Trial reportInvestigative ophthalmology & visual science2013
Treatment of geographic atrophy with subconjunctival sirolimus: results of a phase I/II clinical trial.
Trial report in Investigative ophthalmology & visual science, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00766649 (Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration), which is not on this map. Cited by 40 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration
Who cites it
40 citing papers in PubMed, 72 citations in OpenAlex.
- Intravitreal sirolimus for the treatment of geographic atrophy: results of a phase I/II clinical trial.Investigative ophthalmology & visual science · 2014Trial
- An siRNA targetingMolecular therapy. Nucleic acids · 2026Article
- Hallmarks of aging in age-related macular degeneration and age-related neurological disorders: novel insights into common mechanisms and clinical relevance.Eye (London, England) · 2025Review
- Pten Loss Triggers Progressive Photoreceptor Degeneration in an mTORC1-Independent Manner.Investigative ophthalmology & visual science · 2025Article
- Geographic Atrophy in Age-Related Macular Degeneration: A Tale of Two Stages.Ophthalmology science · 2023Article
- Novel Approaches in the Drug Development and Delivery Systems for Age-Related Macular Degeneration.Life (Basel, Switzerland) · 2023Review
- Vitreous humor proteome: unraveling the molecular mechanisms underlying proliferative and neovascular vitreoretinal diseases.Cellular and molecular life sciences : CMLS · 2022Review
- Neuroprotection for Age-Related Macular Degeneration.Ophthalmology science · 2022Review
- mTOR Signalling Pathway: A Potential Therapeutic Target for Ocular Neurodegenerative Diseases.Antioxidants (Basel, Switzerland) · 2022Review
- Treatments for dry age-related macular degeneration: therapeutic avenues, clinical trials and future directions.The British journal of ophthalmology · 2022Review
- HK2 Mediated Glycolytic Metabolism in Mouse Photoreceptors Is Not Required to Cause Late Stage Age-Related Macular Degeneration-Like Pathologies.Biomolecules · 2021Article
- Innate Immunity in Age-Related Macular Degeneration.Advances in experimental medicine and biology · 2021Article
- Clinical Perspectives and Trends: Microperimetry as a Trial Endpoint in Retinal Disease.Ophthalmologica. Journal international d'ophtalmologie. International journal of ophthalmology. Zeitschrift fur Augenheilkunde · 2021Review
- Complement activation, lipid metabolism, and mitochondrial injury: Converging pathways in age-related macular degeneration.Redox biology · 2020Review
- MTOR-initiated metabolic switch and degeneration in the retinal pigment epithelium.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2020Article
- Improving retinal mitochondrial function as a treatment for age-related macular degeneration.Redox biology · 2020Article
- Altered photoreceptor metabolism in mouse causes late stage age-related macular degeneration-like pathologies.Proceedings of the National Academy of Sciences of the United States of America · 2020Article
- Directional kinetics analysis of the progression of geographic atrophy.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2019Article
- Performance of a Defect-Mapping Microperimetry Approach for Characterizing Progressive Changes in Deep Scotomas.Translational vision science & technology · 2019Article
- mTORC Inhibitors as Broad-Spectrum Therapeutics for Age-Related Diseases.International journal of molecular sciences · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 3 institutions in 1 country.
Funding
Abstract
purposeTo investigate the safety and effects of subconjunctival sirolimus, an mTOR inhibitor and immunosuppressive agent, for the treatment of geographic atrophy (GA).
methodsThe study was a single-center, open-label phase II trial, enrolling 11 participants with bilateral GA; eight participants completed 24 months of follow-up. Sirolimus (440 μg) was administered every 3 months as a subconjunctival injection in only one randomly assigned eye in each participant for 24 months. Fellow eyes served as untreated controls. The primary efficacy outcome measure was the change in the total GA area at 24 months. Secondary outcomes included changes in visual acuity, macular sensitivity, central retinal thickness, and total drusen area.
resultsThe study drug was well tolerated with few symptoms and related adverse events. Study treatment in study eyes was not associated with structural or functional benefits relative to the control fellow eyes. At month 24, mean GA area increased by 54.5% and 39.7% in study and fellow eyes, respectively (P = 0.41), whereas mean visual acuity decreased by 21.0 letters and 3.0 letters in study and fellow eyes, respectively (P = 0.03). Substantial differences in mean changes in drusen area, central retinal thickness, and macular sensitivity were not detected for all analysis time points up to 24 months.
conclusionsRepeated subconjunctival sirolimus was well-tolerated in patients with GA, although no positive anatomic or functional effects were identified. Subconjunctival sirolimus may not be beneficial in the prevention of GA progression, and may potentially be associated with effects detrimental to visual acuity. (ClinicalTrials.gov number, NCT00766649.).
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.