Microperimetry as an Outcome Measure Improves Patient Eligibility and Efficacy Detection in RPGR Gene Therapy Trials.
Amandeep Singh Josan, Laura J Taylor, Shabnam Raji et al.
✦ AI-curated · Sources linked
In 30 seconds
This study evaluated the impact of primary endpoint selection on patient eligibility and efficacy detection in RPGR gene therapy trials, comparing low luminance visual acuity (LLVA) with microperimetry measures. In a retrospective analysis of a phase 1/2 to 3 trial involving 15 patients, microperimetry identified 64% of patients as responders, compared to 20% with LLVA, highlighting its superior sensitivity.
Key findings
- Microperimetry identified 5 of 11 patients (45%) as responders, compared to 20% with LLVA.
- Using central 16-point microperimetry, 7 of 11 patients (64%) met responder criteria.
- LLVA excluded more patients due to floor effects than microperimetry.
- No responders were identified in untreated fellow eyes.
Why it matters
The choice of primary endpoint in clinical trials can significantly influence patient eligibility and the perceived efficacy of treatments. More sensitive measures like microperimetry may provide a clearer understanding of treatment benefits, potentially leading to better patient outcomes.