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16 studies found for:    "Leber congenital amaurosis" OR "Leber Congenital Amaurosis"
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Rank Status Study
1 Active, not recruiting Safety Study in Subjects With Leber Congenital Amaurosis
Condition: Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
2 Completed Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
Condition: Leber Congenital Amaurosis
Intervention:
3 Active, not recruiting
Has Results
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Condition: Leber Congenital Amaurosis
Intervention: Biological: rAAV2-CB-hRPE65
4 Recruiting Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
Condition: Leber Congenital Amaurosis
Intervention:
5 Active, not recruiting Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Condition: Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
6 Active, not recruiting Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations;   Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
7 Recruiting Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Condition: Leber Congenital Amaurosis
Intervention: Genetic: AAV2/5 OPTIRPE65
8 Recruiting Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
Condition: Leber Congenital Amaurosis
Intervention: Genetic: rAAV2-hRPE65
9 Completed Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
Conditions: LCA (Leber Congenital Amaurosis);   RP (Retinitis Pigmentosa)
Intervention: Drug: QLT091001
10 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
Conditions: LCA (Leber Congenital Amaurosis);   RP (Retinitis Pigmentosa)
Intervention: Drug: QLT091001
11 Completed Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Condition: Leber Congenital Amaurosis
Intervention: Drug: rAAV2/4.hRPE65
12 Completed Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Condition: Retinal Degeneration
Intervention: Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
13 Not yet recruiting Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Conditions: Leber Congenital Amaurosis (LCA);   Eye Diseases;   Eye Diseases, Hereditary;   Retinal Diseases
Intervention: Genetic: AAV2/5-OPTIRPE65
14 Completed Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
Conditions: Leber Congenital Amaurosis (LCA);   Retinitis Pigmentosa (RP)
Intervention: Other: No treatment: retrospective chart review
15 Active, not recruiting Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
Conditions: Amaurosis of Leber;   Retinal Diseases
Intervention: Genetic: rAAV2-CBSB-hRPE65
16 Recruiting Inherited Retinal Degenerative Disease Registry
Conditions: Eye Diseases Hereditary;   Retinal Disease;   Achromatopsia;   Bardet-Biedl Syndrome;   Bassen-Kornzweig Syndrome;   Batten Disease;   Best Disease;   Choroidal Dystrophy;   Choroideremia;   Cone Dystrophy;   Cone-Rod Dystrophy;   Congenital Stationary Night Blindness;   Enhanced S-Cone Syndrome;   Fundus Albipunctatus;   Goldmann-Favre Syndrome;   Gyrate Atrophy;   Juvenile Macular Degeneration;   Kearns-Sayre Syndrome;   Leber Congenital Amaurosis;   Refsum Syndrome;   Retinitis Pigmentosa;   Retinitis Punctata Albescens;   Retinoschisis;   Rod-Cone Dystrophy;   Rod Dystrophy;   Rod Monochromacy;   Stargardt Disease;   Usher Syndrome
Intervention:

Study has passed its completion date and status has not been verified in more than two years.