Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS)
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|ClinicalTrials.gov Identifier: NCT04926129|
Recruitment Status : Recruiting
First Posted : June 14, 2021
Last Update Posted : June 14, 2021
|Condition or disease||Intervention/treatment|
|X-Linked Retinitis Pigmentosa||Other: Other: Assessments|
|Study Type :||Observational|
|Estimated Enrollment :||300 participants|
|Official Title:||Natural History of the Progression of X-Linked Retinitis Pigmentosa|
|Actual Study Start Date :||September 29, 2017|
|Estimated Primary Completion Date :||September 10, 2024|
|Estimated Study Completion Date :||September 10, 2024|
Participant's eye with Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) ≥74 letters (Equivalent to: Snellen 6/9 or 20/32; decimal 0.63; Logarithm of the minimum angle of resolution [LogMAR] 0.2) will be enrolled.
Other: Other: Assessments
Administered as specified in the treatment arm.
Other Name: BIIB112
Participant's eye with ETDRS BCVA 34-73 letters, inclusive (Equivalent to: Snellen 6/12 - 6/60 or 20/40 - 20/200; decimal 0.5 - 0.1; LogMAR 0.3-1.0) will be enrolled.
Other: Other: Assessments
Administered as specified in the treatment arm.
Other Name: BIIB112
- Change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) Best-corrected Visual Acuity (BCVA) [ Time Frame: Up to Month 24 ]BCVA will be 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 to be reported as number of letters read correctly by the participant.
- Change from Baseline in Retinal Sensitivity Assessed with Microperimetry [ Time Frame: Up to Month 24 ]Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.
- Change from Baseline in Contrast Sensitivity [ Time Frame: Up to Month 24 ]Change from baseline in contrast sensitivity in the study eye is measured using a Pelli Robson contrast sensitivity chart at 1 meter. The contrast sensitivity chart contains letters that are darkest at the top and then get progressively lighter. Scores range from 0 to 48 and are based on the number of letters read correctly. A negative change from baseline indicates a worsening in contrast sensitivity and a positive change from baseline indicates an improvement.
- Change from Baseline in Low Luminance Visual Acuity (LLVA) [ Time Frame: Up to Month 24 ]LLVA is measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. 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. LLVA is to be reported as number of letters read correctly by the participant.
- Change from Baseline in 25-item Visual Function Questionnaire (VFQ-25) Score [ Time Frame: Up to Month 24 ]VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. In this format scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score.
- Change from Baseline in Retinitis Pigmentosa (RP)-Specific Patient-Reported Outcome (PRO) Questionnaire [ Time Frame: Up to Month 24 ]
- Change from Baseline in EuroQol-5 Dimension 5-level (EQ-5D-5L) [ Time Frame: Up to Month 24 ]The widely validated EQ-5D includes 2 components, the EQ-5D descriptive system and the EQ VAS. The EQ-5D descriptive system provides a profile of the participant's health state in 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each dimension, the subject is instructed to indicate whether he or she has "no problems" (level 1), "slight problems" (level 2), "moderate problems" (level 3), "severe problems (level 4), or "extreme problems/inability" (level 5) on that day. For the EQ VAS, the participant is instructed to mark an "x" on a vertical scale at the point that best describes his or her own health on that day, where 0 represents the "worst health" he or she can imagine and 100 the "best health" he or she can imagine.
- Change from Baseline in Health Utilities Index Mark 3 (HUI3) [ Time Frame: Up to Month 24 ]The HUI3 is a generic 8-item survey, which provides descriptive evidence on multiple dimensions of health status, a score for each dimension of health, and a health-related quality of life score for overall health. Health dimensions include vision, hearing, speech, ambulation/mobility, pain, dexterity, emotion and cognition. Each dimension has five or six response options. Scores on individual items are combined to given a combined health state which can then be converted to health utilities. HUI3 score ranges from 0.36 (worst) to 1 (best).
- Change from Baseline in Visual Field Readings [ Time Frame: Up to Month 24 ]The progression of defects in visual fields will be assessed in both eyes using perimetry equipment.
- Change from Baseline in Microperimetry Readings [ Time Frame: Up to Month 24 ]MAIA microperimetry will be conducted for both eyes to assess changes other than retinal sensitivity.
- Change from Baseline in Multi-Luminance Mobility Test (MLMT) Readings [ Time Frame: Up to Month 24 ]The MLMT measures changes in functional vision, as assessed by the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination.
- Change from Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT) [ Time Frame: Up Month 24 ]SD-OCT measurements will be performed after dilation of the participant's pupil.
- Change from Baseline in Fundus Autofluorescence (FAF) [ Time Frame: Up to Month 24 ]Fundus Autofluorescence will be performed after dilation of the participant's pupil to assess changes in the area of viable retinal tissue.
- Change from Baseline in Fundus Photography [ Time Frame: Up to Month 24 ]Seven-field colour fundus photography will be used for both eyes. Fundus photography will be performed by certified technicians following pupil dilation. Stereo photos should be performed for fields 1, 2 and 3.
- Change from Baseline in Adaptive-Optics Scanning Laser Ophthalmoscopy (AO-SLO) [ Time Frame: Up to Month 24 ]Measurements will be performed after dilation of the participant's pupil.
- Change from Baseline in Intraocular Pressure as Assessed by Goldmann Applanation Tonometry [ Time Frame: Up to Month 24 ]Tonometry is used to measure eye pressure. After numbing the eye with eye drop anesthesia, the Goldmann tonometer presses against the eye. The force with which the eye pushes back is used to estimate the pressure inside the eye.
- Change from Baseline in Morphology of Eye as Assessed by Slit-lamp Examination [ Time Frame: Up to Month 24 ]The slit lamp exam usually forms part of a comprehensive eye exam. The participant will sit in a chair facing the slit lamp with their chin and forehead resting on a support. The doctor can use this instrument to observe the eyes in detail and determine whether or not there are any abnormalities.
- Change from Baseline in Morphology of Eye as Assessed by Dilated Ophthalmoscopy [ Time Frame: Up to Month 24 ]Dilated ophthalmoscopy is performed by dilating the participant's eye to see inside the fundus of the eye and other structures using an ophthalmoscope.
- Change from Baseline in Morphology of Eye as Assessed by Lens Opacities Classification System (LOCS) III Lens Grading [ Time Frame: Up to Month 24 ]LOCS III is a slit lamp based opacification grading method. Photographs of slit lamp cross-sections of the lens are used as references for grading nuclear opalescence (NO) and nuclear color (NC), and photographs of the lens seen by retroillumination are used as references for grading cortical (C) and posterior subcapsular (P) cataract. Opacification severity is graded on a decimal scale, scores can range from 0.1 to 6.9 for NO and NC and from 0.1 to 5.9 for C and P. For each opacification type the higher grading scores indicate greater severity.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04926129
|Contact: US Biogen Clinical Trial Centeremail@example.com|
|Contact: Global Biogen Clinical Trial Centerfirstname.lastname@example.org|
|United States, Arizona|
|Phoenix, Arizona, United States, 85020|
|United States, California|
|Los Angeles, California, United States, 90095-7000|
|San Francisco, California, United States, 94143|
|United States, Florida|
|Miami, Florida, United States, 33136|
|United States, New York|
|New York, New York, United States, 10032|
|United States, Oregon|
|Portland, Oregon, United States, 97239|
|United States, Texas|
|Dallas, Texas, United States, 75231|
|Houston, Texas, United States, 77030|
|United States, Utah|
|Salt Lake City, Utah, United States, 84132|
|United States, Wisconsin|
|Madison, Wisconsin, United States, 53705|
|Montréal, Quebec, Canada, H4A 3J1|
|Vancouver, Canada, V5Z3N9|
|Leeds, United Kingdom|
|Manchester, United Kingdom|
|Oxford, United Kingdom|
|Southampton, United Kingdom|
|Study Director:||Medical Director||Biogen|