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

Completed · Phase 1

Conditions studied: Macular Degeneration, Age-Related Maculopathies, Age-Related Maculopathy, Maculopathies, Age-Related, Maculopathy, Age-Related, Retinal Degeneration, Retinal Neovascularization, Gene Therapy, Therapy, Gene, Eye Diseases

In brief

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).

Key facts

Study ID

NCT01024998

Run by

Genzyme, a Sanofi Company

People needed

19

Starts

2010-01-11

Expected to finish

2018-07-01

Last updated by the study team

2018-08-22

Who can join

Age: 50 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• investigational site number 03WilmerEy — Baltimore, Maryland, United States
• investigational site number 02Ophthalm — Boston, Massachusetts, United States
• investigational site number 01UMassMem — Worcester, Massachusetts, United States
• investigational site number 05RetinaCo — Slingerlands, New York, United States

Full record on ClinicalTrials.gov

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