Leber Congenital Amaurosis - 19 Studies Found
Completed |
: 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 :
: 2009-11-12 : Drug: QLT091001 oral QLT091001 administered once daily for 7 days |
Completed |
: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 : Leber Congenital Amaurosis : 2011-11-24 : Drug: rAAV2/4.hRPE65 One injection in on eye |
Completed |
: Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis : Retinal Degeneration : 2008-03-20 : Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) Single subretinal injection of vector suspension; up to 3x |
Recruiting |
: Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) :
: 2015-10-09 : Other: No treatment: retrospective chart review |
Active, not recruiting |
: Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations :
: 2007-05-31 : Genetic: rAAV2-CBSB-hRPE65 One or two, uniocular, subretinal injections; relative doses: 0.3X (Cohort 1) |
Recruiting |
: Inherited Retinal Degenerative Disease Registry :
: 2015-04-03 |
NOT_YET_RECRUITING |
: A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis : : 2024-05-13 : Low dose Medium dose High dose |
RECRUITING |
: Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) : Leber Congenital Amaurosis : 2024-05-13 : Low dose Medium dose High dose |