2006-001571-37: AN OPEN-LABEL DOSE ESCALATION STUDY OF AN ADENO-ASSOCIATED VIRUS VECTOR (AAV2/2-hRPE65p-hRPE65) FOR GENE THERAPY OF SEVERE EARLY-ONSET RETINAL DEGENERATION |
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| Ongoing | 2 | 12 | Europe | tgAAG76, | University College London | The condition to be investigated is severe early-onset inherited retinal degeneration due to defects in the gene encoding RPE65 | | | | |
NCT00643747: Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis |
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| Completed | 1/2 | 12 | Europe | tgAAG76 (rAAV 2/2.hRPE65p.hRPE65), rAAV 2/2.hRPE65p.hRPE65 | University College, London, Moorfields Eye Hospital NHS Foundation Trust, Targeted Genetics Corporation | Retinal Degeneration | 12/14 | 12/14 | | |
