New drug cocktail shows promise for tough childhood brain cancers
NCT ID NCT04201457
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial is testing whether adding hydroxychloroquine to two targeted cancer drugs (dabrafenib and trametinib) can help children and young adults with certain brain tumors that have come back or not responded to treatment. The study includes patients with specific genetic changes (BRAF mutations or fusions) or neurofibromatosis type 1. The goal is to find the best dose and see if the combination shrinks tumors or slows their growth.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- dabrafenib, trametinib, and hydroxychloroquine
- What this could lead to
- If it works, this could offer a new treatment option for children with certain brain tumors that have not responded to standard therapy.
- What could go wrong
- This is an early-phase trial with only 57 participants, so results may not apply to all patients. Adding hydroxychloroquine may also increase side effects.
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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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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Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Children's 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's Hospital at Stanford University Medical Center
Palo Alto, California, 94304, United States
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Lurie Children's 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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National Cancer Institute Pediatric Oncology Branch
Bethesda, Maryland, 20892, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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St. Jude Children Research Hospital
Memphis, Tennessee, 38105, United States
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Texas Children's Cancer Center
Houston, Texas, 77030, United States
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University of Florida
Gainesville, Florida, 32608, United States
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