Could a repurposed drug boost immune attack on childhood brain tumors?
NCT ID NCT05106296
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing whether adding the drug ibrutinib to a chemo-immunotherapy regimen can safely help children and young adults (ages 6–25) with brain cancers that have come back or not responded to standard treatment. The study combines ibrutinib with indoximod and two chemotherapy drugs to try to boost the immune system's ability to fight the tumor. The main goals are to find the best safe dose and to see if the combination shrinks tumors.
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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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About 37 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2022
- Expected to finish
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Sep 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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3 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: Diagnosis: * Patients must have prior documented progressive or refractory disease with histologically proven initial diagnosis of ependymoma, medulloblastoma, glioblastoma, or another type of primary cancer of the central nervous system with no curative conventional therapy options available. * Metastatic disease is acceptable. * Patients must have MRI confirmation (with and without gadolinium contrast) of current active disease. Patients must be able to swallow pills. Lansky or Karnofsky performance status score must be ≥ 50%. Adequate renal function: * Creatinine clearance (CLcr) \> 25 mL/min (by calculated methods) AND Creatinine ≤ 1.5-times upper limit of age-adjusted normal for age of patient. Adequate liver function: * Alanine aminotransferase (ALT) ≤ 3-times upper limit of normal. * Aspartate aminotransferase (AST) ≤ 3-times upper limit of normal. * Total bilirubin ≤ 1.5-times upper limit of normal unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin. Adequate bone marrow function: * Absolute neutrophil count (ANC) ≥ 1000/mm3 (independent of growth factor support). * Platelets ≥ 100,000/mm3 (independent of transfusion support). * Hemoglobin ≥ 8 g/dL (independent of transfusion support). Seizure disorders must be well controlled on antiepileptic medication. Prior therapy: * Patients previously treated with chemotherapy drugs included in this protocol are eligible for enrollment. * At the time of Screening, patients must be at least 21 days from the administration of any investigational agent (other than indoximod) or prior cytotoxic therapy (including chemotherapy). * At the time of Screening, patients must be at least 28 days from administration of antibody-based therapies (e.g., bevacizumab), tumor-directed vaccines, or cellular immune therapies (e.g., T cells, NK cells, etc.). * At the time of Screening, patients must be at least 56 days from administration of tumor-directed therapies using infectious agents (e.g., viruses, bacteria, etc.). * At the time of Screening, patients must be at least 90 days from any radiation or proton therapy (all modalities, including radiosurgery) that targeted all sites of known disease. * There is no lock-out window for patients who were treated with focal radiation or focal proton therapy (all modalities, including radiosurgery) that did not target all disease sites, if at least one site of active tumor is expected to persist and/or grow. Concurrent anti-neoplastic therapy: * No investigational or commercial agents, including intrathecal drugs, other than that described by this clinical study protocol (GCC2020) may be administered with the intent to treat the patient's malignancy while they remain enrolled on this study. Contraception, pregnancy, and breastfeeding: * Women of childbearing potential and men who are sexually active must be practicing a highly effective method of birth control during and after the study. Men must agree to not donate sperm during and for 3 months after the study. * Women who are pregnant or breastfeeding are ineligible for this study. * Patients who become pregnant while participating in this study will have to stop Study Therapy. Patients, or their parent for patients less than 18 years of age, must sign an Informed Consent Document indicating that they understand the purpose of and procedures required for the study, including biomarkers, and are willing to participate in the study. . Exclusion Criteria: Patients who are unable to swallow pills. Patients with known hypersensitivity to any drugs in the treatment plan. Patients with active autoimmune disease that requires systemic therapy. * Allergies, allergic conditions, and reactive inflammatory conditions that are not autoimmune in nature would not exclude patients (e.g., eczema, asthma, etc.). Pregnant or breastfeeding women. Major surgery or a wound that has not fully healed within 4 weeks of Screening. Known central nervous system lymphoma. Patients with active bleeding or history of thrombotic or hemorrhagic stroke, or intracranial hemorrhage, within 6 months prior to Screening; with the exception of retained blood products from recent prior uncomplicated surgery (e.g., tumor biopsy, debulking, or resection; VP shunt placement, etc.). Requires anticoagulation with warfarin or equivalent vitamin K antagonists (e.g., phenprocoumon). Requires chronic treatment with strong CYP3A inhibitor drugs. Clinically significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of Screening, or any Class 3 (moderate) or Class 4 (severe) cardiac disease as defined by the New York Heart Association Functional Classification. Patients with baseline QTc interval of more than 470 msec at the time of Screening, and patients with congenital long QT syndrome. Vaccinated with live, attenuated vaccines within 4 weeks of Screening. Known history of human immunodeficiency virus (HIV) or active Hepatitis C Virus or active Hepatitis B Virus infection or any uncontrolled active systemic infection. Any life-threatening illness, medical condition, or organ system dysfunction which, in the investigator's opinion, could compromise the patient's safety, interfere with the absorption or metabolism of ibrutinib, indoximod, or chemotherapy, or put the study outcomes at undue risk.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Augusta University, Georgia Cancer Center
RECRUITINGAugusta, Georgia, 30912, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can an existing drug boost bevacizumab against recurrent brain cancer?
- One extra radiation dose after standard brain cancer therapy: can it boost the immune system?
- Radioactive tracer lights up Oxygen-Starved brain tumors
- Can a common virus vaccine help fight brain cancer?
- Radioactive tiles target brain tumors in frail patients