Leber Congenital Amaurosis Clinical Trial
Official title:
Safety and Efficacy of LX101(AAV2-RPE65) in Patients With Leber's Congenital Amaurosis: a Randomised, Controlled, Open-label, Trial
The purpose of this study is to determine whether gene transfer(LX101) will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).
Leber Congenital Amaurosis (LCA)is a severe early onset retinal degeneration. Diagnosis is usually made during the first few months of life in infants who present with severely impaired vision, abnormal eye movements (nystagmus) and abnormal electroretinograms (ERG) indicating decreased retinal function. There is an inevitable progression to total blindness in these individuals due to death of photoreceptor cells. There is presently no treatment for this disease. The primary objective of this study is to determine the safety and tolerability of subretinal administration of Lx101(AAV2-RPE65) to subjects with LCA due to confirmed biallelic RPE65 mutations. The secondary objective is to assess the objective clinical measures of efficacy in human subjects. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT01496040 -
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
|
Phase 1/Phase 2 | |
| Completed |
NCT02970266 -
Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
|
N/A | |
| Completed |
NCT00516477 -
Safety Study in Subjects With Leber Congenital Amaurosis
|
Phase 1 | |
| Active, not recruiting |
NCT03920007 -
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
|
Phase 1/Phase 2 | |
| Recruiting |
NCT05906953 -
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT00999609 -
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
|
Phase 3 | |
| Active, not recruiting |
NCT01208389 -
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
|
Phase 1/Phase 2 | |
| Terminated |
NCT03913130 -
Extension Study to Study PQ-110-001 (NCT03140969)
|
Phase 1/Phase 2 | |
| Recruiting |
NCT04855045 -
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
|
Phase 2/Phase 3 | |
| Recruiting |
NCT02435940 -
Inherited Retinal Degenerative Disease Registry
|
||
| Completed |
NCT00749957 -
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
|
Phase 1/Phase 2 | |
| Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|
||
| Recruiting |
NCT05203939 -
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
|
Phase 1/Phase 2 | |
| Recruiting |
NCT06088992 -
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
|
Early Phase 1 | |
| Recruiting |
NCT06064565 -
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)
|
Early Phase 1 | |
| Completed |
NCT00821340 -
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
|
Phase 1 | |
| Active, not recruiting |
NCT03913143 -
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
|
Phase 2/Phase 3 | |
| Completed |
NCT02714816 -
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
|
||
| Completed |
NCT02781480 -
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
|
Phase 1/Phase 2 |