Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
11 participants
INTERVENTIONAL
2019-08-12
2021-06-10
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
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Low dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single low dose AAV CNGA3
adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Intermediate dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single intermediate dose AAV CNGA3
adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
High dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single high dose AAV CNGA3
adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Interventions
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adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Eligibility Criteria
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Inclusion Criteria
* Have achromatopsia confirmed by a retinal specialist investigator
Exclusion Criteria
* Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
* Have any other condition that the investigator considers makes them inappropriate for entry into the trial
3 Years
ALL
No
Sponsors
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Janssen Research & Development, LLC
INDUSTRY
MeiraGTx UK II Ltd
INDUSTRY
Responsible Party
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Principal Investigators
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James Bainbridge
Role: PRINCIPAL_INVESTIGATOR
Chief Investigator
Locations
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Kellogg Eye Center
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
Document Type: Statistical Analysis Plan
Other Identifiers
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MGT012
Identifier Type: -
Identifier Source: org_study_id
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