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| ID | Type | Description | Link |
|---|---|---|---|
| M23-411 | Other Identifier | AbbVie Inc |
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| Name | Class |
|---|---|
| REGENXBIO Inc. | INDUSTRY |
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This interventional study is being conducted with an investigational gene therapy treatment called ABBV-RGX-314 (also known as RGX-314) and is being developed as a potential one-time gene therapy treatment for neovascular (wet) age-related macular degeneration (wet AMD or nAMD). The typical treatment for nAMD is frequent injections of anti-VEGF therapy. Researchers are testing ABBV-RGX-314 to see if it has similar effects as the current approved standard of care, such as Lucentis® or Eylea® injections.
The duration of this study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314 after week 52, up to 80 weeks post-randomization.
The primary outcome measure for this investigational study is to evaluate the mean change in best-corrected visual acuity (BCVA) for ABBV-RGX-314 compared with ranibizumab monthly at the Week 40 visit.
This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 gene therapy in subjects with nAMD.
ABBV-RGX-314 will be injected into the SCS part of the eye. Approximately 140 participants who meet the inclusion/exclusion criteria will be enrolled into one of 7 cohorts. Participants will be randomized in Cohorts 1 and 2 to receive ABBV-RGX-314 or the intravitreal ranibizumab control. Participants enrolled in Cohorts 3 through 5 will receive ABBV-RGX-314. Participants enrolled in Cohort 6 will receive ABBV-RGX-314 and will be randomized to one of two different post-procedural steroid regimens. Participants randomized in Cohort 7 will receive ABBV-RGX-314 with a protocol mandated steroid regimen or the intravitreal ranibizumab control. Cohort 1 will evaluate ABBV-RGX-314 Dose 1, Cohorts 2 and 3 will evaluate ABBV-RGX-314 Dose 2, Cohorts 4, 5, and 6 will evaluate ABBV-RGX-314 Dose 3, and Cohort 7 will evaluate ABBV-RGX-314 Dose 4.
The duration of the study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314, up to 80 weeks post-randomization.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ranibizumab control | Active Comparator | Control treatment arm |
|
| ABBV-RGX-314 Treatment Arm (Dose 1) | Experimental | ABBV-RGX-314 Dose 1 |
|
| ABBV-RGX-314 Treatment Arm (Dose 2) | Experimental | ABBV-RGX-314 Dose 2 |
|
| ABBV-RGX-314 Treatment Arm (Dose 3) | Experimental | ABBV-RGX-314 Dose 3 |
|
| ABBV-RGX-314 Treatment Arm (Dose 3) and Local Steroid | Experimental | ABBV-RGX-314 Dose 3 and Local Steroid |
|
| ABBV-RGX-314 Treatment Arm (Dose 3) and Topical Steroid | Experimental | ABBV-RGX-314 Dose 3 and Topical Steroid |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ranibizumab | Biological | Ranibizumab (anti-VEGF agent) |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Mean change from baseline in BCVA to Week 40 based on the Early Treatment Diabetic Retinopathy Study (ETDRS) score | The scale used is the early treatment diabetic retinopathy study (ETDRS) letter score from 0-100 and higher score being better vision. | 40 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs) | Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs) | 52 weeks |
| Vector shedding analysis in serum, urine, and tears |
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Inclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| ABBVIE INC. | AbbVie | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Retinal Research Institute /ID# 255925 | Phoenix | Arizona | 85053 | United States | ||
| California Retina Consultants - Bakersfield /ID# 255910 |
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The vision examiners and central reading center (CRC) graders will be masked, meaning they will be unaware of the participants' treatment assignment. All other individuals affiliated with the study (investigators, all study center personnel apart from the vision examiners, the participants, all staff of Sponsor and its research collaborator, all staff affiliated with the contract research organization, and all CRC staff apart from the CRC graders) will have knowledge of the treatment assignment.
|
| ABBV-RGX-314 Treatment Arm (Dose 4) and Topical Steroid | Experimental | ABBV-RGX-314 Dose 4 and Topical Steroid |
|
| ABBV-RGX-314 Dose 1 |
| Genetic |
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1) |
|
|
| ABBV-RGX-314 Dose 2 | Genetic | AAV8 vector containing a transgene for anti-VEGF fab (Dose 2) |
|
|
| ABBV-RGX-314 Dose 3 | Genetic | AAV8 vector containing a transgene for anti-VEGF fab (Dose 3) |
|
|
| Local Steroid | Drug | Local steroid |
|
| Topical Steroid | Drug | Topical steroid |
|
| ABBV-RGX-314 Dose 4 | Genetic | AAV8 vector containing a transgene for anti-VEGF fab (Dose 4) |
|
|
Evaluate the safety and tolerability of ABBV-RGX-314
| 52 Weeks |
| Proportion of participants who experience ocular inflammation following SCS ABBV-RGX-314 administration | To evaluate the incidences of ocular inflammation following SCS ABBV-RGX-314 administration | 52 weeks |
| Mean change from baseline in choroidal neovascularization (CNV) lesion size and leakage area based on fluorescein angiography (FA) | To evaluate the effect of ABBV-RGX-314 on CNV lesion growth and leakage as measured by FA | Up to Week 80 |
| Mean change from baseline in BCVA based on ETDRS score | Evaluate the effect of ABBV-RGX-314 on BCVA | Up to Week 80 |
| Proportion of participants (1) gaining ≥ 15, ≥ 10, ≥ 5, or ≥ 0 letters; (2) losing ≥ 15, ≥ 10, ≥ 5, or > 0 letters (3) maintaining vision (not losing ≥ 15 letters) compared with baseline as per BCVA | To evaluate the effect of ABBV-RGX-314 on BCVA | Up to Week 52 |
| Mean change from baseline in CRT as measured by SD-OCT | To evaluate the effect of ABBV-RGX-314 on CRT, as measured by SD-OCT | Up to Week 80 |
| Mean supplemental anti-VEGF injection annualized rate in the ABBV-RGX-314 treatment arms | To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment | Up to Week 80 |
| Proportion of participants who have a reduction of ≥ 50% and ≥ 75% in supplemental anti-VEGF- injection annualized rate | Proportion of participants who have a reduction of ≥ 50% and ≥ 75% in supplemental anti-VEGF- injection annualized rate through Week 40 and Week 52 compared with the prior 52 weeks preceding the first intravitreal ranibizumab injection received as part of the Screening Period (ABBV-RGX-314 administered participants) | 52 Weeks |
| Proportion of participants with 0, ≤ 1, and ≤ 2 supplemental injections | To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment | Up to Week 80 |
| Mean percent reduction in supplemental anti-VEGF injection annualized rate | To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment | Up to Week 80 |
| Time from ABBV-RGX-314 administration to first supplemental anti-VEGF injection | To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment | 80 Weeks |
| Aqueous humor ABBV-RGX- 314 TP concentration over time | To evaluate the concentration of ABBV-RGX-314 TP in aqueous humor and serum | 52 Weeks |
| Mean change from baseline in serum ABBV-RGX-314 TP concentration over time | To evaluate the concentration of ABBV-RGX-314 TP in aqueous humor and serum | 80 Weeks |
| Bakersfield |
| California |
| 93309-1677 |
| United States |
| Retina Vitreous Assoc Med Grp /ID# 255921 | Beverly Hills | California | 90211 | United States |
| Retinal Diagnostic Center /ID# 272275 | Campbell | California | 95008 | United States |
| Northern California Retina Vitreous Associates Medical Group, Inc /ID# 255920 | Mountain View | California | 94040-4101 | United States |
| Retina Consultants of San Diego /ID# 255911 | Poway | California | 92064-2530 | United States |
| California Retina Consultants - Santa Barbara /ID# 255923 | Santa Barbara | California | 93103 | United States |
| Southeast Retina Center /ID# 255912 | Augusta | Georgia | 30909 | United States |
| Springfield Clinic - First /ID# 272274 | Springfield | Illinois | 62702 | United States |
| Johns Hopkins Hospital /ID# 255919 | Baltimore | Maryland | 21287 | United States |
| Ophthalmic Consultants of Boston /ID# 255917 | Boston | Massachusetts | 02114 | United States |
| Sierra Eye Associates /ID# 255908 | Reno | Nevada | 89502 | United States |
| Eye Associates of New Mexico /ID# 255915 | Albuquerque | New Mexico | 87109 | United States |
| Duke University Medical Center /ID# 267646 | Durham | North Carolina | 27710 | United States |
| Mid Atlantic Retina /ID# 255906 | Philadelphia | Pennsylvania | 19107 | United States |
| Charles Retina Institute /ID# 255922 | Germantown | Tennessee | 38138 | United States |
| Tennessee Retina - Nashville /ID# 255918 | Nashville | Tennessee | 37203-1513 | United States |
| Retina Consultants - The Woodlands /ID# 255924 | The Woodlands | Texas | 77384 | United States |
| ID | Term |
|---|---|
| D000069579 | Ranibizumab |
| D000077725 | Piperacillin, Tazobactam Drug Combination |
| D013256 | Steroids |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D000078142 | Tazobactam |
| D010397 | Penicillanic Acid |
| D010406 | Penicillins |
| D047090 | beta-Lactams |
| D007769 | Lactams |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D010878 | Piperacillin |
| D000667 | Ampicillin |
| D010400 | Penicillin G |
| D013457 | Sulfur Compounds |
| D013450 | Sulfones |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D004338 | Drug Combinations |
| D004364 | Pharmaceutical Preparations |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
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