Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01267422|
Recruitment Status : Completed
First Posted : December 28, 2010
Results First Posted : May 2, 2016
Last Update Posted : January 31, 2018
|Condition or disease||Intervention/treatment||Phase|
|Leber Hereditary Optic Neuropathy||Drug: rAAV2-ND4||Not Applicable|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy|
|Study Start Date :||April 2011|
|Actual Primary Completion Date :||November 2015|
|Actual Study Completion Date :||November 2015|
Other Name: rAAV2-ND4 gene therapy
- The Best Corrected Visual Acuity(BCVA) [ Time Frame: Up to 3 years ]
- Results of CD3/CD4/CD8 Test [ Time Frame: up to 6 months ]The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
- Intraocular Pressure; [ Time Frame: Up to 3 years ]
- Neutralizing Antibody Assay [ Time Frame: up to 3 years ]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 ]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 ]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 ]VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01267422
|Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology|
|Wuhan, Hubei, China, 430030|
|Study Chair:||bin Li, PhD,MD||Deputy Director of Ophthalmology|