Developing Criteria for Treatment of Neurofibromatosis Type 1 Associated Optic Pathway Glioma

Overview

  • Study type

    Observational
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Site IRB
    • Rochester, Minnesota: 15-006047
    Sponsor Protocol Number: 15-006047

About this study

The purpose of this research study is to understand the natural history of vision in patients with OPG and determine if there are factors (e.g. age at diagnosis, male/female, tumor location, features of the MRI exam, etc) that predict future vision loss or change in tumor size.

Another purpose of the study is to collect and store blood and tissue samples to use for future research to evaluate if there are certain variations in DNA, RNA, or proteins that predict the likelihood of an OPG to grow or cause vision loss.

Participation eligibility

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. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

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

tracts)

3) Prior therapy for an OPG (e.g. surgery [including biopsy], radiotherapy, chemotherapy,

etc.)

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

ventriculostomy).

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

ophthalmology visit.

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

imaging.

Additional VF (Perimetry) Ancillary Study Enrollment Criteria

Goldmann VF testing at study entry will not be required if the child is unable to cooperate.

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Amulya Nageswara Rao, M.B.B.S.

Open for enrollment

Contact information:

Debra Schott CCRP

schott.debra@mayo.edu

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CLS-20313409

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