Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy|
- The Best Corrected Visual Acuity(BCVA) [ Time Frame: Up to 3 years ] [ Designated as safety issue: Yes ]
- Results of CD3/CD4/CD8 Test [ Time Frame: up to 6 months ] [ Designated as safety issue: Yes ]The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
- Intraocular Pressure; [ Time Frame: Up to 3 years ] [ Designated as safety issue: Yes ]
- Neutralizing Antibody Assay [ Time Frame: up to 3 years ] [ Designated as safety issue: Yes ]The mean of Neutralizing antibody assay of 8 patients before and after treatment
- Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test [ Time Frame: Up to 3 years ] [ Designated as safety issue: No ]Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
- Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal) [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
- Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal) [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
|Study Start Date:||April 2011|
|Study Completion Date:||November 2015|
|Primary Completion Date:||November 2015 (Final data collection date for primary outcome measure)|
Other Name: rAAV2-ND4 gene therapy
Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.
Material and Method Seven patients with 11778 LHON mutation were randomly treated with a Single IVT Injection of recombinant Adeno-Associated Virus-NADH dehydrogenase, subunit 4 (complex I)（rAAV2-ND4)（0.05ml).The dose was 5 × 10^9 vg/0.05 mL for patients younger than 12 years old, and 1 × 10^10 vg/0.05 mL for patients older than 12 years old. The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, electroretinograms(ERG), retinal nerve fiber layer(RNFL)and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01267422
|Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology|
|Wu Han, Hubei, China, 430030|
|Study Chair:||bin Li, PhD,MD||Deputy Director of Ophthalmology|