An interventional study of rAAV2-ND4 in Leber Hereditary Optic Neuropathy, sponsored by Bin Li. Completed at 1 site in China. Open to participants aged 8 Years to 60 Years. Per ClinicalTrials.gov, last updated 2018-01-31.
Sponsored by Bin Li · Not applicable, Interventional, and Treatment
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.
1,003 studies on the registry are indexed under Peripheral Nervous System Diseases; 177 are open to participants now.
This study's enrollment of 9 is below the median of 60 across 768 interventional studies indexed under Peripheral Nervous System Diseases.
Browse Peripheral Nervous System Diseases studies →Bin Li is the lead sponsor of 2 studies on the registry; none are open to participants now.
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Exclusion Criteria:
injection
Drug: rAAV2-ND4
injection
Also known as: rAAV2-ND4 gene therapy
The Best Corrected Visual Acuity(BCVA)
Time frame: Up to 3 years
Results of CD3/CD4/CD8 Test
The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
Time frame: up to 6 months
Intraocular Pressure;
Time frame: Up to 3 years
Neutralizing Antibody Assay
The mean of Neutralizing antibody assay of 8 patients before and after treatment
Time frame: up to 3 years
Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test
Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
Time frame: Up to 3 years
Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)
MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
Time frame: up to 3 years
Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)
VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
Time frame: up to 3 years
Patients diagnosed with LHON (with a verified point mutation at the 11778 nucleotide site)
| Milestone | Participants Receiving Treatment in Left Eye | Participants Receiving Treatment in Right Eye |
|---|---|---|
| Started | 5 | 4 |
| Completed | 5 | 4 |
| Not completed | 0 | 0 |
| Milestone | Participants Receiving Treatment in Left Eye | Participants Receiving Treatment in Right Eye |
|---|---|---|
| Started | 5 | 4 |
| Completed | 5 | 4 |
| Not completed | 0 | 0 |
| logMAR | BCVA of Un-treated Eyes | BCVA of Treated Eyes |
|---|---|---|
| before treament | 1.40 ± 0.55 | 1.69 ± 0.43 |
| 1 months after treament | 1.36 ± 0.58 | 1.58 ± 0.44 |
| 3 months after treament | 1.31 ± 0.58 | 1.38 ± 0.46 |
| 6 months after treament | 1.20 ± 0.68 | 1.23 ± 0.60 |
| 9 months after treament | 1.23 ± 0.62 | 1.27 ± 0.58 |
The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
| Percentage of total cells | The Mean Percentage of CD3+/CD4+/CD8+ Before Treatment | The Mean Percentage of CD3+/CD4+/CD8+ After Treatment |
|---|---|---|
| Values of CD3+ | 51 (50 to 84) | 53 (50 to 84) |
| Values of CD4+ | 26 (20 to 51) | 20 (20 to 51) |
| Values of CD8+ | 20 (12 to 45) | 13 (12 to 45) |
| mmHg | IOP Before Treatment | IOP After Treatment |
|---|---|---|
| Intraocular Pressure; | 14 (10 to 21) | 16 (10 to 21) |
The mean of Neutralizing antibody assay of 8 patients before and after treatment
| titer | The Mean of Neutralizing Antibody Assay Before Treatment | The Mean of Neutralizing Antibody Assay After Treatment |
|---|---|---|
| Neutralizing Antibody Assay | 0.9221 ± 0.142437 | 0.8723 ± 0.201054 |
Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
| Micrometer | Average RNFL Thickness Before Treatment | Average RNFL Thickness After Treatment |
|---|---|---|
| injected eye | 47.3125 ± 7.87259 | 46.7813 ± 7.5397 |
| uninjected eye | 50.1875 ± 13.402 | 47.9688 ± 9.7031 |
MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
| dB | Mean MD Before Treatment | Mean MD After Treatment |
|---|---|---|
| injected eye | -29.377 ± 5.16805 | -23.813 ± 7.27537 |
| uninjected eye | -28.701 ± 3.65595 | -23.427 ± 8.15747 |
VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
| Percentage of normal | Mean VFI Before Treatment | Mean VFI After Treatment |
|---|---|---|
| injected eye | 11 ± 0.14513 | 28 ± 0.23305 |
| uninjected eye | 10.5 ± 0.28714 | 25 ± 0.24804 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Short-term Affects | — | 0/9 (0%) | 0/9 (0%) |
| Long-term Affects | — | 0/9 (0%) | 0/9 (0%) |
| Age, Continuous(years) | All Study Participants |
|---|---|
| Median | 15 (7.65 to 30.79) |
| Sex: Female, Male(Participants) | All Study Participants |
|---|---|
| Female | 2 |
| Male | 7 |
| Ethnicity (NIH/OMB)(Participants) | All Study Participants |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 9 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | All Study Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 9 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | All Study Participants |
|---|---|
| China | 9 |
Plan to share: No
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