Design Characteristics of Geographic Atrophy Treatment Trials: Systematic Review of Registered Trials in ClinicalTrials.gov

Cheng, Qianqian Ellie; Gao, Jeffrey; Kim, Benjamin J.; Ying, Gui‐Shuang

doi:10.1016/j.oret.2017.08.018

Cited by 23 publications

(19 citation statements)

References 22 publications

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“…In line with other researchers [3][4][5]17,18 we also recommend the use of clinical (anatomical, functional), patient-reported and safety outcomes. One unanticipated finding in this study was that anatomical outcomes (retinal imaging) were deemed to be the most important by patients, in disagreement with another study on the identification of a standardized set of outcomes for AMD trials.…”

Section: Discussionsupporting

confidence: 78%

“…These findings have the potential to help reduce the variability previously observed in outcome selection in GA trials. 4,10 Several reviews highlighted lack of standardisation in outcome selection for GA and AMD trials 4,5,10,11 and a few studies attempted to prioritize outcomes for research associated with AMD treatment 17 and everyday practice 18 , yet our work is the first to focus on identifying a set of optimum outcomes for GA trials. It takes into consideration different stakeholder groups and compares information from consensus exercises with qualitative interviews.…”

Section: Discussionmentioning

confidence: 99%

“…3 A range of treatment options have been recently tested in trials, but the outcome measures used in GA trials have been variable. 4,5 Meaningful, well-defined outcomes that are relevant to patients, clinicians and policy makers are essential for designing clinical trials designed to guide clinical practice. 6 The adoption of carefully selected core outcomes that are relevant to all stakeholders is strongly recommended by the Core Outcome Measures for Effectiveness Trials (COMET) initiative.…”

Section: Introductionmentioning

confidence: 99%

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Core outcomes for geographic atrophy trials

et al. 2019

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Background/AimsOngoing and recent clinical trials for geographic atrophy (GA) have used different outcomes. The goal of this study was to identify a core outcome set (COS) important for patients, clinicians and researchers, and to propose the use of COS in the design of future GA trials.MethodsFive-component project including: Delphi method with patients and experts, focus groups and interviews with patients, relatives and workers supporting patients. Three hundred and one patients (301) with age-related macular degeneration participated in round 1 of a Delphi exercise. Most subjects had GA; 183 patients (61%) were females and the median (range) age was 77 (50–99) years. In round 2, of the 301 of the first round, 100 participants were randomly selected of whom 76 agreed to take part. In a parallel Delphi exercise, panellists comprised a mix of non-clinical scientists and clinicians (43 in the initial and 21 in the final round). In addition, interviews and focus groups consisting of patients (n=20), family members (n=4) and support workers (n=5) were undertaken.ResultsCore outcomes identified as important for age-related macular degeneration trials were the health of the outer retina, multimodal estimation of lesion size, reading speed, best corrected distance and near acuity, low luminance visual acuity, patient reported visual performance and safety.ConclusionThis study identified a set of core outcomes that should be used in GA trials. The COS include patient-reported outcome measures, near visual acuity, reading speed and assessment of the outer retina.

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Section: Discussionsupporting

confidence: 78%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Core outcomes for geographic atrophy trials

et al. 2019

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“…Neovascular AMD and geographic atrophy (GA) are 2 forms of advanced AMD with GA accounting for approximately 20% of legal blindness in the USA [3]. While anti-VEGF drugs provide effective treatment for neovascular AMD, there is no approved or effective treatment for GA, in spite that many GA clinical trials have been completed or are ongoing to find a viable treatment for preventing the onset of GA or for slowing down the growth of existing GA [4]. Our previous review of GA treatment trials registered in ClinicalTrials.Gov found that 71% of phase 3 GA trials used GA growth rate as the primary outcome measure [4].…”

Section: Introductionmentioning

confidence: 99%

Growth Rate of Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Meta-Analysis of Natural History Studies and Implications for Designing Future Trials

Wang

Ying

2020

Ophthalmic Res

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Introduction: There is no effective treatment for geographic atrophy (GA). To help design future GA treatment trials, we conducted a meta-analysis for GA growth rate and its variability in natural history studies. Methods: Literatures were searched up to April 30, 2020. Data were extracted independently by 2 authors and analyzed using random-effects models. Results: The pooled mean GA growth rate was 1.66 mm²/year (SD = 1.27 mm²/year, I² = 97.0%, p < 0.001) from 23 studies (3,078 patients) reported with growth rate in mm²/year and 0.33 mm/year (SD = 0.17 mm/year, I² = 83.0%, p < 0.001) from 12 studies (1,808 patients) reported with growth rate in mm/year. Larger mean baseline GA area in mm² was associated with larger mean growth rate in both mm²/year and mm/year (p < 0.001). The sample size at 85% power for detecting 15% reduction in growth rate in a 2-arm GA trial is 464 and 209 patients per arm, respectively, for using mm²/year and mm/year as the primary outcome. Discussion/Conclusion: GA natural history studies had a pooled mean (SD) GA growth rate of 1.66 (1.27) mm²/year or 0.33 (0.17) mm/year. Because GA growth rate using mm/year requires smaller sample size than using mm²/year, GA growth rate in mm/year is recommended as the primary outcome measure for future GA trials.

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“…Precise understanding of the monocular contribution of visual function and structural biomarkers to the overall (binocular) vision-related quality of life (VRQoL) in patients is a prerequisite for designing clinical trials testing for efficacy in terms of PROMs. 24 …”

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confidence: 99%

Determinants of Quality of Life in Geographic Atrophy Secondary to Age-Related Macular Degeneration

Künzel

Möller

Lindner

et al. 2020

Invest. Ophthalmol. Vis. Sci.

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Purpose To longitudinally evaluate vision-related quality of life (VRQoL) in geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and define its relation to visual function and structural biomarkers. Methods Patients with GA secondary to AMD were recruited in the context of the prospective, non-interventional, natural-history Directional Spread in Geographic-Atrophy study (NCT02051998). Fundus autofluorescence and infrared reflectance images were semi-automatically annotated for GA. Linear mixed-effects models were applied to investigate the association of putative determinants with the National Eye Institute Visual Function Questionnaire 25 (NEI VFQ-25) VRQoL. Results A total of 87 patients with a mean age ± SD of 77.07 ± 7.49 years were included in the analysis. At baseline, median (IQR) best-corrected visual acuity (BCVA) was 0.3 (0.51) for the better eye and 0.89 (0.76) for the worse eye; 46% of the patients showed binocular and 25.3% monocular non-central GA. The VRQoL composite score was impaired: 69.96 (24.03). Sixty-six patients with a median of 2 (2) follow-up visits after 1.08 (0.78) years were examined longitudinally. In the multivariable cross-sectional analysis, predictors of the VRQoL composite score were BCVA, GA size, and low-luminance visual acuity (LLVA) for the better eye and BCVA, foveal sparing status, and LLVA for the worse eye (cross-validated R 2 = 0.32). In the longitudinal analysis, a similar prediction accuracy for VRQoL was determined (cross-validated R 2 = 0.28). Prediction accuracy for VRQoL did not improve when follow-up time was added as an independent variable. Conclusions Vision-related quality of life is significantly impaired in patients with GA secondary to AMD. The cross-sectional and longitudinal association of VRQoL with visual functional and structural biomarkers supports the validity of the NEI VFQ-25 VRQoL.

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Design Characteristics of Geographic Atrophy Treatment Trials: Systematic Review of Registered Trials in ClinicalTrials.gov

Cited by 23 publications

References 22 publications

Core outcomes for geographic atrophy trials

Core outcomes for geographic atrophy trials

Growth Rate of Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Meta-Analysis of Natural History Studies and Implications for Designing Future Trials

Determinants of Quality of Life in Geographic Atrophy Secondary to Age-Related Macular Degeneration

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