The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
This is an open-label, single ascending dose study of EDIT-101 in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Up to 34 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of EDIT-101 in this study. EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
34
Participants will receive a single dose of EDIT-101 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study.
Bascom Palmer Eye Institute
Miami, Florida, United States
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
W.K. Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States
Casey Eye Institute - OSHU
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Frequency of Adverse Events related to EDIT-101
Time frame: 1 year
Number of participants experiencing procedural related adverse events
Time frame: 1 year
Incidence of dose limiting toxicities
Time frame: 1 year
Maximum tolerated dose as determined by occurrence of dose limiting toxicities
Time frame: 1 year
Change from baseline in Mobility course score
Testing the subjects visual function by having the subject walk through obstacle courses. Courses will have different levels of difficulty depending on the light levels of the room and the contrast of the objects in the room.
Time frame: 1 year
Change from baseline in LogMAR measurement of BCVA
The test will evaluate visual acuity in ranges from light perception to normal vision.
Time frame: 1 year
Change from baseline in pupillary response
Measuring the change in pupil diameter in response to a light stimulus.
Time frame: 1 year
Change from baseline in dark adapted visual sensitivity using Full field light sensitivity threshold (FST)
Flashes of light of varying luminance are presented to the eye and the subject reports is the flash was seen.
Time frame: 1 year
Change from baseline in macula thickness
Time frame: 1 year
Change from baseline in contrast sensitivity
The Lea symbols chart will be used for subjects under age 6 and the Pelli-Robson chart for all other subjects. The images or letters on the charts are in decreasing contrast.
Time frame: 1 year
Change from baseline in macular sensitivity as measured by microperimetry
Visual field test measuring the amount of light perceived in specific parts of the macula.
Time frame: 1 year
Change from baseline in color vision score using the Farnsworth 15 score
The Farnsworth D15 tests for congenital and acquired color vision defects. Fifteen color discs will be arranged by the subject. Scoring is accomplished by recording the sequence selected by the patient on a copy of the score sheet. A patient with a color vision deficiency will arrange the color discs in a different order than a person with normal color vision.
Time frame: 1 year
Change from baseline in QOL score for Age <8 years using the Children's Visual Function Questionnaire
Time frame: 1 year
Change from baseline in QOL score for Age 8 to <18 years using the Impact of Vision Impairment for Children
Time frame: 1 year
Change from baseline in QOL score for Age >18 years if BCVA is worse than 1.0 logMAR in both eyes using the Impact of Vision Impairment for Very Low Vision
Time frame: 1 year
Change from baseline in QOL score for Age >18 years if BCVA is 1.0 logMAR or better in both eyes using the Impact of Vision Impairment
Time frame: 1 year
Change from baseline in visual field using kinetic perimetry
Kinetic perimetry looks as the visual field to identify regions of normal and abnormal sensitivity to light
Time frame: 1 year
Change from baseline in Patient Global Impressions of Change score
This QOL has 5 non-numeric choices for the subject to select how they believe their condition has changed.
Time frame: 1 year
Change from baseline in gaze tracking
Video clips of the eyes are used to measure eye position and stability over time.
Time frame: 1 year
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