AAVCAGsCD59 for the Treatment of Wet AMD
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|ClinicalTrials.gov Identifier: NCT03585556|
Recruitment Status : Active, not recruiting
First Posted : July 13, 2018
Last Update Posted : September 7, 2020
|Condition or disease||Intervention/treatment||Phase|
|Wet Age-related Macular Degeneration||Drug: Intravitreal anti-VEGF Biological: Intravitreal AAVCAGsCD59 Drug: Oral prednisolone||Phase 1|
This is a Phase 1, multi-center, open label study to assess the efficacy and safety of two doses of the adeno-associated viral vector serotype 2 (AAVCAGsCD59) expressing sCD59 administered via intravitreal injection seven days after a single intravitreal injection of anti-VEGF. All patients considered for enrollment in this study must have treatment naive wet AMD, adequate pupillary dilation to permit a thorough ocular exam, and best corrected distance visual acuity in the study eye of 20/25 to 20/400 using the Snellen eye chart.
Written informed consent will be obtained from each study patient prior to his/her participation in any study related procedures. Screening will determine patient eligibility for the study according to written inclusion and exclusion criteria, which include both general medical and AMD-specific criteria. Patients will be enrolled into the study upon verification that they fulfill all eligibility criteria and after completion of all screening assessments.
This study consists of a screening and injection of anti-VEGF (Day 0), injection of AAVCAGsCD59 (Day 7), and a monthly follow-up period (Month 1 through Month 12) where enrolled patients are treated as needed with intravitreal anti-VEGF based on an increase in central subfoveal thickness of >50 micrometers on OCT from Day 0, new subretinal hemorrhage, a decrease in > or equal to 10 ETDRS letters from the previous exam, or as needed based on the treating clinician. The purpose of the study is to evaluate the number of anti-VEGF injections that are required after a single intravitreal injection of AAVCAGsCD59 at a dose of 3.56x10e11vg for subjects 1-22 and 1.071x10e12vg for subjects 23-25 is administered on Day 7. Anti-VEGF will be injected at Day 0 to treat the CNV per standard of care and enable the AAVCAGsCD59 adequate time (up to two weeks) to enter the ganglion cells in the retina and start producing the transgene product, sCD59. Up to twenty-five (25) patients will be enrolled at to 2 clinical sites in this study. A long-term follow-up safety exam will occur at Month 24.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||All patients with treatment naive, new onset wet AMD will be treated with an intravitreal anti-VEGF at Day 0 followed by intravitreal AAVCAGsCD59 at Day 7. Patients will be followed for 12 months and treated with additional anti-VEGF monthly as needed if recurrent disease is identified.|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Proof of Concept Study Evaluating Intravitreal AAVCAGsCD59 for the Treatment of Wet Age-Related Macular Degeneration (AMD)|
|Actual Study Start Date :||September 13, 2018|
|Actual Primary Completion Date :||January 29, 2020|
|Estimated Study Completion Date :||January 29, 2022|
Experimental: AAVCAGsCD59 Treated Arm
An anti-VEGF injection will be given at Day 0 followed by an intravitreal injection of AAVCAGsCD59 at Day 7. All eyes will then be treated with intravitreal anti-VEGF monthly as needed based on disease activity.
Drug: Intravitreal anti-VEGF
Intravitreal injection of anti-VEGF at Day 0 then monthly as needed
Biological: Intravitreal AAVCAGsCD59
An intravitreal injection of AAVCAGsCD59 will occur at Day 7 once
Other Name: HMR59
Drug: Oral prednisolone
A 7 day tapering dose of oral prednisone will be taken at Day 30 by all patients
Other Name: oral corticosteroid
- Number of intravitreal anti-VEGF injections [ Time Frame: 12 months ]Measure the number of intravitreal anti VEGF injections from Month 1 through Month 12 following an intravitreal injection of AAVCAGsCD59 at Day 7
- Number of patients with change in vision of ≥ 15 letters [ Time Frame: 12 months ]Evaluate the change in vision in eyes receiving intravitreal AAVCAGsCD59 in conjunction with anti VEGF for new onset wet AMD
- Number of patients with inflammation, endophthalmitis, IOP>30, retinal detachment, cataract, and systemic adverse events [ Time Frame: 24 months ]Evaluate SAEs associated with the use of intravitreal AAVCAGsCD59
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03585556
|United States, Massachusetts|
|Ophthalmic Consultants of Boston|
|Boston, Massachusetts, United States, 02114|
|Worcester, Massachusetts, United States, 01605|