Trial reportTranslational vision science & technology2025
Addressing Multiplicity in Retinal Sensitivity Analysis: An Alternative Approach to Assessing Gene Therapy Efficacy in Inherited Retinal Diseases.
Trial report in Translational vision science & technology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Clinical and Molecular Features ofOphthalmology science · 2026Article
- Clinical endpoints special session, ISCEV 2025, Utrecht.Documenta ophthalmologica. Advances in ophthalmology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: The purpose of this study was to propose an alternative statistical approach that addresses the issue of multiplicity in microperimetry data analysis, offering a more balanced and sensitive measure of the efficacy of gene therapy in inherited retinal diseases (IRDs). Methods: We analyzed microperimetry data from a phase II trial of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP). The Macular Integrity Assessment (MAIA; CenterVue, Padova, Italy) device was used to evaluate test-retest repeatability. A binomial model was used to calculate the probability of ≥7 decibel (dB) improvements due to chance alone across a 68-locus grid. We proposed an alternative approach to detect changes using a threshold of ≥7 loci with ≥7 dB mean improvement. Results: Test-retest repeatability analysis showed a probability of < 5% for observing pointwise improvements ≥7 dB between 2 baseline visits. Applying the binomial distribution model, we found that the probability of observing improvements ≥7 dB in at least 7 unspecified loci purely by chance was 5.3%. Conclusions: The proposed approach provides a balanced way to address multiplicity while maintaining reasonable statistical significance. Using ≥7 unspecified loci as the criterion for assessing sensitivity changes, offers a comprehensive assessment that can detect genuine treatment effects without being overly conservative. Translational Relevance: This alternative statistical method has the potential to improve the evaluation of retinal sensitivity changes in gene therapy trials for IRDs, providing a more accurate measure of therapeutic efficacy and enhancing clinical decision making.
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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.