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AAV2-sFLT01

Phase 1

Macular Degeneration | Gene therapy | Ophthalmology |Sanofi|Trials Updated: Aug 22, 2018

AAV2-sFLT01 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Dec 2009

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment19

FDA Designations

No designations recorded

AAV2-sFLT01 clinical trials

AAV2-sFLT01 · 1 trial · 10 indications

Phase 1 1
NCT01024998Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)Macular Degeneration
COMPLETED19 Analytics
PHASE1COMPLETED
Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)
Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

Maximum tolerated dose of a single uniocular intravitreal injection of AAV2-sFLT01
Time of treatment through Week 52 (referred to as the "core" study)
Number of Treatment Emergent Adverse Events
Time of treatment through Week 52 (referred to as the "core" study)

Secondary Endpoints

Decreased retinal thickness
Time of treatment through Week 52 (referred to as the "core" study)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
2 x 10^8 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
2 x 10^9 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
6 x 10^9 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
2 x 10^10 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -

Interventions

NameTypeDescription
AAV2-sFLT01BIOLOGICAL2 x 10\^8 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 μL.
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Eligibility Criteria

Age Range50 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

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...

Countries:United States
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Frequently asked questions about AAV2-sFLT01

What is AAV2-sFLT01?

AAV2-sFLT01 is an investigational gene therapy developed by Sanofi for neovascular age-related macular degeneration, a form of macular degeneration. It is designed to deliver a therapeutic gene directly to the eye. It has been studied in a single Phase 1 clinical trial and is not approved for any indication.

What is AAV2-sFLT01 used for in macular degeneration?

AAV2-sFLT01 was studied for the treatment of neovascular age-related macular degeneration, often called wet AMD, a condition in which abnormal blood vessels grow under the retina. The Phase 1 trial enrolled patients with this condition. It remains an investigational therapy and is not approved for clinical use.

Who is developing AAV2-sFLT01?

AAV2-sFLT01 is being developed by Sanofi, which trades on the New York Stock Exchange under the ticker SNY. Sanofi sponsored the Phase 1 study of the therapy in patients with neovascular age-related macular degeneration. No other developer is listed for this asset.

What phase is AAV2-sFLT01 in?

AAV2-sFLT01 is in Phase 1 development. It has completed one Phase 1 clinical trial, and no active trials are currently listed. The therapy remains investigational and has not been approved by the FDA for any indication.

What clinical trials is AAV2-sFLT01 in?

AAV2-sFLT01 has been evaluated in one completed Phase 1 trial, NCT01024998, titled a safety and tolerability study in patients with neovascular age-related macular degeneration. The trial enrolled 19 patients in the United States and included a controlled design. No other trials are listed.