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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Phase 1: Low Dose | Experimental | Single intravitreal injection of AGN-151597 |
|
| Phase 1: Mid Dose | Experimental | Single intravitreal injection of AGN-151597 |
|
| Phase 1: High Dose | Experimental | Single intravitreal injection of AGN-151597 |
|
| Phase 2: High Dose | Experimental | Single intravitreal injection of AGN-151597 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AGN-151597 | Drug | AGN-151597 is a gene therapeutic delivered by intravitreal injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597 | 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. | Baseline (Day 1) to 6 Months |
| Visual Acuity in the Study Eye at Baseline and Month 6 | 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. | Baseline (Day 1), 6 Months |
| Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold | 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. | Baseline (Day 1) to Month 6 |
| Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold |
| Measure | Description | Time Frame |
|---|---|---|
| Visual Acuity in the Study Eye at Months 3, 12, and 24 | 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. |
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Inclusion Criteria:
Participants must meet all of the following criteria.
Exclusion Criteria:
Any one of the following will exclude patients from being enrolled into the study:
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| Name | Affiliation | Role |
|---|---|---|
| ABBVIE INC. | AbbVie | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Francisco - Mission Bay /ID# 235717 | San Francisco | California | 94158 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30218651 | Derived | Simunovic MP, Shen W, Lin JY, Protti DA, Lisowski L, Gillies MC. Optogenetic approaches to vision restoration. Exp Eye Res. 2019 Jan;178:15-26. doi: 10.1016/j.exer.2018.09.003. Epub 2018 Sep 13. |
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AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.
For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
The Safety Population included all enrolled participants who received at least 1 dose of study treatment and was used for all analyses.
A total of 14 participants were enrolled in the study in the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| FG002 | 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. |
| FG003 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Open Label Treatment Period |
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| Long-Term Follow-up |
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Safety Population
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597 | 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. | Safety Population | Posted | Number | participants | Baseline (Day 1) to 6 Months |
|
All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Phase 1: Low Dose | Three participants were enrolled and received a low 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ARRHYTHMIA | Cardiac disorders | MedDRA (27.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANTERIOR CHAMBER CELL | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Medical Services | AbbVie | 800-633-9110 | abbvieclinicaltrials@abbvie.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 22, 2022 | Aug 25, 2025 | Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 21, 2019 | Aug 25, 2025 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
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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. |
| Baseline (Day 1) to Month 6 |
| Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold | 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. | Baseline (Day 1) to Month 6 |
| Change From Baseline at Month 6 in Ambulation in the Study Eye (Time) | 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). | Baseline (Day 1) to Month 6 |
| 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). | Baseline (Day 1) to Month 6 |
| Intraocular Pressure (IOP) Measurements in the Study Eye | 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. | Baseline (Day 1), 6 Months |
| 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. | Baseline (Day 1), 6 Months |
| 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Baseline (Day 1) to Month 6 |
| 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Baseline (Day 1) to Month 6 |
| 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Baseline (Day 1) to Month 6 |
| 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Baseline (Day 1) to Month 6 |
| 3, 12, and 24 Months |
| Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold | 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. | Baseline (Day 1) to Months 3, 12, and 24 |
| Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold | 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. | Baseline (Day 1) to Months 3, 12, and 24 |
| Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold | 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. | Baseline (Day 1) to Months 3, 12, and 24 |
| Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) | 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). | Baseline (Day 1) to Months 3, 12, and 24 |
| Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test 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). | Baseline (Day 1) to Months 3, 12, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency) | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) | 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. | Baseline (Day 1) to Months 6 and 24 |
| Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) | 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. | Baseline (Day 1) to Months 6 and 24 |
| Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores | 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. | Baseline (Day 1) to Months 3, 6, and 24 |
| Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence | 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. | Months 3 and 24 |
| 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 | 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). | Baseline to Months 3, 12, and 24 |
| 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 | 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). | Baseline to Months 3, 12, and 24 |
| Cincinnati Eye Institute- Edgewood /ID# 236713 |
| Edgewood |
| Kentucky |
| 41017-3415 |
| United States |
| Duke Eye Center /ID# 235715 | Durham | North Carolina | 27705 | United States |
| Retina Foundation of the Southwest /ID# 235199 | Dallas | Texas | 75231 | United States |
| Adverse Event |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
| 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. |
| BG002 | 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. |
| BG003 | 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. |
| BG004 | Total | Total of all reporting groups |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG001 | 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. |
| OG002 | 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. |
| OG003 | 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. |
|
|
| Primary | Visual Acuity in the Study Eye at Baseline and Month 6 | 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. | Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. Although subjects who passed the Count Fingers assessment did not need to be evaluated for the Hand Motion assessment, and subjects who passed the Hand Motion assessment did not need to be evaluated further for the Light Perception Assessment, some participants who passed the previous assessments were still evaluated in error for subsequent assessments. | Posted | Count of Participants | Participants | Baseline (Day 1), 6 Months |
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| Primary | Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Month 6 |
|
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| Primary | Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at the 6-month visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Month 6 |
|
|
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| Primary | Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at the 6-month visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Month 6 |
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| Primary | Change From Baseline at Month 6 in Ambulation in the Study Eye (Time) | 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). | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | seconds | Baseline (Day 1) to Month 6 |
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| 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). | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | meters | Baseline (Day 1) to Month 6 |
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| Primary | Intraocular Pressure (IOP) Measurements in the Study Eye | 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. | Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. | Posted | Mean | Standard Deviation | mmHg | Baseline (Day 1), 6 Months |
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| 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. | 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). | Posted | Count of Participants | Participants | Baseline (Day 1), 6 Months |
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| Primary | 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and the 6-month visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | mm3 | Baseline (Day 1) to Month 6 |
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| Primary | 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | mm3 | Baseline (Day 1) to Month 6 |
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| Primary | 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | mm3 | Baseline (Day 1) to Month 6 |
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| Primary | 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 | Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | um | Baseline (Day 1) to Month 6 |
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| Secondary | Visual Acuity in the Study Eye at Months 3, 12, and 24 | 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. | Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. Although subjects who passed the Count Fingers assessment did not need to be evaluated for the Hand Motion assessment, and subjects who passed the Hand Motion assessment did not need to be evaluated further for the Light Perception Assessment, some participants who passed the previous assessments were still evaluated in error for subsequent assessments. | Posted | Count of Participants | Participants | 3, 12, and 24 Months |
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| Secondary | Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Months 3, 12, and 24 |
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| Secondary | Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Months 3, 12, and 24 |
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| Secondary | Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | Decibels | Baseline (Day 1) to Months 3, 12, and 24 |
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| Secondary | Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) | 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). | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | seconds | Baseline (Day 1) to Months 3, 12, and 24 |
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| Secondary | Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test 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). | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | meters | Baseline (Day 1) to Months 3, 12, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores | 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. | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | cd/m2 | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants who completed assessment with evaluable data at baseline and at least one postbaseline visit at Month 3, Month 6, or Month 24. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing) | Posted | Mean | Standard Deviation | mV | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing) | Posted | Mean | Standard Deviation | mV | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | mV | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | msec | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | msec | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | msec | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | uV | Baseline (Day 1) to Months 6 and 24 |
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| Secondary | Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) | 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. | Safety Population. The Overall Number of Participants Analyzed is the number of participants who completed assessment with evaluable data at baseline and at least one postbaseline visit at Month 6 or Month 24. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | msec | Baseline (Day 1) to Months 6 and 24 |
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| Secondary | Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores | 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. | Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1) to Months 3, 6, and 24 |
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| Secondary | Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence | 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. | 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). | Posted | Count of Participants | Participants | Months 3 and 24 |
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| Secondary | 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 | 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). | Safety Population. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | mm3 | Baseline to Months 3, 12, and 24 |
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| Secondary | 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 | 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). | Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and the specified postbaseline visit (whose vision abilities allowed them to participate in the testing). | Posted | Mean | Standard Deviation | um | Baseline to Months 3, 12, and 24 |
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| 0 |
| 3 |
| 1 |
| 3 |
| 2 |
| 3 |
| EG001 | 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. | 0 | 4 | 0 | 4 | 3 | 4 |
| EG002 | 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. | 0 | 3 | 0 | 3 | 2 | 3 |
| EG003 | 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. | 1 | 4 | 1 | 4 | 3 | 4 |
| INTESTINAL OBSTRUCTION | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
|
| POST PROCEDURAL HAEMORRHAGE | Injury, poisoning and procedural complications | MedDRA (27.0) | Systematic Assessment |
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| MUSCULOSKELETAL CHEST PAIN | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
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| CEREBROSPINAL FLUID LEAKAGE | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
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| BLEPHARITIS | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| CATARACT NUCLEAR | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| CATARACT SUBCAPSULAR | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| CONJUNCTIVAL HAEMORRHAGE | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| EYE IRRITATION | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| EYE PAIN | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| IRITIS | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| OCULAR HYPERTENSION | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| RETINAL HAEMORRHAGE | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| VISUAL ACUITY REDUCED | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| VITREAL CELLS | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| VITREOUS DETACHMENT | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| VITRITIS | Eye disorders | MedDRA (27.0) | Systematic Assessment |
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| SWELLING | General disorders | MedDRA (27.0) | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| EAR INFECTION | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| FUNGAL FOOT INFECTION | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| ORAL FUNGAL INFECTION | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| SIALOADENITIS | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| TINEA PEDIS | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| TOOTH ABSCESS | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| TOOTH INFECTION | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
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| FALL | Injury, poisoning and procedural complications | MedDRA (27.0) | Systematic Assessment |
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| INTRAOCULAR PRESSURE INCREASED | Investigations | MedDRA (27.0) | Systematic Assessment |
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| HYPERKALAEMIA | Metabolism and nutrition disorders | MedDRA (27.0) | Systematic Assessment |
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| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
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| NECK PAIN | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
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| RHEUMATOID ARTHRITIS | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
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| BENIGN NEOPLASM OF EYELID | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.0) | Systematic Assessment |
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| OPTIC NEURITIS | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
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| DEVICE DISLOCATION | Product Issues | MedDRA (27.0) | Systematic Assessment |
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| POLLAKIURIA | Renal and urinary disorders | MedDRA (27.0) | Systematic Assessment |
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AbbVie requests that any investigator or institution that plans on presenting/publishing results disclosure, provide written notification of their request 60 days prior to their presentation/publication. AbbVie requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if AbbVie needs to secure patent or proprietary protection.
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Count Fingers (6 months) |
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| Hand Motion (Baseline) |
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| Hand Motion (6 months) |
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| Light Perception (Baseline) |
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| Light Perception (6 months) |
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| Month 6 |
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| Any Evidence of Retinal Hemorrhage (Baseline) |
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| Any Evidence of Retinal Detachment (Baseline) |
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| Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline) |
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| Any Evidence of Atrophy in the Fovea (Baseline) |
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| Any Evidence of Other findings (Baseline) |
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| Any findings of Autofluorescence (Baseline) |
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| Any Evidence of Inflammation (Month 6) |
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| Any Evidence of Retinal Hemorrhage (Month 6) |
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| Any Evidence of Retinal Detachment (Month 6) |
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| Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6) |
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| Any Evidence of Atrophy in the Fovea (Month 6) |
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| Any Evidence of Other findings (Month 6) |
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| Any findings of Autofluorescence (Month 6) |
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| Count Fingers (12 months) |
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| Count Fingers (24 months) |
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| Hand Motion (3 months) |
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| Hand Motion (12 months) |
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| Hand Motion (24 months) |
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| Light Perception (3 months) |
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| Light Perception (12 months) |
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| Light Perception (24 months) |
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| Blue Light Threshold (12 months) |
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| Blue Light Threshold (24 months) |
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| Red Light Threshold (12 months) |
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| Red Light Threshold (24 months) |
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| White Light Threshold (12 months) |
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| White Light Threshold (24 months) |
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| Mean Time (seconds) (12 months) |
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| Mean Time (seconds) (24 months) |
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| Mean Distance (meters) (12 months) |
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| Mean Distance (meters) (24 months) |
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| Red Threshold Intensity (3 months) |
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| Blue Threshold Intensity (6 months) |
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| Red Threshold Intensity (6 months) |
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| Blue Threshold Intensity (24 months) |
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| Red Threshold Intensity (24 months) |
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| SE/0.974 Amplitude (mV) - 6 months |
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| SE/0.974 Amplitude (mV) - 24 months |
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| SE/0.649 Amplitude (mV) - 6 months |
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| SE/0.649 Amplitude (mV) - 24 months |
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| SE/0.216 Amplitude (mV) - 6 months |
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| SE/0.216 Amplitude (mV) - 24 months |
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| SE/0.974 Latency (msec) - 6 months |
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| SE/0.974 Latency (msec) - 24 months |
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| SE/0.649 Latency (msec) - 6 months |
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| SE/0.649 Latency (msec) - 24 months |
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| SE/0.216 Latency (msec) - 6 months |
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| SE/0.216 Latency (msec) - 24 months |
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| Dark Adapted 3 ERG a-wave (uV) - 6 months |
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| Dark Adapted 3 ERG b-wave (uV) - 6 months |
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| Light Adapted 3 ERG a-wave (uV) - 6 months |
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| Light Adapted 3 ERG b-wave (uV) - 6 months |
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| Light Adapted 30 Flicker Amplitude (uV) - 6 months |
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| Dark Adapted 0.01 ERG b-wave (uV) - 24 months |
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| Dark Adapted 3 ERG a-wave (uV) - 24 months |
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| Dark Adapted 3 ERG b-wave (uV) - 24 months |
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| Light Adapted 3 ERG a-wave (uV) - 24 months |
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| Light Adapted 3 ERG b-wave (uV) - 24 months |
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| Light Adapted 30 Flicker Amplitude (uV) - 24 months |
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| Dark Adapted 3 ERG Latency (msec) - 6 months |
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| Light Adapted 3 ERG Latency (msec) - 6 months |
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| Light Adapted 3 Flicker Latency (msec) - 6 months |
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| Dark Adapted 0.01 ERG Latency (msec) - 24 months |
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| Dark Adapted 3 ERG Latency (msec) - 24 months |
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| Light Adapted 3 ERG Latency (msec) - 24 months |
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| Light Adapted 3 Flicker Latency (msec) - 24 months |
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| Month 6 |
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| Month 24 |
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| Any Evidence of Retinal Hemorrhage (Month 3) |
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| Any Evidence of Retinal Detachment (Month 3) |
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| Any Evidence of Atrophy in the Fovea (Month 3) |
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| Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 3) |
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| Any Findings of Autofluorescence (Month 3) |
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| Any Evidence of Other findings (Month 3) |
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| Any Evidence of Inflammation (Month 24) |
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| Any Evidence of Retinal Hemorrhage (Month 24) |
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| Any Evidence of Retinal Detachment (Month 24) |
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| Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 24) |
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| Any Evidence of Atrophy in the Fovea (Month 24) |
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| Any Evidence of Other findings (Month 24) |
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| Any Findings of Autofluorescence (Month 24) |
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| Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (3 months) |
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| Total Retinal Volume (mm3) (3 months) |
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| Total Retinal Nerve Fibre Layer Volume (mm3) (12 months) |
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| Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (12 months) |
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| Total Retinal Volume (mm3) (12 months) |
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| Total Retinal Nerve Fibre Layer Volume (mm3) (24 months) |
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| Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (24 months) |
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| Total Retinal Volume (mm3) (24 months) |
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| Total Retinal Thickness (um) (24 months) |
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