Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Inclusion Criteria (for all subjects)
1) NF1: All subjects must have EITHER the clinical diagnosis of NF1 using the NIH
Consensus Conference criteria,55 OR have a constitutional NF1 mutation documented in a
CLIA/CAP certified lab.
2) Age: <18 years of age at the time of study enrollment.
3) Tumor: Newly diagnosed OPG (confirmed by MRI within 1 month of enrollment). The
baseline MRI must be sent to the operations center within 2 weeks of enrollment for
central review. If central review determines that OPG is not present, then the
subject will be taken off-study and deemed ineligible.
Exclusion Criteria (for all subjects)
1) Diagnosis of OPG (by MRI) > 1 month prior to enrollment
2) OPGs involving only the optic radiations (i.e. no involvement of the nerve, chiasm, or
3) Prior therapy for an OPG (e.g. surgery [including biopsy], radiotherapy, chemotherapy,
4) Prior therapy for another (non-OPG) tumor: Prior systemic chemotherapy or prior cranial
radiotherapy will be excluded. All other prior tumor-directed therapies must be
discussed with one of the study chairs to determine potential eligibility.
5) Ophthalmologic or neurologic condition (e.g. orbital-temporal neurofibroma, glaucoma,
etc.) other than OPG presently affecting or having previously impacted vision except for
refractive error and amblyopia.
6) Ophthalmologic or neurologic conditions (e.g. orbital plexiform neurofibroma) other than
OPG that could potentially affect vision must be discussed with one of the study chairs
to determine potential eligibility.
7) History of hydrocephalus requiring surgical intervention (e.g., shunt or endoscopic third
Additional OCT Ancillary Study Enrollment Criteria
OCT at baseline when a subject first enters the study is recommended but not required. This is
to accommodate subjects who were not known to have an OPG prior to initial study
Additional DTI Ancillary Study Enrollment Criteria
DTI imaging at baseline when a subject first enters the study is recommended but not required.
This is to accommodate subjects who were not known to have an OPG prior to initial MR
Additional VF (Perimetry) Ancillary Study Enrollment Criteria
Goldmann VF testing at study entry will not be required if the child is unable to cooperate.