A Prospective, Observational, Non-interventional Clinical Study of Participants With Choroideremia: The NIGHT Study - 07/06/24
, BYRON L. LAM b, M. DOMINIK FISCHER c, #, FRANK G. HOLZ d, MARK E. PENNESI e, †, DAVID G. BIRCH f, EEVA-MARJA SANKILA g, ISABELLE ANNE MEUNIER h, KIMBERLY E. STEPIEN i, JULIANA MARIA FERRAZ SALLUM j, JIANG LI k, DAN YOON k, ‡, SUSHIL PANDA k, ‡, JAMES A. GOW k, ‡for the
NIGHT Study Group1
HIGHLIGHTS |
• | Choroideremia (CHM) is an outer retinal degeneration that leads to blindness. |
• | There is no approved treatment for CHM, and natural history data are scarce. |
• | NIGHT assessed visual acuity decline in participants with CHM over 20 months. |
• | Functional and structural (anatomical) outcomes were assessed over 12 months. |
• | Some outcomes were more sensitive to early degeneration than visual acuity. |
Résumé |
Purpose |
The NIGHT study aimed to assess the natural history of choroideremia (CHM), an X-linked inherited chorioretinal degenerative disease leading to blindness, and determine which outcomes would be the most sensitive for monitoring disease progression.
Design |
A prospective, observational, multicenter cohort study.
Methods |
Males aged ≥18 years with genetically confirmed CHM, visible active disease within the macular region, and best-corrected visual acuity (BCVA) ≥34 Early Treatment Diabetic Retinopathy Study (ETDRS) letters at baseline were assessed for 20 months. The primary outcome was the change in BCVA over time at Months 4, 8, 12, 16, and 20. A range of functional and anatomical secondary outcome measures were assessed up to Month 12, including retinal sensitivity, central ellipsoid zone (EZ) area, and total area of fundus autofluorescence (FAF). Additional ocular assessments for safety were performed.
Results |
A total of 220 participants completed the study. The mean BCVA was stable over 20 months. Most participants (81.4% in the worse eye and 77.8% in the better eye) had change from baseline > −5 ETDRS letters at Month 20. Interocular symmetry was low overall. Reductions from baseline to Month 12 were observed (worse eye, better eye) for retinal sensitivity (functional outcome; −0.68 dB, −0.48 dB), central EZ area (anatomical outcome; −0.276 mm2, −0.290 mm2), and total area of FAF (anatomical outcome; −0.605 mm2, −0.533 mm2). No assessment-related serious adverse events occurred.
Conclusions |
Retinal sensitivity, central EZ area, and total area of FAF are more sensitive than BCVA in measuring the natural progression of CHM.
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| Supplemental Material available at AJO.com. |
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| Meeting Presentations: Interim data were first presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2018, April 29 to May 3, 2018 (Pennesi ME, Lam BL, Fischer MD, et al. The Natural History of the Progression of Choroideremia [NIGHT] Study: Longitudinal Changes in Visual Acuity over 12 Months). Interim data from the NIGHT study were also presented as oral presentations at the virtual European Society of Retina Specialists (EURETINA) Congress 2021, September 9 to 12, 2021 (Lam BL, MacLaren RE, Fischer MD, et al. Updated Results from the NIGHT Study: Natural Progression of Choroideremia) and at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2021, April 1 to 7, 2021 (Lam BL, MacLaren RE, Fischer MD, et al. NIGHT Study: Natural Progression of Choroideremia). |
Vol 263
P. 35-49 - juillet 2024 Retour au numéro
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