Neurofibromatosis clinical trials at UCSF

6 in progress, 3 open to eligible people

Neurofibromatosis is a disease that leads to tumors on nerve tissue. UCSF is studying a new treatment for juvenile leukemia using trametinib and azacitidine. Another trial is comparing selumetinib to standard drugs for brain tumors. UCSF also aims to find genetic factors in skin tumors in adults with Neurofibromatosis Type 1.

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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

  • 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 carboplatin/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.

    San Francisco, California and other locations

  • Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults with Neurofibromatosis Type 1

    open to eligible people ages 40 years and up

    The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1). This will allow for improve understanding of NF1 pathogenesis and more personalized disease management. The investigators will conduct a GWAS analysis to identify common genetic risk variants associated with the development of cutaneous neurofibromas.

    Redwood City, California and other locations

  • Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

    Sorry, in progress, not accepting new patients

    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.

    Oakland, California and other locations

  • Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

    Sorry, in progress, not accepting new patients

    This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory). Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

    San Francisco, California and other locations

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

    Sorry, in progress, not accepting new patients

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

    San Francisco, California and other locations

Our lead scientists for Neurofibromatosis research studies include .

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