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Gene Therapy Trial Report

Summary

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


NCTID NCT01024998 (View at clinicaltrials.gov)
Description
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 (ACTUAL)
Results Posted Not Available

Therapy Information


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
Delivery System Viral transduction
Vector Type AAV2
Dose 1 2E8 vg/eye
Dose 2 2E9 vg/eye
Dose 3 6E9 vg/eye
Dose 4 2E10 vg/eye

Study Record Dates


Current Stage Phase1
Submit Date 2009-12-02
Completion Date 2018-07
Last Update 2018-08-22

Participation Criteria


Eligible Age >=50 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Choroidal neovascular membrane (CNV) secondary to AMD, as confirmed by the patient's medical history and a documented diagnosis of CNV. * Distance BCVA of 20/100 or worse in the study eye. * The fellow eye must have distance BCVA of 20/400 or better. * The study eye, i.e., the eye that receives investigational product, has the worst CVA (As compared to the fellow eye). * Subfoveal disciform scarring in the study eye for the first part of the study (the dose-escalation part). Patients may or may not have macular scarring in the study eye for the second part of the study (MTD phase). In addition, patients enrolled in the second part of the study must have demonstrated responsiveness to an anti-VEGF therapy within 12 months prior to screening and after the patient's most recent treatment of anti-VEGF therapy. * Noted presence of intra- or sub-retinal fluid. * Adequate dilation of pupils to permit thorough ocular examination and testing. * Must be willing to have samples of anterior chamber fluid collected from the study eye. Exclusion Criteria: * CNV in the study eye due to any reason other than AMD. * History of conditions in the study eye during Screening which might alter visual acuity or interfere with study testing. * Active uncontrolled glaucoma. * Had any intraocular surgeries in the study eye within 3 months of enrollment or are known or likely candidates for intraocular surgery (including cataract surgery) in the study eye within 1 year of treatment. * Acute or chronic infection in the study eye. * History of inflammation in the study eye or ongoing inflammation in either eye. * Any contraindication to intravitreal injection. * Received Photo Dynamic Therapy in the study eye within 60 days, or laser photocoagulation within 14 days prior to Screening. * Currently using or have used ranibizumab (Lucentis®), bevacizumab (Avastin™), or pegaptanib sodium (Macugen®) within 1 month prior to Screening. * Currently using or have used Aflibercept (Eylea®) within 4 months prior to Screening. * Currently using any periocular (study eye), intravitreal (study eye), or systemic (oral or intravenous) steroids within 3 months prior to Screening. * Any active herpetic infection, in particular active lesions in the eye or on the face. * Any significant poorly controlled illness that would preclude study compliance and follow-up. * Current or prior use of any medication known to be toxic to the retina or optic nerve. * Previous treatment with any ocular or systemic gene transfer product. * Received any investigational product within 120 days prior to Screening.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


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

Resources/Links