NCT02502708

Study of the IDO Pathway Inhibitor, Indoximod, and Temozolomide for Pediatric Patients With Progressive Primary Malignant Brain Tumors

A Phase I Trial of Indoximod and Temozolomide-Based Therapy for Children With Progressive Primary Brain Tumors

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 81 (actual)
• Sponsor: NewLink Genetics Corporation · Industry
• Sex: All
• Age: 3 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

This is a first-in-children phase 1 trial using indoximod, an inhibitor of the immune "checkpoint" pathway indoleamine 2,3-dioxygenase (IDO), in combination with temozolomide-based therapy to treat pediatric brain tumors. Using a preclinical glioblastoma model, it was recently shown that adding IDO-blocking drugs to temozolomide plus radiation significantly enhanced survival by driving a vigorous, tumordirected inflammatory response. This data provided the rationale for the companion adult phase 1 trial using indoximod (IND#120813) plus temozolomide to treat adults with glioblastoma, which is currently open (NCT02052648). The goal of this pediatric study is to bring IDO-based immunotherapy into the clinic for children with brain tumors. This study will provide a foundation for future pediatric trials testing indoximod combined with radiation and temozolomide in the up-front setting for patients with newly diagnosed central nervous system tumors.

Conditions

• Glioblastoma Multiforme
• Glioma
• Gliosarcoma
• Malignant Brain Tumor
• Ependymoma
• Medulloblastoma
• Diffuse Intrinsic Pontine Glioma
• Primary CNS Tumor

Interventions

Type	Name	Description
DRUG	Indoximod	Indoximod will be administered orally twice daily.
DRUG	Temozolomide	Temozolomide will be administered on days 1-5 of every 28 day cycle.
RADIATION	Conformal Radiation	Conformal radiation will be administered on days 3-7 of induction cycle.
DRUG	Cyclophosphamide	Cyclophosphamide will be administered orally daily.
DRUG	Etoposide	Etoposide will be administered orally daily.

Timeline

• Start date: 2015-10-01
• Primary completion: 2019-12-12
• Completion: 2020-02-28
• First posted: 2015-07-20
• Last updated: 2020-06-04

Locations

5 sites across 1 country: United States