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PBTC-051: Phase I Study of APX005M in Pediatric CNS Tumors


The main purpose of this study is to determine the best dose and side effects of a new drug called APX005M in treating children and young adult patients with brain cancer that is growing, spreading or getting worse. APX005M can potentially cause a direct cytotoxic (cell-killing) effect on CD40+ tumor cells.  There are two stratums (groups) for this study, however patients with ATRT will only be enrolled into one of the groups (Stratum 1).

Trial Information

There are three parts to this study:

  • Screening–Doctors will review the patient’s medical history to make sure that this study treatment is appropriate.

  • Treatment – Treatment can take up to 24 months if the cancer does not continue to grow, and there are no unacceptable side effects.

  • Follow-up – After treatment ends, patients will be followed up at 30 days after the last treatment was completed, until the tumor starts to grow, or until another treatment is started.



  1. APX005M: Patients will receive APX005M at the assigned dose by the treating doctor once every three weeks through an intravenous (through the vein) infusion.  The infusion will take approximately one hour.

  2. Routine blood and urine tests will be performed prior to enrollment, weekly during the first two courses, prior to each infusion courses 3 through 36, and also at end of treatment.

  3. Routine history and physical exams will continue to take place.

  4. Standard MRI brain with and without contrast will be performed within 3 weeks prior to enrollment, at the end of course 2, every 3 courses through course 14, and then every 4 courses till the end of therapy, and at the time of disease progression or completion of treatment.

Additional Information


Full Details on ClinicalTrials.Gov

This study is available at 11 hospitals throughout the United States.


Inclusion Criteria

  • Patient’s must be 1-21 years of age at the time of  determining study eligibility

  • Recurrent, progressive or refractory ATRT

  • Tumor tissue available (obtained prior to trial enrollment)

  • Patient must have fully recovered from all side effects of all prior therapies.

  • Patients with seizure disorders are eligible if seizures are   well controlled

  • Labs confirming adequate organ and bone marrow function

  • Must have adequate cardiac function (LVEF >50%)

  • Must have adequate pulmonary function (> 93% on room air)

  • Females of childbearing age will need to have a negative pregnancy test

  • Patients of childbearing or child fathering age must agree to use appropriate contraception

Exclusion Criteria

  • Use of other anticancer or investigational medications

  • Significant medical disorders (ex. Serious infections, significant cardiac, pulmonary hepatic, psychiatric, GI, or other organ issues)

  • Presence of bulky tumor on imaging

  • History of severe reaction to a monoclonal antibody

  • Patients that have received a allogeneic hematopoietic stem cell transplant are not eligible

  • Patients with a bleeding disorder are not eligible

  • Females that are pregnant or breastfeeding

Contact Information

Study Chair: Ira Dunkel, MD

Phone: (212) 639-2153


Secondary Contact: Shujie Han

Phone: (901) 595-4877


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