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Neurofibromatosis Clinical Trials

A listing of Neurofibromatosis medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (8) clinical trials

A clinical trial sponsored by National Cancer Institute (NCI) for a research study for the treatment of Optic Nerve Glioma, von Recklinghausen's Disease, Plexiform Neurofibroma, Nerve Sheath Tumors, neurofibroma

BACKGROUND Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas (PN), dermal neurofibromas, optic pathway tumors, brain tumors, malignant peripheral nerve sheath tumors ...

Phase N/A

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

Background NF1 is an autosomal dominant genetic disorder characterized by distinct features including the development of benign plexiform neurofibromas (PN) and malignant peripheral nerve sheath tumors (MPNST) tumors of the nervous system. Development of MPNST typically results from malignant transformation in a preexisting PN. Associated symptoms may overlap and be ...


A clinical trial sponsored by National Cancer Institute (NCI) for a research study of Selumetinib (AZD6244 hyd sulfate)

BACKGROUND Gastrointestinal stromal tumor (GIST) is the most common mesenchymal neoplasm of the gastrointestinal tract, and traditional cytotoxic chemotherapy is not effective. Patients with Neurofibromatosis 1 (NF1) have an increased risk of developing GIST, and surgery remains the only standard treatment option for NF1-related GIST. While the tyrosine kinase inhibitors ...


A Phase 2 clinical study for patients using Trametinib

PRIMARY OBJECTIVES: I. To determine the objective response rate to trametinib in children with recurrent or refractory juvenile myelomonocytic leukemia (JMML). SECONDARY OBJECTIVES: I. To further define and describe the toxicities of single agent trametinib in children with recurrent or refractory JMML. II. To further characterize the pharmacokinetics of trametinib ...


A clinical trial seeking patients for a research study for the treatment of BRAF V600E Mutation Analysis, von Recklinghausen's Disease, Recurrent Childhood Visual Pathway Glioma, Glioma, Recurrent Childhood Pilocytic Astrocytoma

PRIMARY OBJECTIVES: I. To estimate the maximum tolerated dose (MTD) or recommend a Phase II dose of AZD6244 (selumetinib) in children with recurrent or refractory low-grade glioma. (Phase I, completed as of April 29, 2013) II. To describe the toxicity profile and define the dose limiting toxicity of AZD6244 in ...


A clinical trial seeking patients for a research study of Binimetinib

Patients 1 year with progressive NF1 and PN(s) by serial imaging or causing significant morbidity and that can be analyzed by volumetric MRI are eligible for this study. Initially, the study will open to adult patients,18 years and older, (Stratum A). The pediatric patients (Statum B) will be receive the ...


Relationship Stressors in Parents of Children With Cancer or Neurofibromatosis Type 1 (NF1)

Background Parents face numerous stressors when their child is diagnosed with cancer or NF1, each of which can strain a marriage/partnership. Marital/partner relationship dynamics are often not assessed or addressed when providing health care for children with cancer or NF1. Many parents struggle to effectively cope with the changes in ...

Phase N/A

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)

Background Neurofibromatosis 1 (NF1) is a genetic disease with multiple clinical manifestations, including plexiform neurofibromas (PN) that can cause pain and may significantly impact daily functioning and quality of life (QOL). Patient-reported outcomes (PROs) are useful in trials for conditions that are disabling and chronic like NF1, where symptom reduction ...

Phase N/A