AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa

Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02556736

Enrollment

Registered

2015-09-22

Start date

2015-12-14

Completion date

2024-10-21

Last updated

2026-01-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Advanced Retinitis Pigmentosa

Keywords

Gene therapy, Optogenetics, Channelrhodopsin, Retina, Retinitis pigmentosa

Brief summary

All participants in phase 1 and phase 2a had hand motion visual acuity or worse. If efficacy was demonstrated from phase 1, better vision subjects could be enrolled; however, efficacy was not demonstrated.

Detailed description

Study RST-001-CP-0001 was an open-label, dose-escalation study to evaluate the safety and tolerability of AGN-151597 (formerly RST-001) administered as a single intravitreal injection in participants with advanced RP. Three groups of approximately 3 participants each were sequentially enrolled in the dose-escalation phase (Phase 1) of this study: Group A (low dose), Group B (mid dose), and Group C (high dose). For each dose group, the safety and tolerability of AGN-151597 was assessed by a data safety monitoring committee (DSMC) in the first participant before the remaining participants were enrolled into the group. If the DSMC considered the safety and tolerability of all participants in the dose group to be satisfactory and enrollment stopping rules had not been met, then enrollment into the next dose group could begin. If the DSMC considered the safety and tolerability satisfactory and the enrollment stopping rules had not been met after a minimum assessment of 1 month (to include the Month 1 Visit) from treatment of the final participant in Groups A, B, or C, then the sponsor could elect to start enrollment of up to approximately 12 participants in Phase 2a to receive AGN-151597 at the maximum tolerated dose. After completion of the 2-year core study visits, each participant could enroll in a long-term follow-up for an additional 3 years to monitor the long-term safety of AGN-151597.

Interventions

DRUGAGN-151597

AGN-151597 is a gene therapeutic delivered by intravitreal injection

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

Participants must meet all of the following criteria. 1. Age \>= 18 years. 2. Signed and dated written informed consent obtained from the patient. 3. Ability to comply with testing and all protocol tests.

Exclusion criteria

Any one of the following will exclude patients from being enrolled into the study: 1. Unable or unwilling to meet requirements of the study. 2. Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months.

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597	Baseline (Day 1) to 6 Months	An adverse event is any untoward medical occurrence in a subject or clinical investigation patient administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Visual Acuity in the Study Eye at Baseline and Month 6	Baseline (Day 1), 6 Months	Visual acuity of the study eye (with fellow eye covered) was measured using low vision assessment of count fingers, hand motion, and light perception. For count fingers testing, the examiner's hand presenting 1, 2, or 5 fingers is held 2 feet in front of the eye being examined. If the participant correctly identifies three of five presentations, then count fingers vision is noted. If not, then the participant must be tested for hand motion vision. For hand motion testing, the examiner's hand is extended 2 feet in front of the eye and moved horizontally or vertically. If the participant correctly identifies hand movement four out of five times, then hand motion vision is noted. If not, then the participant is tested for light perception. For light perception testing, a beam of light is directed in and out of the eye at least four times from a distance of 3 feet. If the participant correctly perceives the light, vision should be recorded as yes to light perception.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold	Baseline (Day 1) to Month 6	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold	Baseline (Day 1) to Month 6	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold	Baseline (Day 1) to Month 6	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Month 6 in Ambulation in the Study Eye (Time)	Baseline (Day 1) to Month 6	Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).
Change From Baseline at Month 6 in Ambulation in the Study Eye (Distance)	Baseline (Day 1) to Month 6	Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).
Intraocular Pressure (IOP) Measurements in the Study Eye	Baseline (Day 1), 6 Months	Intraocular pressure was measured using the Goldmann applanation tonometer or a hand-held tonometer (same instrument used for each participant throughout the study, when possible). Measurements were taken at baseline (pre-injection) and at 6 months.
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Baseline (Day 1), 6 Months	A standardized procedure for the collection of single, non-stereo images of the fundus of both eyes was obtained using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Any changes from baseline in fundus autofluorescence were also documented.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Nerve Fibre Layer Volume	Baseline (Day 1) to Month 6	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Ganglion Cell and Inner Plexiform Layer Volume	Baseline (Day 1) to Month 6	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Volume	Baseline (Day 1) to Month 6	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Thickness	Baseline (Day 1) to Month 6	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Secondary

Measure	Time frame	Description
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude)	Baseline (Day 1) to Months 6 and 24	Full field electroretinography based on standards set by the ERG Standardization Committee of the International Society for Clinical Electrophysiology of Vision (ISCEV) was performed at the Screening visit, 6 months, and at 24 months. ERG tests how well the light sensitive part of the eye (retina) is working. Several assessments were performed to evaluate how well different parts of the retina respond to light in different settings. Amplitude is the strength of the electrical signal that the retina produces in response to light during an ERG test. Higher amplitude in A-wave or b-wave indicates improvement; lower amplitude indicates worsening.
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency)	Baseline (Day 1) to Months 6 and 24	Full field electroretinography based on standards set by the ERG Standardization Committee of the International Society for Clinical Electrophysiology of Vision (ISCEV) was performed at the Screening visit, 6 months, and at 24 months. ERG tests how well the light sensitive part of the eye (retina) is working. Several assessments were performed to evaluate how well different parts of the retina respond to light in different settings. Amplitude is the strength of the electrical signal that the retina produces in response to light during an ERG test. Latency is the time it takes for the retina to respond after a light is flashed in the eye during an ERG test. Shorter latency indicates an improvement; longer latency indicates a worsening.
Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores	Baseline (Day 1) to Months 3, 6, and 24	Change in quality of life, based on composite scores of the NEI VFQ-25 from Baseline at 3, 6, and 24 months. The NEI VFQ-25 consists of 25 vision-targeted questions that represent 11 vision-related quality of life subscales and one general health item. The 11 subscales are general vision, difficulty with near vision activities, difficulty with distance vision activities, limitation in social functioning due to vision, role limitation due to vision, dependency on others due to vision, mental health symptoms due to vision, driving difficulties, limitation with peripheral vision, limitation with color vision, and ocular pain. NEI VFQ-25 scores range from 0 to 100, with a higher score representing better functioning. A positive value change from baseline indicates an improvement in vision-related quality of life.
Visual Acuity in the Study Eye at Months 3, 12, and 24	3, 12, and 24 Months	Visual acuity of the study eye (with fellow eye covered) was measured using low vision assessment of count fingers, hand motion, and light perception. For count fingers testing, the examiner's hand presenting 1, 2, or 5 fingers is held 2 feet in front of the eye being examined. If the participant correctly identifies three of five presentations, then count fingers vision is noted. If not, then the participant must be tested for hand motion vision. For hand motion testing, the examiner's hand is extended 2 feet in front of the eye and moved horizontally or vertically. If the participant correctly identifies hand movement four out of five times, then hand motion vision is noted. If not, then the participant is tested for light perception. For light perception testing, a beam of light is directed in and out of the eye at least four times from a distance of 3 feet. If the participant correctly perceives the light, vision should be recorded as yes to light perception.
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume	Baseline to Months 3, 12, and 24	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at Months 3, 12, and 24 as Measured by Spectral Domain-OCT (SD-OCT).
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Total Retinal Thickness	Baseline to Months 3, 12, and 24	Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at Months 3, 12, and 24 as Measured by Spectral Domain-OCT (SD-OCT).
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Months 3 and 24	A standardized procedure was used for the collection of single, non-stereo images of the fundus of both eyes using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Presence or absence of changes from baseline in fundus autofluorescence were also documented.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold	Baseline (Day 1) to Months 3, 12, and 24	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold	Baseline (Day 1) to Months 3, 12, and 24	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold	Baseline (Day 1) to Months 3, 12, and 24	The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time)	Baseline (Day 1) to Months 3, 12, and 24	Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Distance)	Baseline (Day 1) to Months 3, 12, and 24	Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores	Baseline (Day 1) to Months 3, 6, and 24	Participants were to perform two tests concurrently, first identifying if a light displayed on an LED screen can be seen and then if a series of standard images (square, circle, triangle, or star) presented on the screen can be identified. The shapes were presented in blue or red at varying intensities. Data on light color and threshold intensity of the light was collected; shape detection data was not collected. The change from baseline in threshold intensity at 3, 6, and 24 months is reported. A negative change from baseline suggests an improvement.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency)	Baseline (Day 1) to Months 3, 6, and 24	Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Countries

United States

Participant flow

Recruitment details

A total of 14 participants were enrolled in the study in the United States.

Pre-assignment details

The Safety Population included all enrolled participants who received at least 1 dose of study treatment and was used for all analyses.

Participants by arm

Arm	Count
Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	3
Phase 1: Mid Dose Four participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	4
Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	3
Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	4
Total	14

Withdrawals & dropouts

Period	Reason	FG000	FG002	FG003
Long-Term Follow-up	Lost to Follow-up	1	0	1
Open Label Treatment Period	Adverse Event	0	0	1
Open Label Treatment Period	Withdrawal by Subject	0	1	1

Baseline characteristics

Characteristic	Phase 1: Low Dose	Phase 1: Mid Dose	Phase 1: High Dose	Phase 2: High Dose	Total
Age, Continuous	66.0 Years STANDARD_DEVIATION 17.09	58.8 Years STANDARD_DEVIATION 4.99	64.7 Years STANDARD_DEVIATION 8.33	50.0 Years STANDARD_DEVIATION 24.12	59.1 Years STANDARD_DEVIATION 15.47
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants	4 Participants	3 Participants	4 Participants	14 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	1 Participants	0 Participants	0 Participants	0 Participants	1 Participants
Race (NIH/OMB) Black or African American	1 Participants	0 Participants	0 Participants	1 Participants	2 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) White	1 Participants	4 Participants	3 Participants	3 Participants	11 Participants
Sex: Female, Male Female	1 Participants	2 Participants	0 Participants	1 Participants	4 Participants
Sex: Female, Male Male	2 Participants	2 Participants	3 Participants	3 Participants	10 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk	EG003 affected / at risk
deaths Total, all-cause mortality	0 / 3	0 / 4	0 / 3	1 / 4
other Total, other adverse events	2 / 3	3 / 4	2 / 3	3 / 4
serious Total, serious adverse events	1 / 3	0 / 4	0 / 3	1 / 4

Outcome results

Primary

Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence

A standardized procedure for the collection of single, non-stereo images of the fundus of both eyes was obtained using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Any changes from baseline in fundus autofluorescence were also documented.

Time frame: Baseline (Day 1), 6 Months

Population: Safety Population. The Number Analyzed is the number of participants who completed the assessment with evaluable data the specified visit (whose vision abilities allowed them to participate in the testing).

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Month 6)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6)	1 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Baseline)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Month 6)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Month 6)	1 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Baseline)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline)	1 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any findings of Autofluorescence (Baseline)	1 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Baseline)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Month 6)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Baseline)	1 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Month 6)	0 Participants
Phase 1: Low Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Baseline)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Baseline)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline)	2 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Month 6)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any findings of Autofluorescence (Baseline)	2 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6)	2 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Baseline)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Month 6)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Month 6)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Month 6)	2 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Month 6)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Baseline)	0 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Baseline)	2 Participants
Phase 1: Mid Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Baseline)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Baseline)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline)	2 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Baseline)	1 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Baseline)	1 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any findings of Autofluorescence (Baseline)	2 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Month 6)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Month 6)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Month 6)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6)	1 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Month 6)	1 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Month 6)	1 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Baseline)	0 Participants
Phase 1: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Baseline)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline)	3 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Month 6)	1 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Month 6)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Month 6)	1 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Baseline)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Inflammation (Baseline)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any findings of Autofluorescence (Baseline)	2 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Other findings (Baseline)	1 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Hemorrhage (Baseline)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Baseline)	1 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Atrophy in the Fovea (Month 6)	0 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6)	2 Participants
Phase 2: High Dose	Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence	Any Evidence of Retinal Detachment (Month 6)	0 Participants

Primary

Change From Baseline at Month 6 in Ambulation in the Study Eye (Distance)

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).