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Clinical Trials/NCT02946879
NCT02946879
Completed
Not Applicable

Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)

MeiraGTx UK II Ltd2 sites in 2 countries15 target enrollmentNovember 2016
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ConditionsLeber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, HereditaryRetinal Diseases

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Leber Congenital Amaurosis (LCA)
Sponsor
MeiraGTx UK II Ltd
Enrollment
15
Locations
2
Primary Endpoint
Incidence of Adverse Events related to the treatment
Status
Completed
Last Updated
2 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSitesSimilar Trials

Overview

Brief Summary

This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.

Detailed Description

The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.

Registry
clinicaltrials.gov
Start Date
November 2016
End Date
July 2023
Last Updated
2 years ago
Study Type
Observational
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • •Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65

Exclusion Criteria

  • •Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Outcomes

Primary Outcomes

Incidence of Adverse Events related to the treatment

Time Frame: 5 years

Safety is defined as the absence of ATIMP-related safety events

Secondary Outcomes

  • Improvement in the retinal function(5 years)
  • Improvement in the visual function(5 years)
  • Improvement in quality of life(5 years)

Study Sites (2)

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