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Clinical Trial Summary

The purpose of this study is to determine whether gene transfer will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).


Clinical Trial Description

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 AAV2-hRPE65v2 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT00516477
Study type Interventional
Source Spark Therapeutics
Contact
Status Completed
Phase Phase 1
Start date September 2007
Completion date March 20, 2018

See also
  Status Clinical Trial Phase
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Completed NCT02970266 - Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. N/A
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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Not yet recruiting NCT04731883 - RPE65 Gene Therapy (LX101)for Leber's Congenital Amaurosis N/A
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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
Completed NCT02714816 - Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65