Safety and Efficacy Study of Gene Therapy for RPE65-mediated Inherited Retinal Disease

Registry ID
EUCTR2016-002109-20
Source registry
EUCTR
Phase
PHASE3
Sponsor
Spark Therapeutics, Inc.
Last update
2026-08-16

Conditions

Summary

The primary objective of this study is to determine whether non-simultaneous, bilateral, subretinal administration of AAV2-hRPE65v2 improves the ability to navigate as measured by mobility testing in adults and children, three years of age or older. Mobility test performance one year following vector administrations will be compared to subjects’ pre-administration, baseline mobility test performance; independent, masked reviewers will be trained to assess ability to navigate.

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