Clinical Studies and Translational Research in the Area of Neurofibromatosis
Examples of Research Projects
PI: A pilot, phase II, open-label clinical trial of pirfenidone (an antifibrotic agent) in adult patients with Neurofibromatosis type I and plexiform and paraspinal neurofibromas (PI). The study was coordinated through the Mayo Clinic Cancer Center.
Co-PI: "Phase I Clinical trial of Pirfenidone in Childen and Adolescents with plexifom neurofibromas." Study was a part of multi-center clinical trial, coordinated by NCI.
Co-PI: "Phase II Clinical Trial of Pirfenidone in Children, Adolescents, and Young Adults with Neurofibtromatosis Type I and Progressive Plexiform Neurofibromas," coordinated by NCI, in collaboration with Childrens National Medical Center (PI Dr. Roger Packer).
Protocols in Development:
PI: "A Phase-2 Study of AZD2171 in Adult Patients with Neurofibromatosis Type I and Extensive Plexiform and Paraspinal Neurofibromas," coordinated by Cancer Therapy Evaluation Program (CTEP) and sponsored by AstraZeneca Pharmaceuticals; multicenter protocol with Mayo Clinic lead.
My research also involves several collaborative basic research studies in the field of neurofibromatosis, in conjunction with Mayo's Neuro-Oncology program, Radiology Department, and Neurosurgery Department.
My research interests include overgrowth syndromes, as well as various genetic syndromes and metabolic disorders.