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Clinical Trial Summary

The study is a follow-on to a Phase 1 dose-escalation and safety study.


Clinical Trial Description

The study is a follow-on to a Phase 1 dose-escalation and safety study (closed to enrollment as of June 2009). Up to twelve adults and children with a molecular diagnosis of biallelic RPE65 mutations, who have participated in the earlier Phase 1 study, and who meet all study eligibility criteria, will receive AAV2-hRPE65v2 vector in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of AAV2-hRPE65v2. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01208389
Study type Interventional
Source Spark Therapeutics
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date November 2010
Completion date June 2030

See also
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