New drug combo shows promise against childhood brain tumors
NCT ID NCT04049669
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether adding an experimental drug called indoximod to standard chemotherapy and radiation can help children aged 3 to 21 with certain brain cancers. The drug aims to boost the body's immune system to fight tumors more effectively. The trial includes children with progressive glioblastoma, medulloblastoma, or ependymoma, as well as those newly diagnosed with a type of brainstem tumor called DIPG.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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130 people
The number who actually took part.
- Started
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Oct 2019
- Expected to finish
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Oct 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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3 to 21 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: * Progressive disease with histologically proven initial diagnosis of glioblastoma, medulloblastoma, or ependymoma; With confirmation of progression by either MRI or CSF analysis; Measureable disease is not required for study entry; Patients with progressive disease must have been previously treated with therapeutic radiation as part of treatment for the initial brain cancer diagnosis or for a prior relapse. * Newly diagnosed DIPG (diffuse intrinsic pontine glioma) with no prior therapy (including no prior radiation); Biopsy is not required for DIPG. * Central review of tissue diagnosis is required, except non-biopsied DIPG; Archival tumor tissue must be located and available prior to study entry. * Patients with metastatic disease are eligible. Lansky or Karnofsky performance status score must be ≥ 50%. Adequate renal function: creatinine ≤ 1.5-times upper limit of age-adjusted normal. Adequate liver function: * ALT ≤ 5-times upper limit of normal. * Total bilirubin ≤ 1.5-times upper limit of normal. Adequate Bone marrow function: * Absolute neutrophil count (ANC) ≥ 750/mcL. * Platelets ≥ 75,000/mcL (transfusion independent). * Hemoglobin ≥ 8 g/dL (transfusion independent). Central nervous system: seizure disorders must be well controlled on antiepileptic medication. Prior therapy * DIPG patients must not have been treated with any prior radiation or medical therapy. * Patients previously treated with indoximod are excluded. * Patients previously treated with any other immunotherapy agent, including other IDO-targeted drugs, are eligible for enrollment. * Patients previously treated with chemotherapy drugs included in this protocol are eligible for enrollment. Patients must be 14 days from the administration of any investigational agent or prior cytotoxic therapy with the following exceptions: * Temozolomide dosed at or above 150 mg/m2 (allowed, but must be at least 21 days from the last dose of temozolomide). * Must be 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). * Must be 56 days from administration of tumor-directed therapies using infectious agents (e.g., viruses, bacteria, etc). Pregnant women are excluded from this study, where pregnancy is confirmed by a positive urine or serum hCG laboratory test. Patients must be able to swallow pills. . Exclusion Criteria: Patients who cannot swallow indoximod pills are excluded. Patients previously treated with indoximod are excluded. Patients with DIPG who have been treated with any prior radiation or medical therapy are excluded. Midline glioma that does not include significant brain stem involvement is not considered DIPG for enrollment purposes, and is excluded. Patients with active systemic infection requiring treatment, including any HIV infection or toxoplasmosis, are excluded. Patients with active autoimmune disease that requires systemic therapy are excluded. Pregnant women are excluded
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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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Augusta University, Georgia Cancer Center
Augusta, Georgia, 30912, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Emory University, Children's Heathcare of Atlanta
Druid Hills, Georgia, 30322, United States
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MD Anderson Cancer Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a trio of repurposed drugs shrink recurrent brain tumors?
- 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