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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
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

Brief Summary:
The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).

Condition or disease Intervention/treatment
X-Linked Retinitis Pigmentosa Other: Other: Assessments

Detailed Description:
This study was previously posted by NightstaRx Ltd. In October, 2020, sponsorship of the trial was transferred to Biogen.

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Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Subgroup 1
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

Subgroup 2
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




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. Change from Baseline in Retinitis Pigmentosa (RP)-Specific Patient-Reported Outcome (PRO) Questionnaire [ Time Frame: Up to Month 24 ]
  5. 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.

  6. 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).

  7. 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.

  8. 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.

  9. 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.

  10. 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.

  11. 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.

  12. 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.

  13. 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.

  14. 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.

  15. 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.

  16. 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.

  17. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Up to 300 participants with X-linked Retinitis Pigmentosa (XLRP).
Criteria

Key Inclusion Criteria:

  1. Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  2. Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months
  3. Have an ETDRS BCVA in at least 1 eye of ≥34 letters (Equivalent to Snellen ≥ 6/60 or 20/200; decimal 0.1; LogMAR 1.0).
  4. Mean retinal sensitivity in the eligible eye as assessed by microperimetry:

    • Males with a mean retinal sensitivity of 68 loci ranging from ≥0.1 decibels (dB) and ≤20 dB.
    • Females with a mean retinal sensitivity of 68 loci ranging from ≥0.1 dB and ≤25 dB.
  5. If female, have symptomatic disease with impairment of visual function.

Key Exclusion Criteria:

  1. Have a history of amblyopia in the eligible eye.
  2. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may put the participant at risk because of participation in the study, may influence the results of the study, may influence the participant's ability to perform study diagnostic tests, or impact the participant's ability to participate in the study. This includes clinically significant cataracts.
  3. Have participated in another research study involving an investigational medicinal product in the past 12 weeks or received a gene/cell-based therapy at any time previously (including but not limited to Intelligent Implant System implantation, ciliary neurotrophic factor therapy, nerve growth factor therapy).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply


Information from the National Library of Medicine

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


Contacts
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Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
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United States, Arizona
Research Site Recruiting
Phoenix, Arizona, United States, 85020
United States, California
Research Site Recruiting
Los Angeles, California, United States, 90095-7000
Research Site Recruiting
San Francisco, California, United States, 94143
United States, Florida
Research Site Recruiting
Miami, Florida, United States, 33136
United States, New York
Research Site Recruiting
New York, New York, United States, 10032
United States, Oregon
Research Site Recruiting
Portland, Oregon, United States, 97239
United States, Texas
Research Site Recruiting
Dallas, Texas, United States, 75231
Research Site Recruiting
Houston, Texas, United States, 77030
United States, Utah
Research Site Recruiting
Salt Lake City, Utah, United States, 84132
United States, Wisconsin
Research Site Recruiting
Madison, Wisconsin, United States, 53705
Canada, Quebec
Research Site Recruiting
Montréal, Quebec, Canada, H4A 3J1
Canada
Research Site Recruiting
Vancouver, Canada, V5Z3N9
Finland
Research Site Recruiting
Helsinki, Finland
France
Research Site Recruiting
Montpellier, France
Germany
Research Site Recruiting
Bonn, Germany
Research Site Recruiting
Tübingen, Germany
Netherlands
Research Site Recruiting
Leiden, Netherlands
Research Site Recruiting
Nijmegen, Netherlands
United Kingdom
Research Site Recruiting
Leeds, United Kingdom
Research Site Recruiting
Manchester, United Kingdom
Research Site Recruiting
Oxford, United Kingdom
Research Site Recruiting
Southampton, United Kingdom
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Investigators
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Study Director: Medical Director Biogen
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Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT04926129    
Other Study ID Numbers: 274RP001 (NSR-XLRP-OS1)
First Posted: June 14, 2021    Key Record Dates
Last Update Posted: June 14, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biogen ( NightstaRx Ltd, a Biogen Company ):
RP GTPase regulator (RPGR)
Degenerative retinal disease
XLRP
Natural history
Rare disease
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn