Trial reportInvestigative ophthalmology & visual science2014
Intravitreal sirolimus for the treatment of geographic atrophy: results of a phase I/II clinical trial.
Trial report in Investigative ophthalmology & visual science, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01445548 (Pilot Study of the Evaluation of Intravitreal Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration), which is not on this map. Cited by 34 papers, 1 of them a synthesis that pooled it.
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 Intravitreal Sirolimus in the Treatment of Bilateral Geographic Atrophy Associated With Age-Related Macular Degeneration
Who cites it
34 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Fundus autofluorescence imaging: systematic review of test accuracy for the diagnosis and monitoring of retinal conditions.Eye (London, England) · 2017Pooled it
- Evaluation of Geographic Atrophy from Color Photographs and Fundus Autofluorescence Images: Age-Related Eye Disease Study 2 Report Number 11.Ophthalmology · 2016Trial
- An siRNA targetingMolecular therapy. Nucleic acids · 2026Article
- Clinical characterization of CCT2 and its role in autophagy regulation during age-related macular degeneration.Scientific reports · 2025Article
- Pten Loss Triggers Progressive Photoreceptor Degeneration in an mTORC1-Independent Manner.Investigative ophthalmology & visual science · 2025Article
- Longitudinal Changes of Visual Sensitivity With Geographic Atrophy Progression Assessed by Defect-Mapping Microperimetry.Investigative ophthalmology & visual science · 2024Article
- Antiaging Strategies and Remedies: A Landscape of Research Progress and Promise.ACS chemical neuroscience · 2024Review
- Visual Sensitivity Loss in Geographic Atrophy: Structure-Function Evaluation Using Defect-Mapping Microperimetry.Investigative ophthalmology & visual science · 2024Article
- Visual evoked potentials among Indian patients with optic nerve disorders.Bioinformation · 2024Article
- Activation of Lysosomal Function Ameliorates Amyloid-β-Induced Tight Junction Disruption in the Retinal Pigment Epithelium.Molecules and cells · 2023Article
- Rapamycin, the only drug that has been consistently demonstrated to increase mammalian longevity. An update.Experimental gerontology · 2023Review
- Autophagy in age-related macular degeneration.Autophagy · 2023Review
- Neuroprotection for Age-Related Macular Degeneration.Ophthalmology science · 2022Review
- DARPP32, a target of hyperactive mTORC1 in the retinal pigment epithelium.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Recent Advances in Age-Related Macular Degeneration Therapies.Molecules (Basel, Switzerland) · 2022Review
- mTOR Signalling Pathway: A Potential Therapeutic Target for Ocular Neurodegenerative Diseases.Antioxidants (Basel, Switzerland) · 2022Review
- The Role of Inflammation in Retinal Neurodegeneration and Degenerative Diseases.International journal of molecular sciences · 2021Review
- 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
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
Abstract
purposeTo investigate the safety and effects of intravitreal sirolimus for the potential treatment of geographic atrophy (GA).
methodsThe study was a single-center, open-label, phase I/II trial enrolling six participants with bilateral GA treated with intravitreal sirolimus in only one randomly assigned eye, with the fellow eye as control. The primary efficacy outcome measure was the change in total GA area from baseline on color fundus photography (CFP); secondary outcomes included changes in GA area on fundus autofluorescence (FAF), visual acuity, central retinal thickness (CRT), and macular sensitivity from baseline.
resultsAlthough no systemic adverse events were attributed to treatment, two of six participants had ocular adverse events that were possibly associated. The treated eye of one participant developed abnormal paralesional changes on FAF that were associated with accelerated retinal thinning. This accelerated retinal thinning was also seen in the treated eye of a second participant. Because of concern that these events were associated with treatment, treatment was suspended. Comparisons of treated and fellow eyes for change in visual acuity, change in GA area, and change in CRT showed no evidence of treatment benefit and generally favored the untreated fellow eye.
conclusionsWhile paralesional FAF changes and rapid retinal thinning observed are potentially part of the natural course of GA, they may possibly be related to treatment. No general evidence of anatomical or functional benefit was detected in treated eyes. Further data on intravitreal sirolimus for GA treatment will be available from a larger phase II trial. (ClinicalTrials.gov number, NCT01445548.).
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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.