11 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 Active, not recruiting 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
3 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
4 Recruiting Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
Condition: Leber Congenital Amaurosis
Intervention: Genetic: rAAV2-hRPE65
5 Active, not recruiting 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
6 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
7 Unknown  Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Condition: Leber Congenital Amaurosis
Intervention: Drug: rAAV2/4.hRPE65
8 Recruiting Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Condition: Inherited Retinal Dystrophy Due to RPE65 Mutations
Intervention: Biological: AAV2-hRPE65v2
9 Active, not recruiting Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Condition: Retinal Degeneration
Intervention: Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
10 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
11 Recruiting Clinical and Molecular Investigations Into Ciliopathies
Conditions: Autosomal Recessive Polycystic Kidney Disease;   Congenital Hepatic Fibrosis;   Caroli's Disease;   Polycystic Kidney Disease;   Joubert Syndrome;   Cerebro-Oculo-Renal Syndromes;   COACH Syndrome;   Senior-Loken Syndrome;   Dekaban-Arima Syndrome;   Cogan Oculomotor Apraxia;   Nephronophthisis;   Bardet-Biedl Syndrome;   Alstrom Syndrome;   Oral-Facial-Digital Syndrome
Intervention:

Indicates status has not been verified in more than two years