CBL Syndrome clinical trials at UCSF

1 research study open to eligible people

CBL syndrome is a disorder caused by changes in a specific gene that affects blood cell production. UCSF is testing a new treatment using trametinib and azacitidine for juvenile myelomonocytic leukemia. This study aims to determine how safe and effective these medications are when used together.

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  • Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

    open to eligible people ages 1 month to 21 years

    This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.

    San Francisco, California and other locations

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