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. Author manuscript; available in PMC: 2015 Nov 23.
Published in final edited form as: Expert Rev Ophthalmol. 2015 Apr 10;10(3):281–299. doi: 10.1586/17469899.2015.1035711

Table 1. RPE65-LCA Clinical Trials.

Details of RPE65-LCA clinical trials in gene promoter, viral titer, and volume of the viral solution used. *Results not yet published. **All clinical trial information was obtained from the Clinical Trials online registry, https://clinicaltrials.gov [165].

NCT# Sponsor Phase Vector Promoter Viral titer
(by cohort)		 Volume
(by cohort)		 Subjects Ref
**[165]
00643747 UCL I/II rAAV2/2 hRPE65 1.00×1011 vp/ml 1.0 ml N=12 [2]
00481546 U Penn - Scheie I rAAV2/2 CBSB 1. 5.96×1010 vp/ml
2. 11.92×1010 vp/ml
3. 8. 94×1010 vp/ml
4. 17.88×1010 vp/ml
5. 17.88×1010 vp/ml
(7.95×1010 vp/ml)		 0.15 ml
0.30 ml
0.225 ml
2×0.225 ml
2×0.225 ml
(0.20 ml)		 N=3
N=3
N=3
N=2
N=4
(N=1)		 [3,7,34,35]
00516477 U Penn - CHOP I rAAV2/2 CBA 1. 1.50×1010 vp/ml
2. 4.8×1010 vp/ml
3. 1.50×1011 vp/ml		 0.15 ml
0.15 ml
0.30 ml		 N=3
N=6
N=3		 [4,151–155]
00749957 AGTC I/II rAAV2/2 CBA 1. 4.00×1011 vp/ml
2. 1.33×1012 vp/ml		 0.45 ml
0.45 ml		 N=6
N=6		 [156]
00821340 Hadassah I AAV2/2 CBSB 1.19×1011 vp/ml 0.30 ml N=10 [157]
01496040 Nantes I/II rAAV2/4 hRPE65 6.0×1010 vp/ml 0.4–0.8 ml N=9 [158]
00999609 Sparks
Therapeutics		 III AAV2/2 CBA 1.50×1011 vp/mL 0.30 ml? N=24 *

Abbreviations: NCT#, ClinicalTrials.gov identifier number; UCL, University College of London; U Penn-Scheie, University of Pennsylvania – Scheie Scheie Eye Institute; U Penn-CHOP, University of Pennsylvania – The Children’s Hospital of Philadelphia. hRPE65p, human RPE65 promoter; hRPE65, human RPE65; CBSB, hybrid modified short cytomegalovirus (CMV) enhancer and chicken β-actin promoter (CBA); vp, viral particles; ml, milliliter. Ref, references.