Experimental antibody APX005M takes on childhood brain cancers
NCT ID NCT03389802
First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 3 times
Summary
This early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to find a safe dose and see how well children tolerate the treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- APX005M (an experimental antibody that activates the immune system)
- What this could lead to
- If successful, this could lead to a new treatment option for children with hard-to-treat brain tumors by helping their immune system fight the cancer.
- What could go wrong
- This is a very early (Phase 1) trial with only 32 participants, so it is primarily testing safety and dosing. The drug may not shrink tumors or improve survival, and side effects like cytokine release syndrome are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Children Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90026, United States
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Childrens National Medical Center
Washington D.C., District of Columbia, 20010-2970, United States
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Cincinnati Children Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Lucile Packard Children Hospital Stanford University
Palo Alto, California, 94304, United States
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Lurie Childrens Hospital-Chicago
Chicago, Illinois, 60614, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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St. Jude Children Research Hospital
Memphis, Tennessee, 38105, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can trained immune cells outsmart brain tumors?
- Can the Body's own immune cells beat a recurrent brain tumor?
- Could a parasite drug fight childhood brain tumors?
- Can AI read tumor slides to outsmart childhood cancer?
- Could an antiparasitic drug boost radiation against childhood brain tumors?
- Can mapping the Blood-Brain barrier in brain tumors unlock new clues?