Photo Credit: Namaki

The following is a summary of “A Prospective, Observational, Non-Interventional Clinical Study of Participants With Choroideremia: The NIGHT Study,” published in the February 2024 issue of American Journal of Ophthalmology by MacLaren et al.

Researchers conducted the NIGHT study to evaluate the natural progression of choroideremia (CHM), an X-linked inherited chorioretinal degenerative disease causing blindness, and identify the most sensitive outcome measures for disease progression monitoring.

They evaluated males aged ≥18 years with genetically confirmed CHM, exhibiting visible, active disease within the macular region, and baseline best-corrected visual acuity (BCVA) of ≥34 Early Treatment Diabetic Retinopathy Study (ETDRS) letters over 20 months. The primary focus was assessing BCVA changes at Months 4, 8, 12, 16, and 20. Secondary outcome measures, including retinal sensitivity, central ellipsoid zone (EZ) area, and total fundus autofluorescence (FAF) area, were also evaluated up to Month 12. Ocular safety assessments were conducted.

The results showed that of 220 participants who completed the study, the mean BCVA remained stable over the 20 months. At Month 20, most participants (81.4% in the worse eye and 77.8% in the better eye) exhibited a change from baseline of >-5 ETDRS letters. Interocular symmetry was notably low. Reductions from baseline to Month 12 were observed in both the worse eye and the 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 FAF (anatomical outcome; -0.605 mm², -0.533 mm²). There were no serious adverse events related to the assessment.

Investigators concluded that CHM progression is better tracked by retinal sensitivity, central EZ area, and total FAF area than standard BCVA measurements.

Source: ajo.com/article/S0002-9394(24)00024-2