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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

diffuse low-grade glioma, MAPK pathway–altered glioma low grade glioma malignant glioma neurofibromatosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90026, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010-2970, United States

  • Cincinnati Children Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Lucile Packard Children's Hospital at Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • Lurie Children's Hospital-Chicago

    Chicago, Illinois, 60614, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • National Cancer Institute Pediatric Oncology Branch

    Bethesda, Maryland, 20892, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • St. Jude Children Research Hospital

    Memphis, Tennessee, 38105, United States

  • Texas Children's Cancer Center

    Houston, Texas, 77030, United States

  • University of Florida

    Gainesville, Florida, 32608, United States