Natural History of NF1-Associated Non-Optic Pathway Glioma

Children with NF1 are at increased risk for the development of brain tumors within or outside of the visual pathway. While much research has been done about brain tumors located in the visual pathway, little is known about the natural history of NF1-associated brain tumors that occur outside of the visual pathway. There are currently no evidence-based, “standard of care” guidelines for the screening, monitoring, and treatment of these tumors. In addition, little is known about risk factors for more aggressive tumor behavior. Finally, the long-term outcomes of patients with these NF1-associated brain tumors have not been previously described. To develop these guidelines and improve the management and outcomes of patients with these tumors, a natural history study of children with NF1 and brain tumors outside the visual pathway is needed. This study will enroll at least 270 children with NF1-associated brain tumors. The participants will undergo regular magnetic resonance imaging and physical examinations, in addition to evaluations of quality of life and long-term physical functioning. Successful completion of this study will allow for the establishment of evidence-based guidelines for surveillance and treatment and ultimately improve outcomes for children with NF1 and brain tumors outside of the visual pathway.

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