Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)

NCTID	NCT01024998 (View at clinicaltrials.gov)
Description	This Phase 1 clinical research study will examine the safety and tolerability of an experimental gene transfer agent, AAV2-sFLT01, in patients with Neovascular Age-Related Macular Degeneration (AMD). (Show More)
Development Status	Inactive
Indication	Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term	DOID:10871
Compound Name	RAAV2-sFLT01
Sponsor	Genzyme, a Sanofi Company
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	19
Results Posted	Not Available

Target Gene/Variant	SFLT01
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	2E8 vg/eye
Dose 2	2E9 vg/eye
Dose 3	6E9 vg/eye
Dose 4	2E10 vg/eye
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Sanofi acquired Genzyme in 2011, product was well tolerated but showed limited efficacy and never moved to Phase 2