Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
This study is ongoing, but not recruiting participants.
First Posted: October 22, 2009
Last Update Posted: March 28, 2017
Children's Hospital of Philadelphia
University of Iowa
Information provided by (Responsible Party):
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study is ongoing, but not recruiting participants.|
|Estimated Study Completion Date:||July 2029|
|Primary Completion Date:||July 2015 (Final data collection date for primary outcome measure)|
|Certification or Request for Extension to Delay Results Submission:||February 16, 2016|
Ashtari M, Cyckowski LL, Monroe JF, Marshall KA, Chung DC, Auricchio A, Simonelli F, Leroy BP, Maguire AM, Shindler KS, Bennett J. The human visual cortex responds to gene therapy-mediated recovery of retinal function. J Clin Invest. 2011 Jun;121(6):2160-8. doi: 10.1172/JCI57377. Epub 2011 May 23. Erratum in: J Clin Invest. 2011 Jul 1;121(7):2945.
Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, Ziviello C, Acerra C, Wright JF, McDonnell JW, High KA, Bennett J, Auricchio A. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther. 2010 Mar;18(3):643-50. doi: 10.1038/mt.2009.277. Epub 2009 Dec 1.
Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM. AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.