CBL Syndrome clinical trials at UCSF

1 research study open to eligible people

CBL syndrome is a rare genetic disorder that affects the blood. UCSF is studying a drug combo of trametinib and azacitidine in newly diagnosed juvenile myelomonocytic leukemia. Researchers will record side effects and responses to treatment.

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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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