Natural History of the Progression of X-Linked Retinitis Pigmentosa
NCT ID: NCT04926129
Last Updated: 2023-02-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
201 participants
OBSERVATIONAL
2017-09-13
2022-12-16
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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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: Assessments
Administered as specified in the treatment arm.
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: Assessments
Administered as specified in the treatment arm.
Interventions
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Other: Assessments
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
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).
7 Years
ALL
No
Sponsors
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NightstaRx Ltd, a Biogen Company
OTHER
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Research Site
Phoenix, Arizona, United States
Research Site
Los Angeles, California, United States
Research Site
San Francisco, California, United States
Research Site
Miami, Florida, United States
Research Site
New York, New York, United States
Research Site
Portland, Oregon, United States
Research Site
Dallas, Texas, United States
Research Site
Houston, Texas, United States
Research Site
Salt Lake City, Utah, United States
Research Site
Madison, Wisconsin, United States
Research Site
Montreal, Quebec, Canada
Research Site
Vancouver, , Canada
Research Site
Helsinki, , Finland
Research Site
Montpellier, , France
Research Site
Bonn, , Germany
Research Site
Tübingen, , Germany
Research Site
Leiden, , Netherlands
Research Site
Nijmegen, , Netherlands
Research Site
Leeds, , United Kingdom
Research Site
Manchester, , United Kingdom
Research Site
Oxford, , United Kingdom
Research Site
Southampton, , United Kingdom
Countries
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References
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von Krusenstiern L, Liu J, Liao E, Gow JA, Chen G, Ong T, Lotery AJ, Jalil A, Lam BL, MacLaren RE; XIRIUS Part 1 Study GroupXOLARIS Study Group. Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations. JAMA Ophthalmol. 2023 Mar 1;141(3):275-283. doi: 10.1001/jamaophthalmol.2022.6254.
Other Identifiers
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NSR-XLRP-OS1
Identifier Type: OTHER
Identifier Source: secondary_id
274RP001
Identifier Type: -
Identifier Source: org_study_id
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