Trial Outcomes & Findings for Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis (NCT NCT00749957)
NCT ID: NCT00749957
Last Updated: 2017-12-28
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
12 participants
Primary outcome timeframe
2 years
Results posted on
2017-12-28
Participant Flow
Twelve subjects with DNA sequence-confirmed mutations in RPE65 were enrolled, 10 at the Casey Eye Institute and 2 at the University of Massachusetts, between June 2009 and September 2012.
Participant milestones
| Measure |
Lower Dose of rAAV2-CB-hRPE65
Subjects at least 6 y/o administered a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
Subjects at least 6 y/o administered a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
|
Overall Study
COMPLETED
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Baseline characteristics by cohort
| Measure |
Lower Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
22 years
n=5 Participants
|
31 years
n=7 Participants
|
31 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Lower Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
|---|---|---|
|
Number of Participants Experiencing Ocular or Non-ocular Adverse Events
|
6 participants
|
6 participants
|
SECONDARY outcome
Timeframe: 2 yearsImprovement in the central 30 degree visual field, measured by static perimetry, at one or more time points after treatment, that was greater than the limit of agreement for baseline values .
Outcome measures
| Measure |
Lower Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
|---|---|---|
|
Participants With Changes in Visual Fields
|
3 participants
|
3 participants
|
SECONDARY outcome
Timeframe: 2 yearsIncrease in BCVA of 7 or more letters at Year 2 visit compared to average baseline value
Outcome measures
| Measure |
Lower Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
n=6 Participants
Subjects at least 6 y/o treated with a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
|---|---|---|
|
Participants With Changes in Best Corrected Visual Acuity
|
2 participants
|
1 participants
|
Adverse Events
Lower Dose of rAAV2-CB-hRPE65
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Higher Dose of rAAV2-CB-hRPE65
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Lower Dose of rAAV2-CB-hRPE65
n=6 participants at risk
Subjects at least 6 y/o administered a lower dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
Higher Dose of rAAV2-CB-hRPE65
n=6 participants at risk
Subjects at least 6 y/o administered a higher dose of the rAAV2-CB-hRPE65 vector by subretinal injection
|
|---|---|---|
|
Eye disorders
subconjunctival hemorrhage
|
50.0%
3/6 • Number of events 3 • 2 years
|
83.3%
5/6 • Number of events 5 • 2 years
|
|
Eye disorders
ocular hyperemia
|
50.0%
3/6 • Number of events 3 • 2 years
|
16.7%
1/6 • Number of events 1 • 2 years
|
|
Eye disorders
eye inflammation
|
0.00%
0/6 • 2 years
|
50.0%
3/6 • 2 years
|
|
Eye disorders
eye irritation
|
16.7%
1/6 • Number of events 1 • 2 years
|
16.7%
1/6 • Number of events 1 • 2 years
|
|
Eye disorders
eye pain
|
16.7%
1/6 • Number of events 2 • 2 years
|
0.00%
0/6 • 2 years
|
|
Eye disorders
eye pruritus
|
0.00%
0/6 • 2 years
|
33.3%
2/6 • Number of events 2 • 2 years
|
Additional Information
Chief Medical Officer
Applied Genetic Technologies Corporation
Phone: 386-462-2204
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place