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New drug combo shows promise against childhood brain tumors

NCT ID NCT04049669

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

130 people

The number who actually took part.

Started

Oct 2019

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

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

3 to 21 years

Sex

Anyone

Healthy volunteers

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

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

  • Augusta University, Georgia Cancer Center

    Augusta, Georgia, 30912, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Emory University, Children's Heathcare of Atlanta

    Druid Hills, Georgia, 30322, United States

  • MD Anderson Cancer Center

    Houston, Texas, 77030, United States

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