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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002395-75 | EudraCT Number |
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The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIIB111 | Experimental | Participants will receive a single dose of sub-retinal injection of BIIB111 in each eye at Day 0 separated by an interval of <6 months, 6-12 months, or >12 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIIB111 | Drug | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | BCVA was assessed for both eyes using the ETDRS visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved. | Month 12 |
| Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | IOP, the fluid pressure inside the eye was measured and reported in millimeters mercury (mmHg). | Month 12 |
| Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Slit lamp examinations of study eyes included examination of Cornea, Conjunctiva, Iris, Lens, and Anterior Segment. | Baseline, Month 12 |
| Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy | Dilated Ophthalmoscopy examination of study eyes included examination of Vitreous, Macula, Peripheral retina, Choroid, and Optic nerve. | Baseline, Month 12 |
| Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading | The following lens opacity grades are reported for categories 1-4: Nuclear Opalescence grade, Nuclear Color grade, Cortical Cataract grade, and Posterior Cataract grade. Opacification severity is graded on a decimal scale, scores can range from 0.1 (no opacity) to 6.9 (maximum opacity) for the Nuclear Opalescence and Nuclear Color grades; and scores can range from 0.1 (lens clear) to 5.9 (lens unclear) for the Cortical and Posterior Cataract grades. Category 1 includes values 1, 1.0 and 0.x, Category 2 includes values 2, 2.0 and 1.x, Category 3 includes values 3, 3.0 and 2.x, and Category 4 includes values 4, 4.0, 3.x and any values above 4. For each opacification type the higher grading scores indicate greater severity. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in BCVA as Measured by the ETDRS Chart | BCVA was assessed for both eyes using the ETDRS VA chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. |
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Key Inclusion Criteria:
Are willing and able to give informed consent for participation in the study to have both eyes treated.
Have documentation of a genetically-confirmed diagnosis of CHM.
Have active disease clinically visible within the macular region of both eyes.
Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with BIIB111*
*If previously treated with BIIB111 in an antecedent study, participants may be eligible for participation following Sponsor approval.
For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.
Key Exclusion Criteria:
Have a history of amblyopia or inflammatory disorder in either eye.
Are unwilling to use barrier contraception methods or abstain from sexual intercourse for a period of 3 months following treatment with BIIB111 in either eye.
Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye.
Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study or the participant's ability to participate in the study. This includes but is not limited to a potential participants:
Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within an antecedent study with BIIB111.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Miami | Florida | 33136 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38970425 | Derived | MacLaren RE, Audo I, Fischer MD, Huckfeldt RM, Lam BL, Pennesi ME, Sisk R, Gow JA, Li J, Zhu K, Tsang SF. An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. Hum Gene Ther. 2024 Aug;35(15-16):564-575. doi: 10.1089/hum.2024.017. Epub 2024 Jul 27. | |
| 30194931 |
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A total of 66 unique male participants with Choroideremia were treated in the study. Of which 60 participants were treated in treatment Period 1 and 50 participants completed Period 1. 50 participants from Period 1 enrolled in Period 2 along with 6 unique participants who were treated in the first eye in a previous study (20150371 [NCT02553135], THOR-TUE-01 [NCT02671539]). 53 participants completed Period 2.
Participants were enrolled at the investigative sites in France, Germany, and the United States from 29 November 2017 to 29 June 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Period 1: BIIB111 | After vitrectomy and retinal detachment in the study eye 1, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 1. |
| FG001 | Treatment Period 2: BIIB111 | After vitrectomy and retinal detachment in the study eye 2, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 2. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Treatment Period 1(Day 1 up to Month 12) |
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| Treatment Period 2 (Month13 to Month24) |
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All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. The baseline data were collected and reported for overall population of unique participants instead of period-wise treated population.
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| ID | Title | Description |
|---|---|---|
| BG000 | All Treated Participants | After vitrectomy and retinal detachment in the study eye 1 and study eye 2, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 1 and Period 2 respectively. |
| 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 | Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 | BCVA was assessed for both eyes using the ETDRS visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | letters | Month 12 |
|
Day 0 (surgery) in period 1 up to last follow up visit in period 2 (approximately 2 years)
All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. The AEs data were collected and reported for unique participants enrolled in the study.
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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 | Treatment Period 1: BIIB111 | After vitrectomy and retinal detachment in the study eye 1, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 1. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Visual acuity reduced | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| US Biogen Clinical Trial Center | Biogen | 866-633-4636 | clinicaltrials@biogen.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 12, 2020 | Jun 29, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 3, 2022 | Jun 28, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D015794 | Choroideremia |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D015862 | Choroid Diseases |
| D014603 | Uveal Diseases |
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| ID | Term |
|---|---|
| D015316 | Genetic Therapy |
| ID | Term |
|---|---|
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D005818 | Genetic Engineering |
| D005821 | Genetic Techniques |
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| Month 12 |
| Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12 | SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| SD-OCT: Total Macular Volume at Month 12 | SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| SD-OCT: Central Horizontal Ellipsoid Width at Month 12 | SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| SD-OCT: Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| SD-OCT: Square Root of Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in square root of central ellipsoid area. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| SD-OCT: Choroidal Thickness at Foveal Center at Month 12 | SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. | Month 12 |
| Fundus Autofluorescence (AF): Mean Total Area of Preserved Autofluorescence at Month 12 | Fundus AF was used to assess the total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper autofluorescence (hyper AF) compared with the background areas of surrounding atrophy. | Month 12 |
| AF: Mean Square Root of Total Area of Preserved AF at Month 12 | Fundus AF was used to assess the square root of total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. | Month 12 |
| AF: Mean Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 | Fundus AF was used to assess the distance from foveal center to nearest border of preserved AF. | Month 12 |
| Fundus Photography: Number of Participants With Retinal Pigment Epithelium (RPE) Hyperplasia as Per Severity | Number of participants with RPE hyperplasia are reported for severity grades: mild, moderate, severe. | Month 12 |
| Fundus Photography: Number of Participants With Retinal Arteriolar Narrowing as Per Severity | Number of participants with retinal arteriolar narrowing are reported for severity grades: mild, moderate, severe. | Month 12 |
| Fundus Photography: Number of Participants With Retinal Vessel Sheathing as Per Severity | Number of participants with retinal vessel sheathing are reported for severity grades: mild, moderate, severe. | Month 12 |
| Fundus Photography: Number of Participants With Optic Atrophy/Pallor as Per Severity | Number of participants with optic atrophy/pallor are reported for severity grades: mild, moderate, severe. | Month 12 |
| Fundus Photography: Number of Participants With Optic Disc Swelling as Per Severity | Number of participants with optic disc swelling are reported for severity grades: mild, moderate, severe. | Month 12 |
| Microperimetry: Retinal Mean Sensitivity at Month 12 | Microperimetry was conducted to assess retinal mean sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (decibels) indicate higher retinal sensitivity. | Month 12 |
| Microperimetry: Bivariate Contour Ellipse Area 63% at Month 12 | Microperimetry was conducted to assess bivariate contour ellipse area 63%. | Month 12 |
| Microperimetry: Bivariate Contour Ellipse Area 95% at Month 12 | Microperimetry was conducted to assess bivariate contour ellipse area 95%. | Month 12 |
| Microperimetry: Fixation Losses (in Percentage) at Month 12 | Microperimetry was conducted to assess fixation losses (in percentage) which samples the optic nerve blind spot for positive responses. | Month 12 |
| Percentage of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) | An AE is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug/surgical procedure, whether or not related to the investigational product or with the surgical procedure. TEAEs are defined as AEs starting on or after the day of the first surgery. | Day 0 (surgery) in period 1 up to last follow up visit in period 2 (approximately 2 years) |
| Number of Participants With Vector Shedding Post-treatment at Month 3 | Tears (for both eyes- oculus dexter [OD] and oculus sinister [OS]), blood, urine and saliva samples were collected and tested using an appropriate assay for evidence of vector shedding. Participants with positive result for vector shredding post treatment are reported. | Baseline, at Month 3 |
| Number of Participants With Anti-drug Antibodies Post-treatment at Month 12 | Participants with antibodies to the REP-1 transgenic product are reported. | Month 12 |
| Vital Signs: Change From Baseline in Blood Pressure at Month 12 | Change from baseline in Systolic and diastolic blood pressures (BP) (millimeters of mercury [mmHg]) were reported. | Baseline, Month 12 |
| Vital Signs: Change From Baseline in Pulse Rate at Month 12 | Change from baseline in pulse rate (beats per minute) were reported. | Baseline, Month 12 |
| Baseline, Month 12 |
| AF: Change From Baseline in Total Area of Preserved Autofluorescence at Month 12 | Fundus AF was used to assess change in total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper autofluorescence (hyper AF) compared with the background areas of surrounding atrophy. Here negative values indicate decline in total area of preserved autofluoroscence. | Baseline, Month 12 |
| AF: Change From Baseline in Square Root of Total Area of Preserved AF at Month 12 | Fundus AF was used to assess change in square root of total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. Here negative values indicate decline in total area of preserved autofluoroscence. | Baseline, Month 12 |
| AF: Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 | Fundus Autofluoroscence was used to assess change in distance from foveal center to nearest border of preserved autofluoroscence. Here negative values indicate decline in total area of preserved autofluoroscence. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in Foveal Subfield Thickness at Month 12 | SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in foveal subfield thickness. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in Total Macular Volume at Month 12 | SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in total macular volume. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in the Central Horizontal Ellipsoid Width at Month 12 | SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central horizontal ellipsoid width. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central ellipsoid area. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in Square Root of Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in square root of central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in square root of central ellipsoid area. | Baseline, Month 12 |
| SD-OCT: Change From Baseline in the Choroidal Thickness at Foveal Center at Month 12 | SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in choroidal thickness. | Baseline, Month 12 |
| Microperimetry: Change From Baseline in Retinal Mean Sensitivity at Month 12 | Microperimetry was conducted to assess change in retinal mean sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (decibels) indicate higher retinal sensitivity. A negative change from baseline indicates decline in retinal sensitivity. | Baseline, Month 12 |
| Microperimetry: Change From Baseline in Bivariate Contour Ellipse Area 63% at Month 12 | Microperimetry was conducted to assess change in bivariate contour ellipse area 63%. A negative change from baseline indicates decline in bivariate contour ellipse area 63%. | Baseline, Month 12 |
| Microperimetry: Change From Baseline in the Bivariate Contour Ellipse Area 95% at Month 12 | Microperimetry was conducted to assess change in bivariate contour ellipse area 95%. A negative change from baseline indicates decline in bivariate contour ellipse area 95%. | Baseline, Month 12 |
| Microperimetry: Change From Baseline in Fixation Losses (in Percentage) at Month 12 | Microperimetry was conducted to assess change in fixation losses (in percentage) which samples the optic nerve blind spot for positive responses. A negative change from baseline indicates decline in fixation losses. | Baseline, Month 12 |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| Research Site | Cincinnati | Ohio | 45242 | United States |
| Research Site | Portland | Oregon | 97239 | United States |
| Research Site | Paris | 75571 | France |
| Research Site | Tübingen | 72076 | Germany |
| Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5. |
| Site Terminated by Sponsor |
|
| Reason not Specified |
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| NOT COMPLETED |
|
|
| Years |
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| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Description |
|---|
| OG000 | Treatment Period 1: BIIB111 | After vitrectomy and retinal detachment in the study eye 1, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 1. |
| OG001 | Treatment Period 2: BIIB111 | After vitrectomy and retinal detachment in the study eye 2, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 2. |
|
|
| Primary | Ophthalmic Examination Assessment: Mean Intraocular Pressure (IOP) at Month 12 | IOP, the fluid pressure inside the eye was measured and reported in millimeters mercury (mmHg). | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | mmHg | Month 12 |
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| Primary | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Slit Lamp Examination | Slit lamp examinations of study eyes included examination of Cornea, Conjunctiva, Iris, Lens, and Anterior Segment. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category at specified timepoint. | Posted | Count of Participants | Participants | Baseline, Month 12 |
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| Primary | Ophthalmic Examination Assessment: Number of Participants With Clinically Significant Abnormalities in Dilated Ophthalmoscopy | Dilated Ophthalmoscopy examination of study eyes included examination of Vitreous, Macula, Peripheral retina, Choroid, and Optic nerve. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category at specified timepoint. | Posted | Count of Participants | Participants | Baseline, Month 12 |
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| Primary | Ophthalmic Examination Assessment: Number of Participants With Lens Opacity Grading | The following lens opacity grades are reported for categories 1-4: Nuclear Opalescence grade, Nuclear Color grade, Cortical Cataract grade, and Posterior Cataract grade. Opacification severity is graded on a decimal scale, scores can range from 0.1 (no opacity) to 6.9 (maximum opacity) for the Nuclear Opalescence and Nuclear Color grades; and scores can range from 0.1 (lens clear) to 5.9 (lens unclear) for the Cortical and Posterior Cataract grades. Category 1 includes values 1, 1.0 and 0.x, Category 2 includes values 2, 2.0 and 1.x, Category 3 includes values 3, 3.0 and 2.x, and Category 4 includes values 4, 4.0, 3.x and any values above 4. For each opacification type the higher grading scores indicate greater severity. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified category of specified parameter. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Spectral Domain Optical Coherence Tomography (SD-OCT): Foveal Subfield Thickness at Month 12 | SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | microns (µ) | Month 12 |
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| Primary | SD-OCT: Total Macular Volume at Month 12 | SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | cubic millimeters (mm^3) | Month 12 |
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| Primary | SD-OCT: Central Horizontal Ellipsoid Width at Month 12 | SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | microns (μ) | Month 12 |
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| Primary | SD-OCT: Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | square millimeters (mm^2) | Month 12 |
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| Primary | SD-OCT: Square Root of Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in square root of central ellipsoid area. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Month 12 |
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| Primary | SD-OCT: Choroidal Thickness at Foveal Center at Month 12 | SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | microns (μ) | Month 12 |
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| Primary | Fundus Autofluorescence (AF): Mean Total Area of Preserved Autofluorescence at Month 12 | Fundus AF was used to assess the total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper autofluorescence (hyper AF) compared with the background areas of surrounding atrophy. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified parameter. | Posted | Mean | Standard Deviation | mm^2 | Month 12 |
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| Primary | AF: Mean Square Root of Total Area of Preserved AF at Month 12 | Fundus AF was used to assess the square root of total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Month 12 |
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| Primary | AF: Mean Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 | Fundus AF was used to assess the distance from foveal center to nearest border of preserved AF. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Month 12 |
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| Primary | Fundus Photography: Number of Participants With Retinal Pigment Epithelium (RPE) Hyperplasia as Per Severity | Number of participants with RPE hyperplasia are reported for severity grades: mild, moderate, severe. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Fundus Photography: Number of Participants With Retinal Arteriolar Narrowing as Per Severity | Number of participants with retinal arteriolar narrowing are reported for severity grades: mild, moderate, severe. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Fundus Photography: Number of Participants With Retinal Vessel Sheathing as Per Severity | Number of participants with retinal vessel sheathing are reported for severity grades: mild, moderate, severe. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Fundus Photography: Number of Participants With Optic Atrophy/Pallor as Per Severity | Number of participants with optic atrophy/pallor are reported for severity grades: mild, moderate, severe. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Fundus Photography: Number of Participants With Optic Disc Swelling as Per Severity | Number of participants with optic disc swelling are reported for severity grades: mild, moderate, severe. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Microperimetry: Retinal Mean Sensitivity at Month 12 | Microperimetry was conducted to assess retinal mean sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (decibels) indicate higher retinal sensitivity. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | decibels (dB) | Month 12 |
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| Primary | Microperimetry: Bivariate Contour Ellipse Area 63% at Month 12 | Microperimetry was conducted to assess bivariate contour ellipse area 63%. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | square degrees (deg^2) | Month 12 |
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| Primary | Microperimetry: Bivariate Contour Ellipse Area 95% at Month 12 | Microperimetry was conducted to assess bivariate contour ellipse area 95%. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | deg^2 | Month 12 |
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| Primary | Microperimetry: Fixation Losses (in Percentage) at Month 12 | Microperimetry was conducted to assess fixation losses (in percentage) which samples the optic nerve blind spot for positive responses. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | percentage (%) | Month 12 |
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| Primary | Percentage of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) | An AE is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug/surgical procedure, whether or not related to the investigational product or with the surgical procedure. TEAEs are defined as AEs starting on or after the day of the first surgery. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. The AEs data were collected and reported for unique participants enrolled in the study. | Posted | Number | percentage of participants | Day 0 (surgery) in period 1 up to last follow up visit in period 2 (approximately 2 years) |
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| Primary | Number of Participants With Vector Shedding Post-treatment at Month 3 | Tears (for both eyes- oculus dexter [OD] and oculus sinister [OS]), blood, urine and saliva samples were collected and tested using an appropriate assay for evidence of vector shedding. Participants with positive result for vector shredding post treatment are reported. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified parameter. | Posted | Count of Participants | Participants | Baseline, at Month 3 |
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| Primary | Number of Participants With Anti-drug Antibodies Post-treatment at Month 12 | Participants with antibodies to the REP-1 transgenic product are reported. | The immunogenicity analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2 with baseline sample and at least one post-surgery sample evaluable for immunogenicity. | Posted | Count of Participants | Participants | Month 12 |
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| Primary | Vital Signs: Change From Baseline in Blood Pressure at Month 12 | Change from baseline in Systolic and diastolic blood pressures (BP) (millimeters of mercury [mmHg]) were reported. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | mmHg | Baseline, Month 12 |
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| Primary | Vital Signs: Change From Baseline in Pulse Rate at Month 12 | Change from baseline in pulse rate (beats per minute) were reported. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | beats per minute | Baseline, Month 12 |
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| Secondary | Change From Baseline in BCVA as Measured by the ETDRS Chart | BCVA was assessed for both eyes using the ETDRS VA chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eyes. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | letters | Baseline, Month 12 |
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| Secondary | AF: Change From Baseline in Total Area of Preserved Autofluorescence at Month 12 | Fundus AF was used to assess change in total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper autofluorescence (hyper AF) compared with the background areas of surrounding atrophy. Here negative values indicate decline in total area of preserved autofluoroscence. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm^2 | Baseline, Month 12 |
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| Secondary | AF: Change From Baseline in Square Root of Total Area of Preserved AF at Month 12 | Fundus AF was used to assess change in square root of total area of preserved AF. Areas of preserved AF were identified as well-demarcated regions of relative hyper AF compared with the background areas of surrounding atrophy. Here negative values indicate decline in total area of preserved autofluoroscence. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Baseline, Month 12 |
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| Secondary | AF: Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 | Fundus Autofluoroscence was used to assess change in distance from foveal center to nearest border of preserved autofluoroscence. Here negative values indicate decline in total area of preserved autofluoroscence. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in Foveal Subfield Thickness at Month 12 | SD-OCT was used to assess change in foveal subfield thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in foveal subfield thickness. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | microns (μ) | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in Total Macular Volume at Month 12 | SD-OCT was used to assess change in total macular volume. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in total macular volume. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm^3 | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in the Central Horizontal Ellipsoid Width at Month 12 | SD-OCT was used to assess change in central horizontal ellipsoid width. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central horizontal ellipsoid width. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | microns (μ) | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in central ellipsoid area. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm^2 | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in Square Root of Central Ellipsoid Area at Month 12 | SD-OCT was used to assess change in square root of central ellipsoid area. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in square root of central ellipsoid area. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of specified eye. | Posted | Mean | Standard Deviation | mm | Baseline, Month 12 |
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| Secondary | SD-OCT: Change From Baseline in the Choroidal Thickness at Foveal Center at Month 12 | SD-OCT was used to assess change in choroidal thickness. The measurements were taken after dilation of the participant's pupil. A negative change from baseline indicates decline in choroidal thickness. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | microns (μ) | Baseline, Month 12 |
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| Secondary | Microperimetry: Change From Baseline in Retinal Mean Sensitivity at Month 12 | Microperimetry was conducted to assess change in retinal mean sensitivity within the macula. Retinal mean sensitivity to light was measured in decibels in multiple spots across the central and peripheral retina (entire visual field). Higher numbers (decibels) indicate higher retinal sensitivity. A negative change from baseline indicates decline in retinal sensitivity. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | dB | Baseline, Month 12 |
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| Secondary | Microperimetry: Change From Baseline in Bivariate Contour Ellipse Area 63% at Month 12 | Microperimetry was conducted to assess change in bivariate contour ellipse area 63%. A negative change from baseline indicates decline in bivariate contour ellipse area 63%. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | deg^2 | Baseline, Month 12 |
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| Secondary | Microperimetry: Change From Baseline in the Bivariate Contour Ellipse Area 95% at Month 12 | Microperimetry was conducted to assess change in bivariate contour ellipse area 95%. A negative change from baseline indicates decline in bivariate contour ellipse area 95%. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | deg^2 | Baseline, Month 12 |
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| Secondary | Microperimetry: Change From Baseline in Fixation Losses (in Percentage) at Month 12 | Microperimetry was conducted to assess change in fixation losses (in percentage) which samples the optic nerve blind spot for positive responses. A negative change from baseline indicates decline in fixation losses. | All Treated Subjects analysis set included all participants who completed the 'Day of Surgery' visit of Period 1 or 2. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | percentage | Baseline, Month 12 |
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| 0 |
| 60 |
| 12 |
| 60 |
| 59 |
| 60 |
| EG001 | Treatment Period 2: BIIB111 | After vitrectomy and retinal detachment in the study eye 2, participants received a single administration of BIIB111, 1 * 10^11 gp, as a sub-retinal injection on Day 0 (surgery day) of Period 2. | 1 | 56 | 8 | 56 | 53 | 56 |
| Noninfective retinitis | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Blindness unilateral | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Choroidal neovascularisation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eye inflammation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Macular hole | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Retinal degeneration | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Retinal detachment | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Tractional retinal detachment | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreous haemorrhage | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Appendicitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| COVID-19 pneumonia | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Completed suicide | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
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| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Anterior chamber cell | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eye pain | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Foreign body sensation in eyes | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctival hyperaemia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eye irritation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreal cells | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitritis | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Ocular discomfort | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Visual impairment | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctival oedema | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vision blurred | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Anterior chamber flare | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Dry eye | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Metamorphopsia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Photopsia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Visual acuity reduced | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Cataract | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Cystoid macular oedema | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eye pruritus | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Subretinal fluid | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreous floaters | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Cataract subcapsular | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eyelid ptosis | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Ocular hypertension | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Intraocular pressure increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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Our agreement is subject to confidentiality but generally, the PI can publish, for non-commercial purposes only, results and methods of the trial, but no other Sponsor's Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of the Sponsor's intellectual property contained in the proposed publication.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D008919 |
| Investigative Techniques |
| Cornea: Study Eye 1: Month 12 |
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| Cornea: Study Eye 2: Baseline |
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| Cornea: Study Eye 2: Month 12 |
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| Conjunctiva: Study Eye 1: Baseline |
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| Conjunctiva: Study Eye 1: Month 12 |
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| Conjunctiva: Study Eye 2: Baseline |
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| Conjunctiva: Study Eye 2: Month 12 |
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| Iris: Study Eye 1: Baseline |
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| Iris: Study Eye 1: Month 12 |
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| Iris: Study Eye 2: Baseline |
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| Iris: Study Eye 2: Month 12 |
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| Lens: Study Eye 1: Baseline |
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| Lens: Study Eye 1: Month 12 |
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| Lens: Study Eye 2: Baseline |
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| Lens: Study Eye 2: Month 12 |
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| Anterior Segment: Study Eye 1: Baseline |
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| Anterior Segment: Study Eye 1: Month 12 |
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| Anterior Segment: Study Eye 2: Baseline |
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| Anterior Segment: Study Eye 2: Month 12 |
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| Vitreous: Study Eye 1: Month 12 |
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| Vitreous: Study Eye 2: Baseline |
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| Vitreous: Study Eye 2: Month 12 |
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| Macula: Study Eye 1: Baseline |
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| Macula: Study Eye 1: Month 12 |
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| Macula: Study Eye 2: Baseline |
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| Macula: Study Eye 2: Month 12 |
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| Peripheral Retina: Study Eye 1: Baseline |
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| Peripheral Retina: Study Eye 1: Month 12 |
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| Peripheral Retina: Study Eye 2: Baseline |
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| Peripheral Retina: Study Eye 2: Month 12 |
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| Choroid: Study Eye 1: Baseline |
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| Choroid: Study Eye 1: Month 12 |
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| Choroid: Study Eye 2: Baseline |
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| Choroid: Study Eye 2: Month 12 |
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| Optic Nerve: Study Eye 1: Baseline |
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| Optic Nerve: Study Eye 1: Month 12 |
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| Optic Nerve: Study Eye 2: Baseline |
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| Optic Nerve: Study Eye 2: Month 12 |
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| Nuclear Opalescence Grade: Category 2: Study Eye 1 |
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| Nuclear Opalescence Grade: Category 3: Study Eye 1 |
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| Nuclear Opalescence Grade: Category 4: Study Eye 1 |
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| Nuclear Opalescence Grade: Category 1: Study Eye 2 |
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| Nuclear Opalescence Grade: Category 2: Study Eye 2 |
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| Nuclear Opalescence Grade: Category 3: Study Eye 2 |
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| Nuclear Opalescence Grade: Category 4: Study Eye 2 |
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| Nuclear Color Grade: Category 1: Study Eye 1 |
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| Nuclear Color Grade: Category 2: Study Eye 1 |
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| Nuclear Color Grade: Category 3: Study Eye 1 |
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| Nuclear Color Grade: Category 4: Study Eye 1 |
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| Nuclear Color Grade: Category 1: Study Eye 2 |
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| Nuclear Color Grade: Category 2: Study Eye 2 |
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| Nuclear Color Grade: Category 3: Study Eye 2 |
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| Nuclear Color Grade: Category 4: Study Eye 2 |
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| Cortical Cataract Grade: Category 1: Study Eye 1 |
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| Cortical Cataract Grade: Category 2: Study Eye 1 |
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| Cortical Cataract Grade: Category 3: Study Eye 1 |
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| Cortical Cataract Grade: Category 4: Study Eye 1 |
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| Cortical Cataract Grade: Category 1: Study Eye 2 |
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| Cortical Cataract Grade: Category 2: Study Eye 2 |
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| Cortical Cataract Grade: Category 3: Study Eye 2 |
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| Cortical Cataract Grade: Category 4: Study Eye 2 |
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| Posterior Cataract Grade: Category 1: Study Eye 1 |
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| Posterior Cataract Grade: Category 2: Study Eye 1 |
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| Posterior Cataract Grade: Category 3: Study Eye 1 |
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| Posterior Cataract Grade: Category 4: Study Eye 1 |
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| Posterior Cataract Grade: Category 1: Study Eye 2 |
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| Posterior Cataract Grade: Category 2: Study Eye 2 |
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| Posterior Cataract Grade: Category 3: Study Eye 2 |
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| Posterior Cataract Grade: Category 4: Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Study Eye 2 |
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| Mild: Study Eye 2 |
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| Moderate: Study Eye 1 |
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| Moderate: Study Eye 2 |
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| Severe: Study Eye 1 |
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| Severe: Study Eye 2 |
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| Mild: Study Eye 2 |
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| Moderate: Study Eye 1 |
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| Moderate: Study Eye 2 |
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| Severe: Study Eye 1 |
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| Severe: Study Eye 2 |
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| Mild: Study Eye 2 |
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| Moderate: Study Eye 1 |
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| Moderate: Study Eye 2 |
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| Severe: Study Eye 1 |
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| Severe: Study Eye 2 |
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| Mild: Study Eye 2 |
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| Moderate: Study Eye 1 |
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| Moderate: Study Eye 2 |
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| Severe: Study Eye 1 |
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| Severe: Study Eye 2 |
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| Mild: Study Eye 2 |
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| Moderate: Study Eye 1 |
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| Moderate: Study Eye 2 |
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| Severe: Study Eye 1 |
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| Severe: Study Eye 2 |
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| Tears OS |
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| Blood |
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| Saliva |
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| Urine |
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| Systolic BP: Change at Month 12 |
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| Diastolic BP: Baseline |
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| Diastolic BP: Change at Month 12 |
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| Change at Month 12 |
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| Change at Month 12: Study Eye 2 |
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| Change at Month 12: Study Eye 2 |
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| Change at Month 12: Study Eye 2 |
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| Change at Month 12: Study Eye 2 |
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| Change at Month 12: Study Eye 2 |
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| Change at Month 12: Study Eye 2 |
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