December 27, 2010
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December 28, 2010
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May 21, 2015
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May 2, 2016
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January 31, 2018
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April 2011
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November 2015 (Final data collection date for primary outcome measure)
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The visual acuity [ Time Frame: 6 month ] Vision testing before and after treatment
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Complete list of historical versions of study NCT01267422 on ClinicalTrials.gov Archive Site
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- 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.
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- Intraocular pressure [ Time Frame: 6 month ]
Intraocular pressure testing before and after treatment
- VEP [ Time Frame: 6 month ]
VEP testing before and after treatment
- computerized visual field [ Time Frame: 6 month ]
computerized visual field testing before and after treatment
- OCT [ Time Frame: 6 month ]
OCT testing before and after treatment
- ERG [ Time Frame: 6 month ]
ERG testing before and after treatment
- Liver and kidney function in plasma [ Time Frame: 6 month ]
Liver and kidney function in plasma testing before and after treatment
- ELISA [ Time Frame: 6 month ]
Antibodies against AAV2 capsid was analyzed by ELISA using serum samples collected before injection and different time points after the injection.
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Not Provided
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Not Provided
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Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) |
Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy |
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation. |
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. |
Interventional |
Not Applicable |
Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
Leber Hereditary Optic Neuropathy |
Drug: rAAV2-ND4
injection
Other Name: rAAV2-ND4 gene therapy |
Experimental: rAAV2-ND4
injection
Intervention: Drug: rAAV2-ND4 |
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Completed |
9 |
6 |
November 2015 |
November 2015 (Final data collection date for primary outcome measure) |
Inclusion Criteria:
- comply with Leber hereditary optic neuropathy diagnostic criteria.
- in patients with informed consent, voluntary participation.
- signed informed consent.
- 8 ≤ Age ≤ 60 years old, good health, the patient can tolerate local anesthesia surgery.
- to comply with doctor's instructions, can in the time of referral.
Exclusion Criteria:
- Cardiopulmonary and renal function in severe weakness, cancer, a variety of bleeding disorders, acute sensing disease, high fever, high fever disease, women during pregnancy, heart disease, such as post-operative recovery period.
- Are participating in other clinical studies of patients.
- Patients with mental disorders.
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Sexes Eligible for Study: |
All |
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8 Years to 60 Years (Child, Adult) |
No |
Contact information is only displayed when the study is recruiting subjects |
China |
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NCT01267422 |
RAVCT-2 |
Yes |
Not Provided |
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Bin Li, Huazhong University of Science and Technology |
Bin Li |
Huazhong University of Science and Technology |
Study Chair: |
bin Li, PhD,MD |
Deputy Director of Ophthalmology |
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Huazhong University of Science and Technology |
January 2018 |