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. ;
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