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AAV2-sFLT01

Phase 1

Macular Degeneration | Monoclonal antibody | Ophthalmology |Sanofi|Last Updated: Aug 22, 2018

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment19

FDA Designations

No designations recorded

Clinical trial landscape

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 used for?

AAV2-sFLT01 is an investigational gene therapy being studied for the treatment of neovascular age-related macular degeneration (AMD), a condition that causes vision loss due to abnormal blood vessel growth in the retina. It is administered to patients aged 50 years and older and is currently in clinical development.

What does AAV2-sFLT01 target?

AAV2-sFLT01 is designed to deliver a gene that encodes a soluble form of the vascular endothelial growth factor (VEGF) receptor, known as sFLT01. This protein is intended to bind to VEGF and inhibit its activity, thereby reducing abnormal blood vessel growth in the eye associated with neovascular age-related macular degeneration.

Who is developing AAV2-sFLT01?

AAV2-sFLT01 is being developed by Sanofi, a multinational pharmaceutical company listed on the stock exchange under the ticker symbol SNY. The company is conducting clinical research to evaluate the safety and tolerability of this gene therapy for patients with neovascular age-related macular degeneration.

What phase is AAV2-sFLT01 in?

AAV2-sFLT01 is in Phase 1 clinical development. It is an investigational therapy that has not yet been approved by regulatory authorities. The Phase 1 trial has been completed, and the therapy remains under investigation for its safety and tolerability in patients with neovascular age-related macular degeneration.

What clinical trials is AAV2-sFLT01 in?

AAV2-sFLT01 has been studied in one completed Phase 1 clinical trial with the identifier NCT01024998. This trial, titled 'Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD),' enrolled 19 participants in the United States and was a controlled study.

Is AAV2-sFLT01 the same as sFLT01 gene therapy?

AAV2-sFLT01 is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver the sFLT01 gene, which encodes a soluble VEGF receptor. It is specifically designed for the treatment of neovascular age-related macular degeneration and is being developed by Sanofi under this name.