ArticleFrontiers in pharmacology2019
Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.
Article in Frontiers in pharmacology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it.
- ISCEV and IPS guideline for the full-field stimulus test (FST).Documenta ophthalmologica. Advances in ophthalmology · 2024Guideline
- Quantification of Ocular Gene Therapy-Related Inflammation Using the VRMACS Grading System.Ophthalmology science · 2026Review
- Full-Field Stimulus Threshold: A Key Functional Outcome Measure in Retinal Diseases and Clinical Trials.Journal of clinical medicine · 2026Review
- Functional Outcomes and Ocular Safety of Subretinal AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: A Systematic Review and Meta-Analysis.Journal of clinical medicine · 2026Review
- The role of full-field stimulus threshold in evaluating Bietti crystalline dystrophy.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2026Observational
- Drusen-specific dark adaptation profiles in intermediate age-related macular degeneration.International journal of retina and vitreous · 2026Article
- Suprachoroidal Delivery of Viral and Nonviral Vectors for Treatment of Retinal and Choroidal Vascular Diseases.American journal of ophthalmology · 2025Review
- An Analysis of Scotopic Microperimetry in Healthy Adults.Translational vision science & technology · 2025Article
- Assessment ofBiomedicines · 2025Article
- Optical Coherence Tomography Split-Spectrum Amplitude-Decorrelation Optoretinography Detects Early Central Cone Photoreceptor Dysfunction in Retinal Dystrophies.Translational vision science & technology · 2024Article
- Choroideremia: The Endpoint Endgame.International journal of molecular sciences · 2023Review
- A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol.BMC ophthalmology · 2023Article
- Assessment of Scotopic Function in Rod-Cone Inherited Retinal Degeneration With the Scotopic Macular Integrity Assessment.Translational vision science & technology · 2023Article
- Victorian evolution of inherited retinal diseases natural history registry (VENTURE study): Rationale, methodology and initial participant characteristics.Clinical & experimental ophthalmology · 2022Article
- The Effect of Cataract on Color Vision Measurement with the Low-Vision Cambridge Colour Test: Providing an Adjustment Factor for Clinical Trials.Ophthalmology science · 2022Article
- Rapid Quantification of the Binocular Visual Field for Clinical Trials: Performance of a Modified Esterman Supra-Threshold Test Implemented with the Open Perimetry Interface.Clinical ophthalmology (Auckland, N.Z.) · 2022Article
- Retinal gene therapy: an eye-opener of the 21st century.Gene therapy · 2021Article
- Cellular senescence in the aging retina and developments of senotherapies for age-related macular degeneration.Journal of neuroinflammation · 2021Review
- Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Review
- Validation of electronic visual acuity (EVA) measurement against standardised ETDRS charts in patients with visual field loss from inherited retinal degenerations.The British journal of ophthalmology · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Multiple gene therapy trials are occurring for a variety of ophthalmic diseases around the world. The safety of gene therapy in the eye has been established, and the next step is to reliably assess efficacy. This is primarily done through the use of imaging techniques and visual function measures. Standardized visual function assessments, however, were originally developed for a clinical setting and may not be suitable for detecting and quantifying therapeutic changes. This scoping review takes a comprehensive look at current practice in terms of the outcome measures defined at trial registration. These were compared to the outcome measures reported in the literature. All published trials reported the pre-registered primary outcome measure. A range of additional secondary outcomes were reported that were not originally planned. Gaps in gene therapy assessment exist and further discussion are required to find a way forward, particularly as more conditions progress to phase 2 and 3 trials. Several factors impacting on trial design and outcome measure choice are discussed.
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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.