Trial Outcomes & Findings for Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (NCT NCT01267422)
NCT ID: NCT01267422
Last Updated: 2018-01-31
Results Overview
COMPLETED
NA
9 participants
Up to 3 years
2018-01-31
Participant Flow
Patients diagnosed with LHON (with a verified point mutation at the 11778 nucleotide site)
All patients were administered rAAV2-ND4 by intravitreal injection to one eye and then followed for 36 months.
Participant milestones
| Measure |
Participants Receiving Treatment in Left Eye
5 participants receiving treatment in left eye
|
Participants Receiving Treatment in Right Eye
4 participants receiving treatment in right eye
|
|---|---|---|
|
Intravitreal rAAV2-ND4 Injection
STARTED
|
5
|
4
|
|
Intravitreal rAAV2-ND4 Injection
COMPLETED
|
5
|
4
|
|
Intravitreal rAAV2-ND4 Injection
NOT COMPLETED
|
0
|
0
|
|
Safety and Efficacy Study
STARTED
|
5
|
4
|
|
Safety and Efficacy Study
COMPLETED
|
5
|
4
|
|
Safety and Efficacy Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
Baseline characteristics by cohort
| Measure |
All Study Participants
n=9 Participants
Age,Gender,Ethnicity,Race,Region of Enrollment
|
|---|---|
|
Age, Continuous
|
15 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
China
|
9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3 yearsPopulation: The data are expressed as mean±standard error. Comparisons before and after treatment of the BCVA were analyzed using the paired t-test.A probability (P) value of less than 0.05 was considered statistically significant.Lower logMAR represents a better outcome.
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=9 logMAR
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=9 logMAR
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
The Best Corrected Visual Acuity(BCVA)
before treament
|
1.40 logMAR
Standard Error 0.55
|
1.69 logMAR
Standard Error 0.43
|
|
The Best Corrected Visual Acuity(BCVA)
1 months after treament
|
1.36 logMAR
Standard Error 0.58
|
1.58 logMAR
Standard Error 0.44
|
|
The Best Corrected Visual Acuity(BCVA)
3 months after treament
|
1.31 logMAR
Standard Error 0.58
|
1.38 logMAR
Standard Error 0.46
|
|
The Best Corrected Visual Acuity(BCVA)
6 months after treament
|
1.20 logMAR
Standard Error 0.68
|
1.23 logMAR
Standard Error 0.60
|
|
The Best Corrected Visual Acuity(BCVA)
9 months after treament
|
1.23 logMAR
Standard Error 0.62
|
1.27 logMAR
Standard Error 0.58
|
PRIMARY outcome
Timeframe: up to 6 monthsThe mean percentage of CD3+/CD4+/CD8+ test before and after treatment
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=9 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=9 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Results of CD3/CD4/CD8 Test
Values of CD3+
|
51 Percentage of total cells
Interval 50.0 to 84.0
|
53 Percentage of total cells
Interval 50.0 to 84.0
|
|
Results of CD3/CD4/CD8 Test
Values of CD4+
|
26 Percentage of total cells
Interval 20.0 to 51.0
|
20 Percentage of total cells
Interval 20.0 to 51.0
|
|
Results of CD3/CD4/CD8 Test
Values of CD8+
|
20 Percentage of total cells
Interval 12.0 to 45.0
|
13 Percentage of total cells
Interval 12.0 to 45.0
|
SECONDARY outcome
Timeframe: Up to 3 yearsOutcome measures
| Measure |
BCVA of Un-treated Eyes
n=9 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=9 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Intraocular Pressure;
|
14 mmHg
Interval 10.0 to 21.0
|
16 mmHg
Interval 10.0 to 21.0
|
SECONDARY outcome
Timeframe: up to 3 yearsPopulation: Neutralizing antibody assay of 8 patients except Patient 1 because he once accepted the other eye's treatment
The mean of Neutralizing antibody assay of 8 patients before and after treatment
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=8 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=8 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Neutralizing Antibody Assay
|
0.9221 titer
Standard Deviation 0.142437
|
0.8723 titer
Standard Deviation 0.201054
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: RNFL thickness of 8 patients except Patient 1 because he once accepted the other eye's treatment
Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=8 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=8 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test
injected eye
|
47.3125 Micrometer
Standard Deviation 7.87259
|
46.7813 Micrometer
Standard Deviation 7.5397
|
|
Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test
uninjected eye
|
50.1875 Micrometer
Standard Deviation 13.402
|
47.9688 Micrometer
Standard Deviation 9.7031
|
SECONDARY outcome
Timeframe: up to 3 yearsPopulation: Patient 2 refused the vision field test,there are MD outcome measure of 8 patients.
MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=8 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=8 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)
injected eye
|
-29.377 dB
Standard Deviation 5.16805
|
-23.813 dB
Standard Deviation 7.27537
|
|
Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)
uninjected eye
|
-28.701 dB
Standard Deviation 3.65595
|
-23.427 dB
Standard Deviation 8.15747
|
SECONDARY outcome
Timeframe: up to 3 yearsPopulation: Patient 2 refused the vision field test,there are VFI outcome measure of 8 patients.
VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
Outcome measures
| Measure |
BCVA of Un-treated Eyes
n=8 Participants
BCVA of un-treated eyes in 9 patients
|
BCVA of Treated Eyes
n=8 Participants
BCVA of trearted eyes in 9 patients
|
|---|---|---|
|
Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)
injected eye
|
11 Percentage of normal
Standard Deviation 0.14513
|
28 Percentage of normal
Standard Deviation 0.23305
|
|
Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)
uninjected eye
|
10.5 Percentage of normal
Standard Deviation 0.28714
|
25 Percentage of normal
Standard Deviation 0.24804
|
Adverse Events
Short-term Affects
Long-term Affects
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Bin Li
Department of Ophthalmology, Tongji Hospital Tongji Medical College, Huazhong University of Science and Technology
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place