Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
NCT ID: NCT02714816
Last Updated: 2023-08-14
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
37 participants
OBSERVATIONAL
2016-04-30
2023-07-22
Brief Summary
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Detailed Description
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In preparation for human clinical trials, a detailed prospective phenotypic study will be undertaken to investigate the natural history of RPE65-LCA. Such a study will help identify suitable patients for therapeutic intervention. Furthermore through greater phenotyping an optimal window for intervention and specific parameters to help quantify effect and identify clinical end points may have been ascertained .
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Minimum subject age of 3 years
* Able to give consent/parent or guardian able to give consent
Exclusion Criteria
* Have received a gene therapy treatment in both eyes
3 Years
ALL
No
Sponsors
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MeiraGTx UK II Ltd
INDUSTRY
Responsible Party
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Principal Investigators
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Michel Michealides, Prof
Role: PRINCIPAL_INVESTIGATOR
UCL/Moorfileds
Locations
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Kellogg Eye Center
Ann Arbor, Michigan, United States
Moorfields Eye Hospital
London, , United Kingdom
Countries
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References
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Kumaran N, Georgiou M, Bainbridge JWB, Bertelsen M, Larsen M, Blanco-Kelly F, Ayuso C, Tran HV, Munier FL, Kalitzeos A, Michaelides M. Retinal Structure in RPE65-Associated Retinal Dystrophy. Invest Ophthalmol Vis Sci. 2020 Apr 9;61(4):47. doi: 10.1167/iovs.61.4.47.
Kumaran N, Rubin GS, Kalitzeos A, Fujinami K, Bainbridge JWB, Weleber RG, Michaelides M. A Cross-Sectional and Longitudinal Study of Retinal Sensitivity in RPE65-Associated Leber Congenital Amaurosis. Invest Ophthalmol Vis Sci. 2018 Jul 2;59(8):3330-3339. doi: 10.1167/iovs.18-23873.
Other Identifiers
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MGT005
Identifier Type: -
Identifier Source: org_study_id
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