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Indoximod

Phase 2

Glioblastoma | Small molecule | Oncology |Johnson & Johnson|Last Updated: Jan 9, 2026

Target and mechanism

Molecular targetMTOR, RPTOR, MLST8
Target classActivator
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment130

FDA Designations

No designations recorded

Clinical trial landscape

Indoximod · 2 trials · 5 indications

Phase 2 1Phase 1 1
NCT04049669Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPGGlioblastoma
ACTIVE NOT_RECRUITING130 Analytics
PHASE2ACTIVE NOT_RECRUITING
Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG
GlioblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

8-month iRANO-PFS (Progression-Free Survival, defined by immune-adapted iRANO criteria)
Up to 5 years

For patients with relapsed glioblastoma, medulloblastoma, or ependymoma.

12-month Overall Survival (OS)
Up to 5 years

For patients with newly diagnosed DIPG (diffuse intrinsic pontine glioma).

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

Secondary Endpoints

Median Overall Survival (OS)
Up to 5 years
Median iRANO-PFS (Progression-Free Survival, defined by immune-adapted iRANO criteria)
Up to 5 years
Median Time to Regimen Failure (TTRF)
Up to 5 years
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Core Regimen, sub-cohort AEXPERIMENTALFor patients not eligible for re-irradiation; Start with Core Regimen chemo-immunotherapy (indoximod with oral temozolomide).
Core Regimen, sub-cohort BEXPERIMENTALFor patients who are eligible for partial re-irradiation; Start with indoximod plus up-front re-irradiation, using a palliative low-dose or partial-field radiation plan (low-dose radiation or not all disease sites included); Followed by Core Regimen chemo-immunotherapy (indoximod with oral temozolomide).
Core Regimen, sub-cohort CEXPERIMENTALFor patients who are eligible for full-dose radiation; (All newly diagnosed DIPG patients and some relapsed ependymoma patients); Start with indoximod plus up-front radiation, using a palliative full-dose radiation plan to all known sites of disease (\>50 Gy to brain, \>45 Gy to spine); Followed by Core Regimen chemo-immunotherapy (indoximod with oral temozolomide).
Salvage Regimen 1EXPERIMENTALFor patients who wish to continue access to indoximod after progression on the Core Regimen; Cross-over to indoximod with oral metronomic cyclophosphamide and etoposide).
Salvage Regimen 2EXPERIMENTALFor patients who wish to continue access to indoximod after progression on the Core Regimen; Cross-over to indoximod with oral lomustine and temozolomide).
Regimen AEXPERIMENTALPatients will be treated with ibrutinib plus indoximod, cyclophosphamide, and etoposide. Cycles are a minimum of 28 days.
Regimen BEXPERIMENTALPatients will be treated with ibrutinib plus indoximod and temozolomide. Cycles are a minimum of 28 days.

Interventions

NameTypeDescription
IndoximodDRUGIndoximod will be taken by mouth twice daily during radiation and throughout each chemo-immunotherapy treatment cycle.
Partial RadiationRADIATIONPalliative low-dose or partial-field radiation plan (low-dose radiation or not all disease sites included).
Full-dose RadiationRADIATIONPalliative full-dose radiation plan to all known sites of disease (\>50 Gy to brain, \>45 Gy to spine).
TemozolomideDRUGTemozolomide will be taken by mouth once daily, on days 1-5 of each chemo-immunotherapy treatment cycle.
CyclophosphamideDRUGCyclophosphamide will be taken by mouth once daily, on days 1-21 of each chemo-immunotherapy treatment cycle.
EtoposideDRUGEtoposide will be taken by mouth once daily, on days 1-21 of each chemo-immunotherapy treatment cycle.
LomustineDRUGLomustine will be taken by mouth once daily, on day 1 of each chemo-immunotherapy treatment cycle.
IbrutinibDRUGFor Regimen A, Ibrutinib will be taken by mouth once daily, on days 1-21 of each treatment cycle.
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Eligibility Criteria

Age Range3 Years to 21 Years
SexALL
Healthy VolunteersNo
Study Sites5

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 ...

Countries:United States
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Frequently asked questions about Indoximod

What is Indoximod used for?

Indoximod is an investigational small molecule being studied for the treatment of pediatric brain tumors, including ependymoma and glioblastoma. It is also being evaluated in medulloblastoma and diffuse intrinsic pontine glioma. The drug is in clinical development and has not been approved by the FDA.

What does Indoximod target?

Indoximod is an investigational small molecule that targets the IDO pathway, which is involved in immune suppression in the tumor microenvironment. By modulating this pathway, Indoximod is being studied to enhance the immune system's ability to fight cancer cells in pediatric brain tumors.

Who makes Indoximod?

Indoximod is being developed by Johnson & Johnson (NYSE: JNJ). The company is conducting clinical trials to evaluate the drug's safety and efficacy in pediatric patients with brain tumors.

What phase is Indoximod in?

Indoximod is in Phase 1 clinical development. It is being studied in combination with ibrutinib for pediatric brain cancer. The drug is investigational and has not received FDA approval.

What clinical trials is Indoximod in?

Indoximod is being studied in two clinical trials. NCT04049669 is a Phase 2 trial of Indoximod with chemotherapy and radiation for relapsed brain tumors or newly diagnosed DIPG. NCT05106296 is a Phase 1 trial of chemo-immunotherapy using ibrutinib plus Indoximod for pediatric brain cancer.

Is Indoximod the same as other drugs?

Indoximod is a distinct investigational drug and is not known to be the same as any other approved medication. It is being studied under its own name in clinical trials for pediatric brain tumors.