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OCT may have role in treatment of neurofibromatosis type 1


The Gilbert Family Foundation is collaborating with the Children’s Oncology Group and Children’s Hospital of Philadelphia to validate a tool to measure progressive vision loss.

The Gilbert Family Foundation (GFF) announced the launch of a clinical study that aims to validate optical coherence tomography (OCT) as a tool to objectively assess the visual system and its response to treatment in neurofibromatosis type 1 (NF1) patients with optic pathway gliomas.

NF1 is a multisystem genetic disorder characterized primarily by the growth of benign and malignant nervous system tumors. Approximately 1 in 5 NF1 patients develop a tumor in their visual system called an optic pathway glioma, causing many of them to lose their sight.

According to the foundation, clinicians typically base their decision on whether to treat a patient with an NF1 optic pathway glioma on changes in visual acuity, which is highly dependent on patient cooperation. This becomes particularly challenging with younger patients, meaning there is a great need for a more reliable, quantitative biomarker for vision loss.

Led by Robert Avery, MD, and Michael Fisher, MD, at Children’s Hospital of Philadelphia (CHOP), this study is an exploratory aim of the ongoing Children’s Oncology Group Phase III randomly assigned clinical trial ACNS1831 ( NCT03871257 ).

According to the foundation, this trial compares the treatment response of a new targeted therapy (selumetinib) to the present standard of care (carboplatin and vincristine) in children with newly diagnosed or previously untreated NF1 low-grade gliomas, including NF1 optic pathway gliomas.

The clinical study will collect three sets of OCT measurements for 60 NF1 optic pathway glioma patients enrolled in the Phase III trial: pre-treatment and after 6 and 12 months of receiving treatment.

The foundation noted that Avery and Fisher will then determine whether OCT measurements can predict treatment response prior to treatment initiation or provide early indication of treatments that are not working.

Previous studies have established the relationship between the data collected with OCT and visual acuity and visual field in children with optic pathway gliomas, suggesting that OCT could serve as a technique to predict progressive vision loss. This evidence could greatly support the clinical decision on when to treat a patient and prevent patients from receiving unnecessary treatment.

According to Avery, understanding how OCT metrics change during this trial will provide information to understand how they can optimize treatment outcomes and their role in clinical practice.

“Depending on the outcomes of the primary study, our OCT results could elucidate important factors about why children’s visual outcomes may differ,” he said in a statement. “This ancillary study would also help us justify OCT’s inclusion as a secondary outcome in future clinical trials.”

ACNS1831, formally titled “Quantitative Ophthalmic Biomarkers of NF1 Associated Optic Pathway Gliomas in a Phase 3 Clinical Trial,” is sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health, and supported by AstraZeneca under a Cooperative Research and Development Agreement between NCI and AstraZeneca.

This study will also be supported as part of GFF’s Vision Restoration Initiative. In April 2019, GFF announced an initial investment of $11 million in its Vision Restoration Initiative to fund research focused on developing innovative therapies that either repair or replace damaged visual systems caused by NF1-associated optic pathway gliomas. In parallel with the primary clinical trial, this study will take place over the course of six years.

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