Age-Related Macular Degeneration
Conditions
Brief summary
Phase 2 clinical trial, single site, randomized, subject-masked study to determine safety and efficacy of intravitreal injections of Sirolimus in subjects with wet Age-Related Macular Degeneration (wet AMD) with persistent intraretinal or subretinal edema due to neovascular AMD despite previous AntiVEGF treatment.
Detailed description
Forty subjects will be randomized to receive intravitreal Sirolimus or standard of care treatment in a 1:1 ratio. Over the course of the six month trial plus a 6 month optional extension, subjects will be evaluated monthly. Treatment with Sirolimus will be given at baseline, month 2 and month 4 with sham injections given at months 1, 3 and 5. For subjects in the extension Sirolimus will also be given at months 6, 8 and 10 with sham at month 7, 9, and 11. Subjects in the standard of care group will receive anti-VEGF injections or sham monthly. Retreatment criteria will be based upon continued subretinal edema, intraretinal edema or active chorioretinal neovascularization (CNV)
Interventions
DRUGSirolimus
intravitreal injection
DRUGStandard of Care intravitreal injections of anti-VEGF
intravitreal injections of anti-VEGF
Sponsors
Maturi, Raj K., M.D., P.C.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* best corrected visual acuity of 5-65, inclusive, in study eye * presence of choroid neovascularization secondary to AMD * persistent edema despite at least 3 previous intravitreal anti-VEGF injections in the past 5 months
Exclusion criteria
* greater than 100 micron decrease in central subfield thickness on Optical Coherence Topography (OCT) since last standard of care visit * history of major ophthalmic surgery in the study eye in the past 3 months * history of significant ocular disease or condition other than exudative AMD that may confound results
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Edema From Baseline to Month 6 Central Subfield Thickness ) on Heidelberg Optical Coherence Topography | Baseline to 6 months | Change in edema from baseline to month 6 as measured by mean difference in microns of central subfield thickness on Heidelberg optical coherence topography in each treatment group |
| Visual Acuity | Baseline to 6 months | number of subjects with gain of 0-4 letters of visual acuity |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Visual Acuity (Best Corrected Visual Acuity) | baseline to 6 months | number of subjects with gain of 5 or more letters of visual acuity |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Sirolimus Intravitreal Sirolimus
Sirolimus: intravitreal injection | 20 |
| Standard of Care Intravitreal antiVEGF antiVEGF intravitreal injections
Standard of Care intravitreal injections of antiVEGF: intravitreal injections of antiVEGF | 20 |
| Total | 40 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| First 6 Months | Death | 2 | 0 |
Baseline characteristics
| Characteristic | Sirolimus | Standard of Care Intravitreal antiVEGF | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 20 Participants | 18 Participants | 38 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 2 Participants | 2 Participants |
| Region of Enrollment United States | 20 participants | 20 participants | 40 participants |
| Sex: Female, Male Female | 12 Participants | 16 Participants | 28 Participants |
| Sex: Female, Male Male | 8 Participants | 4 Participants | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 2 / 20 | 0 / 20 |
| other Total, other adverse events | 14 / 20 | 15 / 20 |
| serious Total, serious adverse events | 1 / 20 | 1 / 20 |
Outcome results
Primary
Change in Edema From Baseline to Month 6 Central Subfield Thickness ) on Heidelberg Optical Coherence Topography
Change in edema from baseline to month 6 as measured by mean difference in microns of central subfield thickness on Heidelberg optical coherence topography in each treatment group
Time frame: Baseline to 6 months
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Sirolimus | Change in Edema From Baseline to Month 6 Central Subfield Thickness ) on Heidelberg Optical Coherence Topography | -30.0 microns |
| Standard of Care Intravitreal Anti-VEGF | Change in Edema From Baseline to Month 6 Central Subfield Thickness ) on Heidelberg Optical Coherence Topography | 7.5 microns |
Primary
Visual Acuity
number of subjects with gain of 0-4 letters of visual acuity
Time frame: Baseline to 6 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Sirolimus | Visual Acuity | 7 Participants |
| Standard of Care Intravitreal Anti-VEGF | Visual Acuity | 11 Participants |
Secondary
Visual Acuity (Best Corrected Visual Acuity)
number of subjects with gain of 5 or more letters of visual acuity
Time frame: baseline to 6 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Sirolimus | Visual Acuity (Best Corrected Visual Acuity) | 9 participants |
| Standard of Care Intravitreal Anti-VEGF | Visual Acuity (Best Corrected Visual Acuity) | 9 participants |