Ignite Creation Date:
2025-12-24 @ 11:02 PM
Ignite Modification Date:
2026-01-01 @ 7:25 AM
Study NCT ID:
NCT04049669
Status:
RECRUITING
Last Update Posted:
2025-07-16
First Post:
2019-08-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG
Sponsor:
Theodore S. Johnson
Organization:
Study Overview
Official Title:
Phase 2 Trial of Indoximod With Chemotherapy and Radiation for Children With Progressive Brain Tumors or Newly Diagnosed DIPG
Status:
RECRUITING
Status Verified Date:
2025-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Indoximod was developed to inhibit the IDO (indoleamine 2,3-dioxygenase) enzymatic pathway, which is important in the natural regulation of immune responses. This potent immune suppressive mechanism has been implicated in regulating immune responses in settings as diverse as infection, tissue/organ transplant, autoimmunity, and cancer. By inhibiting the IDO pathway, we hypothesize that indoximod will improve antitumor immune responses and thereby slow the growth of tumors.
The central clinical hypothesis for the GCC1949 study is that inhibiting the pivotal IDO pathway by adding indoximod immunotherapy during chemotherapy and/or radiation is a potent approach for breaking immune tolerance to pediatric tumors that will improve outcomes, relative to standard therapy alone.
This is an NCI-funded (R01 CA229646, MPI: Johnson and Munn) open-label phase 2 trial using indoximod-based combination chemo-radio-immunotherapy for treatment of patients age 3 to 21 years who have progressive brain cancer (glioblastoma, medulloblastoma, or ependymoma), or newly-diagnosed diffuse intrinsic pontine glioma (DIPG). Statistical analysis will stratify patients based on whether their treatment plan includes up-front radiation (or proton) therapy in combination with indoximod. Central review of tissue diagnosis from prior surgery is required, except non-biopsied DIPG. This study will use the "immune-adapted Response Assessment for Neuro-Oncology" (iRANO) criteria for measurement of outcomes. Planned enrollment is up to 140 patients.
The central clinical hypothesis for the GCC1949 study is that inhibiting the pivotal IDO pathway by adding indoximod immunotherapy during chemotherapy and/or radiation is a potent approach for breaking immune tolerance to pediatric tumors that will improve outcomes, relative to standard therapy alone.
This is an NCI-funded (R01 CA229646, MPI: Johnson and Munn) open-label phase 2 trial using indoximod-based combination chemo-radio-immunotherapy for treatment of patients age 3 to 21 years who have progressive brain cancer (glioblastoma, medulloblastoma, or ependymoma), or newly-diagnosed diffuse intrinsic pontine glioma (DIPG). Statistical analysis will stratify patients based on whether their treatment plan includes up-front radiation (or proton) therapy in combination with indoximod. Central review of tissue diagnosis from prior surgery is required, except non-biopsied DIPG. This study will use the "immune-adapted Response Assessment for Neuro-Oncology" (iRANO) criteria for measurement of outcomes. Planned enrollment is up to 140 patients.
Detailed Description:
Disease-specific Cohorts :
Cohort 1A, 1B (closed to enrollment): relapsed or refractory glioblastoma
Cohort 2A, 2B: relapsed or refractory medulloblastoma
Cohort 3A, 3B, 3C: relapsed or refractory ependymoma
Cohort 4C (closed to enrollment): newly-diagnosed DIPG (must have no prior radiation or other therapy)
.
Radiation (or proton) plan sub-cohorts:
Sub-cohort A: for patients not eligible for re-irradiation
Sub-cohort B: for patients who are eligible for partial re-irradiation
Sub-cohort C: for patients who are eligible for full-dose radiation (All newly diagnosed DIPG patients and some relapsed ependymoma patients)
Cohort 1A, 1B (closed to enrollment): relapsed or refractory glioblastoma
Cohort 2A, 2B: relapsed or refractory medulloblastoma
Cohort 3A, 3B, 3C: relapsed or refractory ependymoma
Cohort 4C (closed to enrollment): newly-diagnosed DIPG (must have no prior radiation or other therapy)
.
Radiation (or proton) plan sub-cohorts:
Sub-cohort A: for patients not eligible for re-irradiation
Sub-cohort B: for patients who are eligible for partial re-irradiation
Sub-cohort C: for patients who are eligible for full-dose radiation (All newly diagnosed DIPG patients and some relapsed ependymoma patients)
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| R01CA229646 | NIH | None | https://reporter.nih.gov/quic… | View |