Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
NCT ID: NCT03278873
Last Updated: 2025-06-11
Study Results
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View full resultsBasic Information
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TERMINATED
34 participants
OBSERVATIONAL
2017-06-29
2024-04-04
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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Low dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3
Prior exposure to AAV-CNGA3 or AAV-CNGB3
Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.
Intermediate dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3
Prior exposure to AAV-CNGA3 or AAV-CNGB3
Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.
Other dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3
Prior exposure to AAV-CNGA3 or AAV-CNGB3
Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.
High dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3
Prior exposure to AAV-CNGA3 or AAV-CNGB3
Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.
Interventions
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Prior exposure to AAV-CNGA3 or AAV-CNGB3
Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.
Eligibility Criteria
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Inclusion Criteria
2. Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
3. Are willing to adhere to the protocol and long-term follow-up
Individuals will be excluded who:
Are unwilling or unable to meet the requirements of the study
3 Years
100 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 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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2016-003856-59
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MGT007
Identifier Type: -
Identifier Source: org_study_id
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