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Neurofibromatosis clinical trials at UC Cancer

4 research studies open to eligible people

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  • A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

    open to eligible people ages 2-21

    This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatine/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.

    at UCSF

  • Experimental MEK Inhibitor Mirdametinib (PD-0325901) for Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

    open to eligible people ages 2 years and up

    This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).

    at UC Davis UCLA

  • Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

    open to eligible people ages 1 year and up

    This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.

    at UCLA UCSD UCSF

  • Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

    open to eligible people ages 1 month to 21 years

    This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back or does not respond to treatment. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

    at UCSF

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