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. Author manuscript; available in PMC: 2011 Jan 1.
Published in final edited form as: Pharmacol Res. 2009 Oct 17;61(1):14–26. doi: 10.1016/j.phrs.2009.10.002

Table 1.

Clinical trials of gene therapy using viral vectors for neurodegenerative diseases in the CNS

Condition IdentifierA,B Viral Vector Gene(s) StatusC Phase Primary Sponsor Location
Alzheimer's disease NCT00017940A,
US-322B 		retrovirus (ex
vivo) 		Nerve Growth Factor
(NGF) / CERE-110 		Completed (4) I The Shiley Family
Trust 		USA
NCT00087789A
US-623B 		AAV Serotype 2 Nerve Growth Factor
(NGF) / CERE-110 		In Progress, not recruiting - March 2009D I Ceregene USA
NCT00876863A
US-930B 		AAV Serotype 2 Nerve Growth Factor
(NGF) / CERE-110 		Actively recruiting - September 2009D II Ceregene USA
Epilepsy US-669B AAV 1/2 Neuropeptide Y N/A I N/A USA
Parkinson’s Disease NCT00252850A
US-689B 		AAV Serotype 2 Neurturin (NTN) /
CERE-120 		Completed March 2007 (133) I Ceregene USA
NCT00400634A
US-788B 		AAV Serotype 2 Neurturin (NTN) /
CERE-120 		Completed November 2008D II Ceregene USA
NCT00985517A
US-981B 		AAV Serotype 2 Neurturin (NTN) /
CERE-120 		In Progress, Actively recruiting - September 2009D I/II Ceregene USA
ES-008B AAV Serotype 2 Neurturin (NTN) /
CERE-120 		Open , Date approved/initiated September 2008 II N/A Spain
SE-006B AAV Serotype 2 Neurturin (NTN) /
CERE-120 		Open , Date approved/initiated September 2008 II N/A Sweden
NCT00229736A
US-593B 		AAV Serotype 2 Aromatic L-Amino Acid
Decarboxylase		 In progress, Not recruiting - July, 2009 I Genzyme USA
NCT00195143A,
US-469B 		AAV Serotype 2 Glutamic Acid
Decarboxylase 65–67		 Completed March 2008 (179, 180) I Neurologix, Inc. USA
NCT00643890A
US-877B 		AAV Serotype 2 Glutamic Acid
Decarboxylase 65–67		 In Progress, Actively recruiting -July 2009D II Neurologix, Inc. USA
FR-041B Lentivirus Tyrosinase, GTP
cyclohydrolase 1, Dopa-
Decarboxylase		 In Progress, not recruiting - Septermber 2009D I/II Oxford Biomedica France
Batten Disease,
Leukodystrophy, Late
Infantile Neuronal
Lipofuscinosis		 NCT00151216A
US-619B 		AAV Serotype 2 Tripeptidyl Peptidase
(CLN2)		 In Progress, Actively recruiting - May 2009* I Weill Medical College
of Cornell University		 USA
US-381B AAV Serotype 2 aspartocyclase (ASPA) Complete - Septermber 2009, Not yet reported Robert Wood Johnson
Medical School		 USA
Leber Congenital
Amaurosis, Retinal
degeneration		 NCT00481546A
US-740B 		AAV Serotype 2 RPE65 In Progress, Actively recruiting - May 2009* I University of
Pennsylvania		 USA
NCT00516477A
US-677B 		AAV Serotype 2 RPE65 In Progress, Actively recruiting -September 2009* I Children's Hospital of
Philadelphia		 USA
NCT00643747A
UK-141B 		AAV Serotype 2 RPE65 In Progress, Actively recruiting -February 2009* I/II University College,
London		 UK
NCT00749957A
US-918B 		AAV Serotype 2 RPE65 In Progress, Actively recruiting - May 2009* I/II Applied Genetic
Technologies Corp		 USA
NCT00821340A AAV Serotype 2 RPE65 Not yet recruiting - January 2009* I Hadassah Medical
Organization		 Israel
A

Identifier for clinicaltrials.gov

B

Identifier for www.wiley.co.uk/genmed/clinical/, last 3 numbers are OBA protocol number for Genetic Modification Clinical Research Information system (GeMCRIS) www.gemcris.od.nih.gov

C

Date of status based on most recent update of three databases (Clinicaltrials.gov, GeMCRIS, Journal of Gene Medicine Clinical Trial Site)

D

See sponsor website for press releases

*

See trial database sites for additional information and references

Studies using NTFs shown in bold

N/A = not available