Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer

{ "Chemo-immunotherapy for Pediatric Brain Cancer (Ependymoma, Medulloblastoma, Glioblastoma)", "This study is testing a new combination of treatments for children and young adults (ages 3-25) with certain types of brain cancer that have progressed or returned. It combines chemotherapy (Cyclophosphamide and Etoposide) with two immunotherapy drugs, Indoximod and Ibrutinib. Researchers believe that combining Ibrutinib (which targets a protein called BTK) with Indoximod (which targets a protein called IDO) during chemotherapy might boost the body's immune response against the cancer. The main goals are to find a safe dose of Ibrutinib and see how many patients respond to the treatment. This study is currently recruiting about 37 participants.", "design": "This is an open-label Phase 1 study, meaning both you and your doctors will know which treatments you are receiving. It plans to enroll about 37 participants.", "commitments": "You would take Indoximod by mouth twice daily throughout each treatment cycle. Depending on your assigned regimen, you would also take Ibrutinib by mouth once daily for 14 or 21 days of each cycle, and Cyclophosphamide and Etoposide by mouth once daily for 21 days of each cycle.", "compensation": "Not stated in the trial record.", "follow_up": "Researchers will monitor for side effects during the first 90 days of treatment and track how well the treatment works for up to 5 years.", }

Study design: Not specified.
What's involved: Not specified in the trial record.
Compensation: Not stated in the trial record.
Follow-up: Not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer

Recruiting

PHASE1Ages 3–25InterventionalTreatment

Theodore S. Johnson

~37 participants

Updated 2026-01-09 on ClinicalTrials.gov

What's tested:IndoximodIbrutinibCyclophosphamideEtoposideTemozolomide

At a glance

Recruiting sites

1 of 1 listed site is recruiting right now

RecruitingSuspended, closed, or not yet open

What they're measuring

Incidence of regimen-limiting toxicity (RLT) for Regimen A

Measured over First 90 days of treatment

+3 more outcomes measured

Conditions

Ependymoma

Medulloblastoma

Glioblastoma

Primary Brain Tumor

Where it's being run

1 sites across 1 states

Georgia1

Study leadership

Theodore S. Johnson, MD, PhD · PRINCIPAL_INVESTIGATOR · Augusta University

Who to contact

Theodore S. Johnson, MD, PhD

[email protected]

706-721-4962

Email the study team

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Eligibility criteria

Inclusion

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.

Creatinine clearance (CLcr) \> 25 mL/min (by calculated methods) AND Creatinine ≤ 1.5-times upper limit of age-adjusted normal for age of patient.

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.

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).

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.

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.

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.

Exclusion

Allergies, allergic conditions, and reactive inflammatory conditions that are not autoimmune in nature would not exclude patients (e.g., eczema, asthma, etc.).

What this trial measures

Incidence of regimen-limiting toxicity (RLT) for Regimen AFirst 90 days of treatment
To determine the pediatric recommended phase 2 dose (RP2D) of ibrutinib, when combined with indoximod-based chemo-immunotherapy (Regimen A)
Objective Response Rate (ORR) for Regimen AUp to 5 years
Defined as the proportion of patients with a best objective response of either complete response (CR) or partial response (PR), using "immunotherapy Response Assessment for Neuro-Oncology" (iRANO) criteria
Incidence of regimen-limiting toxicity (RLT) for Regimen BFirst 90 days of treatment
To determine the pediatric recommended phase 2 dose (RP2D) of ibrutinib, when combined with indoximod-based chemo-immunotherapy (Regimen B)
Objective Response Rate (ORR) for Regimen BUp to 5 years
Defined as the proportion of patients with a best objective response of either complete response (CR) or partial response (PR), using "immunotherapy Response Assessment for Neuro-Oncology" (iRANO) criteria

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