Usher Syndrome, Type 1B
Conditions
Brief summary
Natural History Study in Subjects With Usher Syndrome ((USH1B) is a multi-centre, longitudinal, observational study designed to evaluate disease progression in subjects with USH1B by several vision-related assessments.
Detailed description
Natural History Study in Subjects With Usher Syndrome (USH1B) is being conducted to understand the progression of disease in USH1B patients as measured by visual acuity and visual field testing and a number of other vision-related assessments. Disease progression will be evaluated as change over time in these measures and associations between the endpoints will be examined. The primary objective of this study is to evaluate the natural progression of disease over time in USHIB patients using visual field testing and best corrected visual acuity. The secondary objectives of this study are to evaluate the progression of disease over time in USHIB patients through additional assessments: * Microperimetry (only in selected centres) * Fundus autofluorescence (FAF) * Optical Coherence Tomography (OCT) * Full-field Electroretinogram (ERG), * Multifocal Electroretinogram, * Vision-related function and quality of life, as measured by the 25-Item National Eye Institute Visual Function Questionnaire (NEI VFQ-25)
Interventions
None listed
Sponsors
Fondazione Telethon
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
8 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Must be willing to adhere to protocol for long-term follow-up as evidenced by written informed consent or parental permission and subject assent. 2. Subjects diagnosed with USH1. 3. Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes). 4. Age eight years old or older at the time of baseline. 5. Visual acuity ≥ 20/640 in at least one eye
Exclusion criteria
1. Unable or unwilling to meet requirements of the study. 2. Unable to communicate with suitable verbal/auditory and/or tactile sign language (in the opinion of the investigator) 3. Participation in a clinical study with an investigational drug in the past six months. 4. Pre-existing eye conditions that would interfere with the interpretation of study endpoints (for example, glaucoma, corneal or significant lenticular opacities, cystoid macular oedema, macular hole). 5. Complicating systemic diseases in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example, radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Also excluded would be subjects with immuno- compromising diseases, as there could be susceptibility to opportunistic infection \[such as cytomegalovirus (CMV) retinitis\]. 6. Subjects with diabetes or sickle cell disease would be excluded if they had any manifestation of advanced retinopathy (e.g. macular edema or proliferative changes). 7. Prior ocular surgery within three months. 8. Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| BCVA - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes. BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit. |
| III4e Area - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes. Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| V4e Area - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes. Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| BCVA - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the worse seeing eyes. BCVA in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in the ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit. |
| III4e Area - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes. Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| V4e Area - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes. Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| EZ Band Horizontal - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the worse-seeing eyes. EZ Band Horizontal is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| MS - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the better-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| EZ Band Vertical - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the worse-seeing eyes. EZ Band Vertical is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| CMT - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the better-seeing eyes. CMT was reported in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| EZ Band Horizontal - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the better-seeing eyes. EZ Band Horizontal is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| EZ Band Vertical - Better Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the better-seeing eyes. EZ Band Vertical is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| MS - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the worse-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
| CMT - Worse Seeing Eyes | 1 year follow up visit | Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the worse-seeing eyes. CMT is expressed in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit. |
Countries
Italy, Netherlands, Spain
Participant flow
Recruitment details
53 subjects meeting the inclusion/exclusion criteria were enrolled in the 18-month recruitment period.
Participants by arm
| Arm | Count |
|---|---|
| Group of Subjects With Usher Syndrome Type 1B In this study, there is 1 group of subjects with Usher Syndrome type 1B enrolled, over the 18-month recruitment period, to evaluate disease progression by several vision-related assessments.
Subjects will be assessed using the following schedule:
* Baseline visit
* 1 year follow-up visit
* 2 year follow-up visit | 53 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 3 |
Baseline characteristics
| Characteristic | Group of Subjects With Usher Syndrome Type 1B |
|---|---|
| Age, Categorical <=18 years | 11 Participants |
| Age, Categorical >=65 years | 3 Participants |
| Age, Categorical Between 18 and 65 years | 39 Participants |
| Age, Continuous | 33.6 years STANDARD_DEVIATION 16.7 |
| BCVA - better seeing eyes | 66.4 ETDRS Score STANDARD_DEVIATION 17.9 |
| BCVA - worse seeing eyes | 59.5 ETDRS score STANDARD_DEVIATION 21.7 |
| CMT - better seeing eyes | 259.6 Micrometers STANDARD_DEVIATION 63 |
| CMT - worse seeing eyes | 250.7 Micrometers STANDARD_DEVIATION 63.3 |
| EZ Band Horizontal - better seeing eyes | 2807.5 Micrometers STANDARD_DEVIATION 2374.6 |
| EZ Band Vertical - better seeing eyes | 2436.1 Micrometers STANDARD_DEVIATION 2286.4 |
| EZ Hor. - worse seeing eyes | 2615.5 Micrometers STANDARD_DEVIATION 2370.4 |
| EZ Vert. - worse seeing eyes | 2332.4 Micrometers STANDARD_DEVIATION 666.5 |
| III4e Area - better seeing eyes | 3365.8 Degree Squared STANDARD_DEVIATION 4142.1 |
| III4e Area - worse seeing eyes | 4176.4 Degree squared STANDARD_DEVIATION 4400.3 |
| MS - better seeing eyes | 9.7 Decibels STANDARD_DEVIATION 9.9 |
| MS - worse seeing eyes | 9 Decibels STANDARD_DEVIATION 10.2 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 53 Participants |
| Region of Enrollment Italy | 29 participants |
| Region of Enrollment Netherlands | 8 participants |
| Region of Enrollment Spain | 16 participants |
| Sex: Female, Male Female | 23 Participants |
| Sex: Female, Male Male | 30 Participants |
| V4e Area - better seeing eyes | 5605.2 Degree squared STANDARD_DEVIATION 5290.2 |
| V4e Area - worse seeing eyes | 5613.22 Degree squared STANDARD_DEVIATION 5127.2 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 53 |
| other Total, other adverse events | 0 / 53 |
| serious Total, serious adverse events | 0 / 53 |
Outcome results
Primary
BCVA - Better Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes. BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visit
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | BCVA - Better Seeing Eyes | -2.26 ETDRS score | Standard Deviation 3.838 |
p-value: <0.01Regression, Linear
Primary
BCVA - Better Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the better-seeing eyes. BCVA in the better seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
Time frame: 2 year follow up visit
Population: patients undergoing two-year follow-up visit
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | BCVA - Better Seeing Eyes | -1.468 ETDRS score | Standard Deviation 4.999 |
p-value: <0.01Regression, Linear
Primary
BCVA - Worse Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity (BCVA) in the worse seeing eyes. BCVA in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in the ETDRS score collected at the 1 year follow-up visit compared with the score at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | BCVA - Worse Seeing Eyes | -0.625 ETDRS score | Standard Deviation 5.35 |
p-value: 0.4178Regression, Linear
Primary
BCVA - Worse Seeing Eyes
Change (from Baseline) in Best Corrected Visual acuity in the worse seeing eyes. Best corrected visual acuity in the worse seeing eyes was assessed using the ETRS score (minimum value 0 and maximum value 100, with 0 representing the worst, and 100 the best visual functioning). The results are reported as the change in the ETDRS score collected at the 2 year follow-up visit compared with the score at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | BCVA - Worse Seeing Eyes | -0.909 ETDRS score | Standard Deviation 7.041 |
p-value: 0.2677Regression, Linear
Primary
III4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes. Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | III4e Area - Better Seeing Eyes | 561.966 degree squared | Standard Deviation 2043.612 |
p-value: 0.3269Regression, Linear
Primary
III4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the better seeing eyes. Visual field area using III4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 year follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | III4e Area - Better Seeing Eyes | 70.286 degree squared | Standard Deviation 1451.128 |
p-value: 0.4265Regression, Linear
Primary
III4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes. Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | III4e Area - Worse Seeing Eyes | -445.569 degree squared | Standard Deviation 2920.491 |
p-value: 0.3364Regression, Linear
Primary
III4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using III4e stimulus size in the worse-seeing eyes. Visual field area using III4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | III4e Area - Worse Seeing Eyes | -407.928 degree squared | Standard Deviation 3209.147 |
p-value: 0.1124Regression, Linear
Primary
V4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes. Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | V4e Area - Better Seeing Eyes | 273.309 degree squared | Standard Deviation 2432.785 |
p-value: 0.4782Regression, Linear
Primary
V4e Area - Better Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the better seeing eyes. Visual field area using V4e stimulus size in the better seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 year follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | V4e Area - Better Seeing Eyes | 700.245 degree squared | Standard Deviation 3942.6 |
p-value: 0.2716Regression, Linear
Primary
V4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes. Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | V4e Area - Worse Seeing Eyes | 391.212 degree squared | Standard Deviation 2726.331 |
p-value: 0.2649Regression, Linear
Primary
V4e Area - Worse Seeing Eyes
Change (from Baseline) in visual field area using V4e stimulus size in the worse-seeing eyes. Visual field area using V4e stimulus size in the worse-seeing eyes is expressed in degree squared (minimum value 0, maximum value 20000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | V4e Area - Worse Seeing Eyes | 610.017 degree squared | Standard Deviation 5658.524 |
p-value: 0.216Regression, Linear
Secondary
CMT - Better Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the better-seeing eyes. CMT was reported in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | CMT - Better Seeing Eyes | 7 micrometers | Standard Deviation 21.532 |
p-value: 0.0242Regression, Linear
Secondary
CMT - Better Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the better-seeing eyes. CMT was reported in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | CMT - Better Seeing Eyes | -5.667 micrometers | Standard Deviation 18.877 |
p-value: 0.5092Regression, Linear
Secondary
CMT - Worse Seeing Eyes
Change (from Baseline) in Central Macular Thickness, evaluated with Optical Coherence tomography, in the worse-seeing eyes. CMT is expressed in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | CMT - Worse Seeing Eyes | -2.026 micrometers | Standard Deviation 22.358 |
p-value: 0.4224Regression, Linear
Secondary
CMT - Worse Seeing Eyes
Change (from Baseline) in Central Macular Thickness (CMT), evaluated with Optical Coherence tomography, in the worse-seeing eyes. CMT is expressed in micrometers (minimum value 0, maximum value 1000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | CMT - Worse Seeing Eyes | 4.786 micrometers | Standard Deviation 15.682 |
p-value: 0.0542Regression, Linear
Secondary
EZ Band Horizontal - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the better-seeing eyes. EZ Band Horizontal is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Horizontal - Better Seeing Eyes | -42.795 micrometers | Standard Deviation 324.893 |
p-value: 0.3634Regression, Linear
Secondary
EZ Band Horizontal - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the better-seeing eyes. EZ Band Horizontal is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Horizontal - Better Seeing Eyes | -160.556 micrometers | Standard Deviation 628.761 |
p-value: 0.0677Regression, Linear
Secondary
EZ Band Horizontal - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the worse-seeing eyes. EZ Band Horizontal is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Horizontal - Worse Seeing Eyes | -122.763 micrometers | Standard Deviation 486.734 |
p-value: 0.1247Regression, Linear
Secondary
EZ Band Horizontal - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (horizontal scan), in the worse-seeing eyes. EZ Band Horizontal is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Horizontal - Worse Seeing Eyes | -21.658 micrometers | Standard Deviation 237.373 |
p-value: 0.1461Regression, Linear
Secondary
EZ Band Vertical - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the better-seeing eyes. EZ Band Vertical is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Vertical - Better Seeing Eyes | -289.75 micrometers | Standard Deviation 1107.784 |
p-value: 0.156Regression, Linear
Secondary
EZ Band Vertical - Better Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the better-seeing eyes. EZ Band Vertical is reported in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Vertical - Better Seeing Eyes | 190.553 micrometers | Standard Deviation 1276.729 |
p-value: 0.3148Regression, Linear
Secondary
EZ Band Vertical - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the worse-seeing eyes. EZ Band Vertical is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Vertical - Worse Seeing Eyes | -15.861 micrometers | Standard Deviation 236.02 |
p-value: 0.0901Regression, Linear
Secondary
EZ Band Vertical - Worse Seeing Eyes
Change (from Baseline) in Ellipsoid Zone Band Width, evaluated with Optical Coherence tomography (Vertical scan), in the worse-seeing eyes. EZ Band Vertical is expressed in micrometers (minimum value 0, maximum value 4000). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | EZ Band Vertical - Worse Seeing Eyes | -149.051 micrometers | Standard Deviation 528.203 |
p-value: 0.0699Regression, Linear
Secondary
MS - Better Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the better-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | MS - Better Seeing Eyes | 0.137 decibels | Standard Deviation 2.89 |
p-value: 0.9032Regression, Linear
Secondary
MS - Better Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the better-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | MS - Better Seeing Eyes | -0.315 decibels | Standard Deviation 7.174 |
p-value: 0.7889Regression, Linear
Secondary
MS - Worse Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the worse-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 1 year follow-up visit compared with the values at the baseline visit.
Time frame: 1 year follow up visit
Population: patients undergoing one-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | MS - Worse Seeing Eyes | -0.056 decibels | Standard Deviation 3.279 |
p-value: 0.9349Regression, Linear
Secondary
MS - Worse Seeing Eyes
Change (from Baseline) in macular sensitivity, assessed by microperimetry, in the worse-seeing eyes. Macular sensitivity, assessed by microperimetry is expressed in decibels (minimum value 0, maximum value 30). The results are reported as the change in the values collected at the 2 year follow-up visit compared with the values at the baseline visit.
Time frame: 2 years follow up visit
Population: patients undergoing two-year follow-up visits
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group of Subjects With Usher Syndrome Type 1B | MS - Worse Seeing Eyes | -0.758 decibels | Standard Deviation 7.062 |
p-value: 0.7196Regression, Linear