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Report for Clinical Trial NCT03153293

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy

This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.

NCT03153293 Leber Hereditary Optic Neuropathy
MeSH: Optic Nerve Diseases Optic Atrophy, Hereditary, Leber
HPO: Leber optic atrophy

1 Interventions

Name: rAAV2-ND4

Description: a Single IVT Injection

Type: Drug

rAAV2-ND4


Primary Outcomes

Description: The Best Corrected Visual Acuity

Measure: BCVA

Time: Change from Baseline at 12 months .

Measure: Computerized Visual Field

Time: Change from Baseline at 12 months .

Secondary Outcomes

Description: visual evoked potential

Measure: VEP

Time: Change from Baseline at 12 months .

Description: retinal nerve fiber layer

Measure: RNFL

Time: Change from Baseline at 12 months .

Measure: Liver function in plasma

Time: Change from Baseline at 12 months .

Measure: kidney function in plasma

Time: Change from Baseline at 12 months .

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 G11778A

Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA. --- G11778A ---

This is a multi - center , prospective study of 142 patients with the G11778A mutation in Mt-DNA . --- G11778A ---



HPO Nodes


HPO:
Leber optic atrophy
Genes 11
ND2 ATP6 ND4L ND5 CYTB ND1 TBC1D24 ND4 CPLX1 ND6 COX3