Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
NCT ID: NCT02781480
Last Updated: 2021-07-12
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2016-04-30
2018-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Low dose AAV-RPE65
Subretinal administration of a single low dose of range AAV-RPE65
AAV RPE65
Comparison of different dosages of AAV RPE65
Intermediate dose AAV-RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
AAV RPE65
Comparison of different dosages of AAV RPE65
High dose AAV-RPE65
Subretinal administration of a single high dose of range AAV-RPE65
AAV RPE65
Comparison of different dosages of AAV RPE65
Interventions
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AAV RPE65
Comparison of different dosages of AAV RPE65
Eligibility Criteria
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Inclusion Criteria
* Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Exclusion Criteria
* Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
3 Years
ALL
No
Sponsors
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MeiraGTx UK II Ltd
INDUSTRY
Responsible Party
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Locations
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Kellogg Eye Centre, University of Michigan
Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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MGT003
Identifier Type: -
Identifier Source: org_study_id
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