Age-Related Macular DegenerationPhase 1/2SIRGANCT00766649
Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral GA Associated With AMD
A Phase 1/2 study testing Sirolimus in people with Age-Related Macular Degeneration, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
This study will determine if a drug called sirolimus is safe to give to people with geographic atrophy GA and if it can help preserve vision in patients. GA is an advanced form of dry age-related macular degeneration (AMD). AMD affects the macula, the central part of the retina needed for sharp, clear vision. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Age-Related Macular Degeneration
- What is being tested?
- Sirolimus
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2008to Jul 2012
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must be older than 55 years of age.
- Participant must understand and sign the protocol's informed consent document.
- Participant must have at least ½ disc area (approximately 1 mm^2) of GA compatible with AMD present in each eye. GA is defined as one or more well-defined, usually more or less circular patches of partial or complete de-pigmentation of the RPE, typically with exposure of underlying choroidal blood vessels. Even if much of the RPE appears to be preserved and large choroidal vessels are not visible, a round patch of RPE partial de-pigmentation may still be classified as early GA. The GA in each eye must be able to be photographed in their entirety.
- Participant must have at least one large druse (more than or equal to 125 μm) in each eye.
- Participant must have a steady fixation in both eyes in the foveal or parafoveal area and media clear enough for good quality photographs. This will permit randomization.
- Participant must have visual acuity between 20/20 and 20/400 in each eye.
- Female participants must be considered post-menopausal and must not be breast-feeding. Female participants over age 55 who have not had a period for one year will be considered post-menopausal.
Who cannot join
- Participant is in another investigational study and actively receiving study therapy.
- Participant is unable to comply with study procedures or follow-up visits.
- Participant has evidence of ocular disease other than AMD in either eye that may confound the outcome of the study (e.g., diabetic retinopathy with 10 or more hemorrhages or microaneurysms, uveitis, pseudovitelliform macular degeneration moderate/severe myopia).
- Participant has any of the following: a) a history of macular laser, b) a history of photodynamic therapy (PDT), c) received an intravitreal injection of anti-vascular endothelial growth factor (VEGF) agent for wet/exudative AMD at any point, d) received an intravitreal injection of any other agent (not an anti-VEGF agent) within four months prior to study enrollment or e) received topical treatment of any agent for advanced AMD within one month prior to enrollment. Participants currently taking or who have previously taken AREDS vitamin supplementation are not excluded.
- Participant has had a vitrectomy.
- Participant is expected to need ocular surgery during the course of the trial.
Show all 15Show fewer
- Participant has undergone lens removal in the last three months or yttrium aluminum garnet (YAG) laser capsulotomy within the last month.
- Participant is on chemotherapy.
- Participant is on immunosuppressive medication.
- Participant is on ocular or systemic medications known to be toxic to the lens, retina or optic nerve.
- Participant with a history of ocular herpes simplex virus (HSV).
- A condition that, in the opinion of the investigator, would preclude participation in the study (e.g., unstable medical status including blood pressure and glycemic control).
- History of cancer (other than a non-melanoma skin cancer) diagnosed within the past five years.
- Laboratory values outside normal limits and considered clinically significant by the investigator.
- Participant is currently taking one of the following drugs: amprenavir, atazanavir, clarithromycin, darunavir, delavirdine, erythromycin, fluconazole (at doses of 200 mg or greater), fluvoxamine, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, quinupristin, ritonavir, saquinavir, telithromycin, troleandomycin, verapamil or voriconazole.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration
This study will determine if a drug called sirolimus is safe to give to people with geographic atrophy GA and if it can help preserve vision in patients. GA is an advanced form of dry age-related macular degeneration (AMD). AMD affects the macula, the central part of the retina needed for sharp, clear vision. There are two types of AMD, wet and dry. In dry AMD, cells in the macula die.GA may at least partly be caused by inflammation. Sirolimus helps prevent inflammation and therefore may help treat GA.
People with GA in both eyes with visual acuity between 20/20 and 20/400 in each eye may be eligible for this study. Participants undergo the following tests and procedures:
- Sirolimus injections in the study eye at each 3-month clinic visit. The drug is injected under the outer layer of the eye after the patient receives antiseptic and numbing drops. Antibiotic drops are continued for 2 days after the injection.
- Eye examinations before the first injection, 1 month after the first injection, during each clinic visit (11 to 15 visits over 2 to 3 years) and 3 months after the final injection. The examination includes testing visual acuity, measuring eye pressure and checking eye movements. To examine the inside of the eye, the pupil is dilated with eye drops. Regular photographs of the inside of the eye and optical coherence tomography photographs, which allow measurement of the thickness of the retina, may be taken during the eye examination.
- Autofluorescence imaging. The patient sits in a chair with his or her head placed in a chin rest in front of a camera. A light in the camera is used to scan the eye.
- Blood tests. Blood is drawn at the first visit and at up to seven study visits to check blood chemistries, such as liver and kidney function.
- Urine pregnancy test for women who are able to become pregnant.
Read the detailed description
Objective: Age-related macular degeneration (AMD), the leading cause of blindness in people over age 55 in the United States, is a heterogeneous clinical entity in which retinal degeneration occurs predominantly in the macula in the context of aging and leads to impairment of central visual acuity. AMD occurs in two general forms, one of which involves choroidal neovascularization (CNV) with subsequent formation of a disciform scar. This is often referred to as the neovascular or "wet" form. A second form, the subject of this study, is termed "dry" or atrophic macular degeneration and involves a constellation of clinical features that can include drusen, pigment clumping and/or retinal pigment epithelium (RPE) dropout and geographic atrophy (GA). GA can begin as a thinning of the RPE with involvement of the underlying choriocapillaris and lead subsequently to an atrophic change in the macula. Inflammation may play a role in the pathogenesis of GA. Sirolimus inhibits the production, signaling and activity of many inflammatory factors relevant to the development of GA. Therefore, the objective of this study is to investigate the safety and possible efficacy of multiple sirolimus subconjunctival injections in participants with bilateral GA.
Study Population: Ten participants with bilateral GA associated with AMD, with the potential to replace up to five participants if some fail to reach one year of follow-up.
Design: In this controlled, unmasked, Phase I/II study, one eye of eligible participants will be randomized to treatment while the fellow eye will be observed. Participants will receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter. The study will be completed once all participants have received two years of study medication and follow-up.
Outcome Measures: The primary outcome is the rate of change in area of GA, based on masked grading by an external Reading Center, of fundus photography in the study eye and fellow eye at two years compared with baseline. Secondary outcomes will include worsening of best-corrected visual acuity (BCVA) of three or more lines (15 or more letters), changes in area of GA as measured on autofluorescence, changes in drusen volume as measured by optical coherence tomography, changes in photoreceptor outer segment (PROS) thickness as measured by optical coherence tomography, as well as changes in drusen area based on masked digital grading of fundus photographs. Safety outcomes will include the number and severity of systemic and ocular toxicities, adverse events and infections.
- Conditions
- Age-Related Macular Degeneration
- Being tested
- Sirolimus
- Keywords
- Age Related Macular Degeneration (AMD), Geographic Atrophy, Sirolimus, Age-Related Macular Degeneration, AMD
- Medical subject headings
- Macular Degeneration, Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune®, rapamycin
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Change in Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Rate of Change in Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Secondary outcomes (9)
Number of Study Eyes With a 15 Letter Drop From Baseline at 24 Months
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Measured: Baseline and Month 24
Absolute Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
This outcome measure was calculated by subtracting the GA value for the study eye at baseline from the GA value for the study eye at Month 24.
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Absolute Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
This outcome measure was calculated by subtracting the GA value for the fellow eye at baseline from the GA value for the study eye at Month 24.
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Relative Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
This outcome measure was calculated by dividing the absolute change in the total area of GA in the study eye at 24 months by the baseline value.
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina. As the unit of measure for both the absolute change in the total area of GA and the baseline value for the total area of GA is MPS DA, the unit of measure for the relative change in the total area of GA is "ratio."
Measured: Baseline to Month 24
Relative Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
This outcome measure was calculated by dividing the absolute change in the total area of GA in the fellow eye at 24 months by the baseline value.
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina. As the unit of measure for both the absolute change in the total area of GA and the baseline value for the total area of GA is MPS DA, the unit of measure for the relative change in the total area of GA is "ratio."
Measured: Baseline and Month 24
Change in Total Area of Drusen in the Study Eye at 24 Months as Compared to Baseline
The total area occupied by drusen was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Change in Total Area of Drusen in the Fellow Eye at 24 Months as Compared to Baseline
The total area occupied by drusen was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA).
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Measured: Baseline and Month 24
Change in Drusen Volume as Measured by Optical Coherence Tomography (OCT) at 24 Months as Compared to Baseline
Measured: Baseline and Month 24
Change in Photoreceptor Outer Segment (PROS) Thickness as Measured by Optical Coherence Tomography at 24 Months as Compared to Baseline
Measured: Baseline and Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2008 (estimated)
- Study startOct 2008
- Primary completionApr 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Age-Related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Wai T. Wong, MD, PhD, Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00766649
- Sponsor's protocol ID
- 090008
- Other IDs
- 09-EI-0008
- Acronym
- SIRGA
- Status last verified
- Apr 2014
- First submitted
- 3 Oct 2008
- Last updated
- 14 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Klein R, Klein BE, Jensen SC, Meuer SM. The five-year incidence and progression of age-related maculopathy: the Beaver Dam Eye Study. Ophthalmology. 1997 Jan;104(1):7-21. doi: 10.1016/s0161-6420(97)30368-6.
PubMed 9022098Klein R, Klein BE, Linton KL. Prevalence of age-related maculopathy. The Beaver Dam Eye Study. Ophthalmology. 1992 Jun;99(6):933-43. doi: 10.1016/s0161-6420(92)31871-8.
PubMed 1630784Seddon JM, Ajani UA, Sperduto RD, Hiller R, Blair N, Burton TC, Farber MD, Gragoudas ES, Haller J, Miller DT, et al. Dietary carotenoids, vitamins A, C, and E, and advanced age-related macular degeneration. Eye Disease Case-Control Study Group. JAMA. 1994 Nov 9;272(18):1413-20.
PubMed 7933422Wong WT, Dresner S, Forooghian F, Glaser T, Doss L, Zhou M, Cunningham D, Shimel K, Harrington M, Hammel K, Cukras CA, Ferris FL, Chew EY. Treatment of geographic atrophy with subconjunctival sirolimus: results of a phase I/II clinical trial. Invest Ophthalmol Vis Sci. 2013 Apr 26;54(4):2941-50. doi: 10.1167/iovs.13-11650.
PubMed 23548622
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration
- ClinicalTrials.gov ID
- NCT00766649
- Acronym / sponsor ID
- SIRGA / 090008
- Conditions
- Age-Related Macular Degeneration
- Keywords
- Age Related Macular Degeneration (AMD), Geographic Atrophy, Sirolimus, Age-Related Macular Degeneration, AMD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- Oct 2008
- Primary completion
- Apr 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 6 Oct 2008 (estimated)
- Results
- Posted 1 Jul 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will determine if a drug called sirolimus is safe to give to people with geographic atrophy GA and if it can help preserve vision in patients. GA is an advanced form of dry age-related macular degeneration (AMD). AMD affects the macula, the central part of the retina needed for sharp, clear vision. There are two types of AMD, wet and dry. In dry AMD, cells in the macula die.GA may at least partly be caused by inflammation. Sirolimus helps prevent inflammation and therefore may help treat GA.
People with GA in both eyes with visual acuity between 20/20 and 20/400 in each eye may be eligible for this study. Participants undergo the following tests and procedures:
- Sirolimus injections in the study eye at each 3-month clinic visit. The drug is injected under the outer layer of the eye after the patient receives antiseptic and numbing drops. Antibiotic drops are continued for 2 days after the injection.
- Eye examinations before the first injection, 1 month after the first injection, during each clinic visit (11 to 15 visits over 2 to 3 years) and 3 months after the final injection. The examination includes testing visual acuity, measuring eye pressure and checking eye movements. To examine the inside of the eye, the pupil is dilated with eye drops. Regular photographs of the inside of the eye and optical coherence tomography photographs, which allow measurement of the thickness of the retina, may be taken during the eye examination.
- Autofluorescence imaging. The patient sits in a chair with his or her head placed in a chin rest in front of a camera. A light in the camera is used to scan the eye.
- Blood tests. Blood is drawn at the first visit and at up to seven study visits to check blood chemistries, such as liver and kidney function.
- Urine pregnancy test for women who are able to become pregnant.
Detailed description
Objective: Age-related macular degeneration (AMD), the leading cause of blindness in people over age 55 in the United States, is a heterogeneous clinical entity in which retinal degeneration occurs predominantly in the macula in the context of aging and leads to impairment of central visual acuity. AMD occurs in two general forms, one of which involves choroidal neovascularization (CNV) with subsequent formation of a disciform scar. This is often referred to as the neovascular or "wet" form. A second form, the subject of this study, is termed "dry" or atrophic macular degeneration and involves a constellation of clinical features that can include drusen, pigment clumping and/or retinal pigment epithelium (RPE) dropout and geographic atrophy (GA). GA can begin as a thinning of the RPE with involvement of the underlying choriocapillaris and lead subsequently to an atrophic change in the macula. Inflammation may play a role in the pathogenesis of GA. Sirolimus inhibits the production, signaling and activity of many inflammatory factors relevant to the development of GA. Therefore, the objective of this study is to investigate the safety and possible efficacy of multiple sirolimus subconjunctival injections in participants with bilateral GA.
Study Population: Ten participants with bilateral GA associated with AMD, with the potential to replace up to five participants if some fail to reach one year of follow-up.
Design: In this controlled, unmasked, Phase I/II study, one eye of eligible participants will be randomized to treatment while the fellow eye will be observed. Participants will receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter. The study will be completed once all participants have received two years of study medication and follow-up.
Outcome Measures: The primary outcome is the rate of change in area of GA, based on masked grading by an external Reading Center, of fundus photography in the study eye and fellow eye at two years compared with baseline. Secondary outcomes will include worsening of best-corrected visual acuity (BCVA) of three or more lines (15 or more letters), changes in area of GA as measured on autofluorescence, changes in drusen volume as measured by optical coherence tomography, changes in photoreceptor outer segment (PROS) thickness as measured by optical coherence tomography, as well as changes in drusen area based on masked digital grading of fundus photographs. Safety outcomes will include the number and severity of systemic and ocular toxicities, adverse events and infections.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusParticipants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter. |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participant must be older than 55 years of age.
- Participant must understand and sign the protocol's informed consent document.
- Participant must have at least ½ disc area (approximately 1 mm^2) of GA compatible with AMD present in each eye. GA is defined as one or more well-defined, usually more or less circular patches of partial or complete de-pigmentation of the RPE, typically with exposure of underlying choroidal blood vessels. Even if much of the RPE appears to be preserved and large choroidal vessels are not visible, a round patch of RPE partial de-pigmentation may still be classified as early GA. The GA in each eye must be able to be photographed in their entirety.
- Participant must have at least one large druse (more than or equal to 125 μm) in each eye.
- Participant must have a steady fixation in both eyes in the foveal or parafoveal area and media clear enough for good quality photographs. This will permit randomization.
Key exclusion criteria (15 listed)
- Participant is in another investigational study and actively receiving study therapy.
- Participant is unable to comply with study procedures or follow-up visits.
- Participant has evidence of ocular disease other than AMD in either eye that may confound the outcome of the study (e.g., diabetic retinopathy with 10 or more hemorrhages or microaneurysms, uveitis, pseudovitelliform macular degeneration moderate/severe myopia).
- Participant has any of the following: a) a history of macular laser, b) a history of photodynamic therapy (PDT), c) received an intravitreal injection of anti-vascular endothelial growth factor (VEGF) agent for wet/exudative AMD at any point, d) received an intravitreal injection of any other agent (not an anti-VEGF agent) within four months prior to study enrollment or e) received topical treatment of any agent for advanced AMD within one month prior to enrollment. Participants currently taking or who have previously taken AREDS vitamin supplementation are not excluded.
- Participant has had a vitrectomy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: Rapamune®, rapamycinParticipants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter. | Drug | Sirolimus |
Primary outcome
Rate of Change in Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
Rate of Change in Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm^2 on the retina.
9 secondary outcomes
Number of Study Eyes With a 15 Letter Drop From Baseline at 24 Months
Time frame: Baseline and Month 24
Absolute Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Absolute Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Relative Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
Time frame: Baseline to Month 24
Relative Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Change in Total Area of Drusen in the Study Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Change in Total Area of Drusen in the Fellow Eye at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Change in Drusen Volume as Measured by Optical Coherence Tomography (OCT) at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Change in Photoreceptor Outer Segment (PROS) Thickness as Measured by Optical Coherence Tomography at 24 Months as Compared to Baseline
Time frame: Baseline and Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 9022098Klein R, Klein BE, Jensen SC, Meuer SM. The five-year incidence and progression of age-related maculopathy: the Beaver Dam Eye Study. Ophthalmology. 1997 Jan;104(1):7-21. doi: 10.1016/s0161-6420(97)30368-6.
- Background: PubMed 1630784Klein R, Klein BE, Linton KL. Prevalence of age-related maculopathy. The Beaver Dam Eye Study. Ophthalmology. 1992 Jun;99(6):933-43. doi: 10.1016/s0161-6420(92)31871-8.
- Background: PubMed 7933422Seddon JM, Ajani UA, Sperduto RD, Hiller R, Blair N, Burton TC, Farber MD, Gragoudas ES, Haller J, Miller DT, et al. Dietary carotenoids, vitamins A, C, and E, and advanced age-related macular degeneration. Eye Disease Case-Control Study Group. JAMA. 1994 Nov 9;272(18):1413-20.
- Results publication: PubMed 23548622Wong WT, Dresner S, Forooghian F, Glaser T, Doss L, Zhou M, Cunningham D, Shimel K, Harrington M, Hammel K, Cukras CA, Ferris FL, Chew EY. Treatment of geographic atrophy with subconjunctival sirolimus: results of a phase I/II clinical trial. Invest Ophthalmol Vis Sci. 2013 Apr 26;54(4):2941-50. doi: 10.1167/iovs.13-11650.
Related trials for Age-Related Macular Degeneration
RecruitingPhase 3
A Study of 36-Week Refill Exchanges of Port Delivery System (PDS) With Ranibizumab in nAMD
Neovascular Age-related Macular Degeneration
RecruitingObservational study
Pivotal Trial of an Automated AI-based System for Early Diagnosis and Prediction of Late Age-related Macular Degeneration
Age-related Macular Degeneration (AMD)
Active, not recruitingObservational study
Real World Evidence in China: Faricimab Use in Diabetic Macular Edema, Retinal Vein Occlusion, and Neovascular Age-Related Macular Degeneration (The Farseeing Study)
Diabetic Macular Edema, Neovascular Age-related Macular Degeneration
Active, not recruitingObservational study
Retinal Pigment Epithelium Safety Study For Patients In B4711001
Age Related Macular Degeneration
RecruitingObservational study
National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
Inherited Ophthalmic Diseases, Hypopigmentation Disorder
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wai T. Wong, MD, PhD principal investigator
National Eye Institute (NEI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sirolimus in people with Age-Related Macular Degeneration, run by National Eye Institute (NEI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Jul 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Change in Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline (time frame: Baseline and Month 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00766649, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2020.