Clinical trialNCT03389802CompletedPhase 1Interventional

Phase I Study of APX005M in Pediatric Central Nervous System Tumors

SponsorPediatric Brain Tumor ConsortiumEnrollment32CNS Primary Tumor, Not Otherwise Specified (NOS)Diffuse Intrinsic Pontine Gliomas (DIPG)Ependymoma, Not Otherwise Specified (NOS)Glioblastoma MultiformeHigh-grade Astrocytoma Not Otherwise Specified (NOS)Medulloblastoma

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor that is growing, spreading, or getting worse (progressive), or newly diagnosed diffuse intrinsic pontine glioma. APX005M can trigger activation of B cells, monocytes, and dendritic cells and stimulate cytokine release from lymphocytes and monocytes. APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Ira Dunkel
Memorial Sloan Kettering Cancer Center
Study Chair
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

11 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Children'S Hospital Los Angeles
Los Angeles, California, United States
Lucile Packard Children Hospital Stanford University
Palo Alto, California, United States
Children'S Hospital Colorado
Aurora, Colorado, United States
Childrens National Medical Center
Washington D.C., District of Columbia, United States
Children'S Healthcare of Atlanta
Atlanta, Georgia, United States
Lurie Childrens Hospital-Chicago
Chicago, Illinois, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Cincinnati Children Hospital Medical Center
Cincinnati, Ohio, United States
Children Hospital of Pittsburgh of Upmc
Pittsburgh, Pennsylvania, United States
St. Jude Children Research Hospital
Memphis, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

APX005M treatment for newly diagnosed DIPG patientsAPX005M treatment for recurrent or refractory primary malignant CNS tumor patients

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 1 Mar 2018
Primary completion: 30 Sept 2023
Completion: 20 Jul 2026
Last update posted: 13 Aug 2026
Registered enrollment: 32
Registry id: NCT03389802

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.