Status | Study |
Completed |
Study Name: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 Condition: Leber Congenital Amaurosis Date: 2011-11-24 Interventions: Drug: rAAV2/4.hRPE65 One injection in on eye |
Active, not recruiting |
Study Name: Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Condition: Leber Congenital Amaurosis Date: 2010-09-22 Interventions: Biological: AAV2-hRPE65v2 One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector to the c |
Completed |
Study Name: Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations Condition: LCA (Leber Congenital Amaurosis) RP (Retinitis Pigmentosa) Date: 2009-11-12 Interventions: Drug: QLT091001 oral QLT091001 administered once daily for 7 days |
Active, not recruiting |
Study Name: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Condition: Inherited Retinal Dystrophy Due to RPE65 Mutations Leber Congenital Am Date: 2009-10-21 Interventions: Biological: AAV2-hRPE65v2 Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector |
Recruiting |
Study Name: Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Condition: Leber Congenital Amaurosis Date: 2009-01-12 Interventions: Genetic: rAAV2-hRPE65 Uniocular subretinal injections; relative doses: Cohort 1 - basic (lowest) viral d |
Active, not recruiting |
Study Name: Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis Condition: Leber Congenital Amaurosis Date: 2008-09-08 Interventions: Biological: rAAV2-CB-hRPE65 Recombinant adeno-associated virus vector expressing RPE65 |
Completed |
Study Name: Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis Condition: Retinal Degeneration Date: 2008-03-20 Interventions: Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) Single subretinal injection of vector suspension; up to 3x |
Active, not recruiting |
Study Name: Safety Study in Subjects With Leber Congenital Amaurosis Condition: Leber Congenital Amaurosis Date: 2007-08-13 Interventions: Biological: AAV2-hRPE65v2 Subjects will be dosed unilaterally (one eye) beginning with the lowest dose. |
Active, not recruiting |
Study Name: Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations Condition: Amaurosis of Leber Retinal Diseases Date: 2007-05-31 Interventions: Genetic: rAAV2-CBSB-hRPE65 One or two, uniocular, subretinal injections; relative doses: 0.3X (Cohort 1) |