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Clinical Trial Summary

The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.


Clinical Trial Description

n/a


Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label


Related Conditions & MeSH terms


NCT number NCT01496040
Study type Interventional
Source Nantes University Hospital
Contact
Status Completed
Phase Phase 1/Phase 2
Start date September 2011
Completion date August 2014

See also
  Status Clinical Trial Phase
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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
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Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
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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 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