Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01024998
Other study ID # sFLT01-AMD-00106
Secondary ID 0810-948MSC12870
Status Completed
Phase Phase 1
First received
Last updated
Start date January 11, 2010
Est. completion date July 2018

Study information

Verified date August 2018
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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).


Description:

A new treatment for neovascular age-related macular degeneration (AMD) is being investigated. Neovascular AMD is sometimes referred to as the "wet" form of AMD. The purpose of this Phase 1 clinical research study is to examine the safety and ability of an experimental study drug to treat a complication of the disease which leads to vision loss. The name of the study drug is "AAV2-sFLT01." This experimental study drug uses a virus to transfer a gene (genetic code) into cells within the eye. The gene codes for a protein that is intended to diminish the growth of abnormal blood vessels under the retina. The duration of the gene's effect is currently unknown, but might last for years.

This clinical research study will look at the safety of a single administration of AAV2-sFLT01 injected directly into the eye. There are 2 parts to this study, but patients will take part in only one of them. In the first part of the study, 4 different doses of the study drug will be studied in 4 separate groups of patients. Patients in the first part of the study will not be randomized. In the second part of the study, the highest dose that was safe and well tolerated will be studied in 10 more patients. Patients in this part of the study may have a ranibizumab (Lucentis®) injection 26 weeks after their AAV2-sFLT01 injection to verify their responsiveness to anti-VEGF therapy, if they have not demonstrated a response to AAV2-sFLT01. The initial two parts of this protocol are expected to be completed in July, 2013.

All patients injected with AAV2-sFLT01 will be asked to participate in an Extended Follow-Up (EFU) program for up to an additional 4 years. Participation is voluntary but strongly encouraged as it allows for the long term collection of safety information as well as information about the potential long term effects of the study drug. Study visits will take place at the site every 6 months.

Up to thirty-four (34) patients at multiple centers will take part in this study in the United States.


Recruitment information / eligibility

Status Completed
Enrollment 19
Est. completion date July 2018
Est. primary completion date July 2014
Accepts healthy volunteers No
Gender All
Age group 50 Years and older
Eligibility 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.

Study Design


Intervention

Biological:
AAV2-sFLT01
2 x 10^8 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 µL.
AAV2-sFLT01
2 x 10^9 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 µL.
AAV2-sFLT01
6 x 10^9 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 µL.
AAV2-sFLT01
2 x 10^10 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 µL.

Locations

Country Name City State
United States investigational site number 03WilmerEy Baltimore Maryland
United States investigational site number 02Ophthalm Boston Massachusetts
United States investigational site number 05RetinaCo Slingerlands New York
United States investigational site number 01UMassMem Worcester Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Maximum tolerated dose of a single uniocular intravitreal injection of AAV2-sFLT01 Time of treatment through Week 52 (referred to as the "core" study)
Primary Number of Treatment Emergent Adverse Events Time of treatment through Week 52 (referred to as the "core" study)
Primary Number of Treatment Emergent Adverse Events Up to 4 years after the "core" study (referred to as the "Extended Follow-up" period)
Secondary Decreased retinal thickness Time of treatment through Week 52 (referred to as the "core" study)
Secondary Decreased retinal thickness Up to 4 years after the "core" study (referred to as the "Extended Follow-up" period)
See also
  Status Clinical Trial Phase
Recruiting NCT06262737 - Single-center Study Measuring OSDI Dry Eye Score in Patients Undergoing an Anti-VEGF Induction Protocol
Completed NCT02540954 - Efficacy and Safety of Two Different Aflibercept Regimens in Subjects With Neovascular Age-related Macular Degeneration (nAMD) Phase 3
Completed NCT00385333 - Metabolic Mapping to Measure Retinal Metabolism Phase 2
Completed NCT02510794 - Study of the Efficacy and Safety of the Ranibizumab Port Delivery System for Sustained Delivery of Ranibizumab in Patients With Subfoveal Neovascular Age-Related Macular Degeneration Phase 2
Terminated NCT02228304 - Study of the Intravitreal Implantation of NT-503-3 Encapsulated Cell Technology (ECT) for the Treatment of Recurrent Choroidal Neovascularization (CNV) Secondary to Age-related Macular Degeneration (AMD) Phase 1/Phase 2
Completed NCT02390245 - Philadelphia Telemedicine Glaucoma Detection and Follow-Up Study N/A
Completed NCT02181504 - A Study of Abicipar Pegol in Japanese Patients With Neovascular Age-related Macular Degeneration Phase 2
Recruiting NCT01521065 - An Open-label Study to Evaluate the Clinical and Economic Benefits of I-Ray in Patients With Choroidal Neovascularization Secondary to Age-related Macular Degeneration Phase 2
Completed NCT01204541 - A Single-Center Pilot Study to Assess Macular Function N/A
Completed NCT00769392 - Pilot Study: A Randomized Trial Of Anesthetic Agents For Intravitreal Injection N/A
Completed NCT00533520 - Evaluation of Dosing Interval of Higher Doses of Ranibizumab Phase 4
Withdrawn NCT00538538 - Subretinal Lucentis for Hemorrhagic Age-related Macular Degeneration (AMD) Phase 1
Completed NCT00536016 - A Phase 1 Safety Study of Single and Repeated Doses of JSM6427 (Intravitreal Injection) to Treat AMD Phase 1
Terminated NCT00403442 - Bevacizumab in Combination With Verteporfin Reduced and Standard Fluence in the Treatment of Hemorrhaged Lesions in Neovascular AMD Phase 1
Recruiting NCT00157976 - Double-Masked Study of Photrex (Rostaporfin) Photodynamic Therapy in the Treatment of Age-Related Macular Degeneration Phase 3
Completed NCT00242580 - A Safety and Efficacy Study Comparing the Combination Treatments of Verteporfin Therapy Plus One of Two Different Doses of Intravitreal Triamcinolone Acetonide and the Verteporfin Therapy Plus Intravitreal Pegaptanib Phase 3
Completed NCT00239928 - Clinical Study Of Pegaptanib Sodium (EYE001) For Wet-Type Age-Related Macular Degeneration Phase 2
Completed NCT00211458 - Treatment of Age-Related Macular Degeneration With Anecortave Acetate Phase 2
Completed NCT00095433 - Extension Study of rhuFab V2 in Subjects With Neovascular Age-Related Macular Degeneration (AMD) Phase 3
Completed NCT00006202 - Lutein for Age-Related Macular Degeneration Phase 2