment. 11. Has known history of, or any evidence of active non-infectious pneumonitis. 12. Has an active infection requiring systemic therapy. 13. Has a known hypersensitivity to any of the study therapy products. 14. Has a known history of positive test for human immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS). * NOTE: Testing for HIV must be performed at sites where mandated locally 15. Any prior positive test result for hepatitis B virus or hepatitis C virus indicating presence of virus, e.g., hepatitis B surface antigen (HBsAg, Australia antigen) positive, or hepatitis C antibody (anti-HCV) positive (except if HCV-ribonucleic acid (RNA) negative). 16. Participants who have had prior allogenic hematopoietic stem cell transplant (HSCT). 17. Any serious or uncontrolled medical disorder that, in the opinion of the investigator may increase the risk associated with study participation or study drug administration, impair the ability of the participant to receive protocol therapy or interfere with interpretation of study results.
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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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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20310, United States
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Duke Children's Hospital & Health Center
Durham, North Carolina, 27705, United States
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Hackensack Meridian Children's Health at Joseph M. Sanzari Children's Hospital
Hackensack, New Jersey, 07601, United States
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Johns Hopkins University
Baltimore, Maryland, 21287, United States
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Perth Children's' Hospital
Perth, Western Australia, 6009, Australia
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Rady Children's Hospital
San Diego, California, 92123, United States
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Riley Children's Hospital
Indianapolis, Indiana, 46202, United States
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Sydney Children's Hospital
Sydney, New South Wales, 1291, Australia
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The Children's Hospital at Westmead
Westmead, New South Wales, 2152, Australia
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University of Alabama at Birmingham, Children's of Alabama
Birmingham, Alabama, 35233, United States
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University of California, San Francisco
San Francisco, California, 94115, United States
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University of Florida
Gainesville, Florida, 32611, United States
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University of Utah
Salt Lake City, Utah, 84113, United States
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Washington University St. Louis
St Louis, Missouri, 63110, United States
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Women's and Children's Hospital
North Adelaide, South Australia, 5006, Australia
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