Indication
Leber congenital amaurosis
9 clinical trials
12 products
Product
ATSN-101Clinical trial
A Phase 1/2 Dose Escalation Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2DStatus: Active (not recruiting), Estimated PCD: 2023-05-19
Product
PrednisoneProduct
Triamcinalone AcetonideProduct
PrednisoloneProduct
Trimethoprim + Polymyxin BProduct
TriamcinaloneClinical trial
A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]Status: Active (not recruiting), Estimated PCD: 2015-07-01
Clinical trial
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutation(s) in CEP290 GeneStatus: Recruiting, Estimated PCD: 2024-12-01
Product
OCU400Clinical trial
An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 GeneStatus: Completed, Estimated PCD: 2019-10-02
Product
QR-110Product
HG004Clinical trial
A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)Status: Recruiting, Estimated PCD: 2025-12-01
Clinical trial
An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 GeneStatus: Terminated, Estimated PCD: 2022-10-03
Product
Voretigene neparvovecClinical trial
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 StudyStatus: Active (not recruiting), Estimated PCD: 2030-03-01
Clinical trial
An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis With RPE65 Mutation (LCA2)Status: Recruiting, Estimated PCD: 2024-01-30
Clinical trial
An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)Status: Not yet recruiting, Estimated PCD: 2028-09-30
Product
LX101