Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Study Results

Results pending

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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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

41 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-06-30

Study Completion Date

2030-01-31

Brief Summary

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Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject. The study is a non-interventional, follow-up study of subjects who participated in previous AAV2-hRPE65v2 gene therapy clinical trials.

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Detailed Description

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This is an observational follow-up study of subjects who participated in previous Phase 1 and Phase 3 clinical trials of AAV2-hRPE65v2 gene therapy (voretigene neparvovec-rzyl) to evaluate long term durability and safety for 15 years after subretinal administration.

Conditions

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Inherited Retinal Dystrophy Due to RPE65 Mutations

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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AAV2-hRPE65v2

Intervention Type BIOLOGICAL

Other Intervention Names

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Voretigene Neparvovec-rzyl

Eligibility Criteria

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Inclusion Criteria

1\. Subjects who participated in prior subretinal AAV2-hRPE65v2 gene therapy clinical studies

Exclusion Criteria

1. Subjects who will not consent for study.
2. Subjects who the investigators believe are not capable of performing study assessments

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Albert Maguire, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pennsylvania

Stephen Russell, MD

Role: PRINCIPAL_INVESTIGATOR

University of Iowa

Other Identifiers

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AAV2-hRPE65v2-LTFU-01

Identifier Type: -

Identifier Source: org_study_id

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