Retrospective Study of the Clinical Features and Outcomes of Children and Adults with NF1-Associated High Grade Glioma and Adults with NF1-Associated Low Grade Glioma

Children and adults with NF1 are at increased risk for the development of brain tumors, ranging from benign, slow growing tumors to malignant, life-threatening tumors. Malignant tumors occur more frequently in patients with NF1 compared to the general population. Despite increased risk for these aggressive tumors, there are very few studies examining risk factors for the development of these tumors among patients with NF1. Further, the optimal treatment of these tumors and long-term outcomes are unknown due to a lack of large research studies. An increased understanding of risk factors for tumor development and the approaches to and outcomes of treatment is necessary to improve the care of patients with NF1 who develop brain tumors. In addition, a greater understanding of the molecular alterations in the tumors is needed to advance treatment outcomes and drug development. This study will address significant knowledge gaps by conducting a large study of greater than 100 adults and children with NF1 and malignant brain tumors and 50 adults with NF1 and low-grade brain tumors.

Investigators

Dr. Chelsea Kotch

Children's Hospital of Philadelphia

Dr. Chelsea Kotch

Children's Hospital of Philadelphia

Current Stage

Clinical Trial Phase 1

Discovery
In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3