Leber Congenital Amaurosis Clinical Trial
Official title:
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
MGT005 is a natural history study to collect longitudinal prospective data from patients with Leber Congenital Amaurosis associated with defects in RPE65.
Leber Congenital Amaurosis (LCA) is a diagnosis for a group of severe, autosomal recessively inherited rod - cone dystrophies that typically result in complete visual loss in the third or fourth decade of life. One form, LCA2, is caused by a mutation in the gene encoding RPE56, an RPE-specific 65-kDa isomerase. Non-functional RPE65 results in photoreceptor cells that are unable to respond to light resulting in these patients being visually impaired. In preparation for human clinical trials, a detailed prospective phenotypic study will be undertaken to investigate the natural history of RPE65-LCA. Such a study will help identify suitable patients for therapeutic intervention. Furthermore through greater phenotyping an optimal window for intervention and specific parameters to help quantify effect and identify clinical end points may have been ascertained . ;
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
|
||
Not yet recruiting |
NCT04731883 -
RPE65 Gene Therapy (LX101)for Leber's Congenital Amaurosis
|
N/A | |
Recruiting |
NCT05203939 -
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
|
Phase 1/Phase 2 | |
Recruiting |
NCT06064565 -
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)
|
Early Phase 1 | |
Recruiting |
NCT06088992 -
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 |
NCT02781480 -
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
|
Phase 1/Phase 2 |