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Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

This study is currently recruiting participants.

Verified April 2013 by Children's Hospital of Philadelphia

Sponsor:

Collaborator:

University of Iowa

Information provided by (Responsible Party):

Children's Hospital of Philadelphia

ClinicalTrials.gov Identifier:

NCT00999609

First received: October 21, 2009

Last updated: April 5, 2013

Last verified: April 2013

History of Changes

No Study Results Posted on ClinicalTrials.gov for this Study

About Study Results Reporting on ClinicalTrials.gov

Study Status:	This study is currently recruiting participants.
Estimated Study Completion Date:	April 2029
Estimated Primary Completion Date:	April 2015 (Final data collection date for primary outcome measure)

Publications:

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 1;121(6):2160-8. 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. 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.