Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease

NCTID	NCT00985517 (View at clinicaltrials.gov)
Description	The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease. (Show More)
Development Status	Inactive
Indication	Idiopathic Parkinson's Disease
Disease Ontology Term	DOID:14330
Compound Name	CERE-120
Compound Description	AAV2.CAG.pre-pro-NGF-NTN cDNA. hGH polyA
Sponsor	Sangamo Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	57
Results Posted	View Results

Target Gene/Variant	NRTN
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraparenchymal
Drug Product Type	Viral vector
Target Tissue/Cell	Putamen, substantia nigra
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	Phase 1: 1.3E11 vg (n=6)
Dose 2	Phase 1: 5.4E11 vg (n=6)
Dose 3	Phase 2: 5.4E11 vg (n=38)
Dose 4	Phase 2: 1.0E12 vg (putamen) + 2.0E11 vg (substantia nigra)
Dose 5

No.of Trial Sites	11
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Therapy was originally developed by Ceregene, Inc. Company was acquired by Sangamo Therapeutics in 2013 after CERE-120 failed its Phase 2b trial for Parkinson's disease