Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa
Michel Michaelides; Cagri G. Besirli; Yesa Yang; Thales A. C. de Guimarães; Sui Chien Wong; Rachel M. Huckfeldt; Jason Comander; José‐Alain Sahel · 2024 · American Journal of Ophthalmology
WASTE classifies this as Failed Experiment Report · AI classification, approximate
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Abstract
PURPOSE: To assess the safety and efficacy of AAV5-hRKp.RPGR in participants with retinitis pigmentosa GTPase regulator (RPGR)-associated X-linked retinitis pigmentosa (XLRP). DESIGN: Open-label, phase 1/2 dose escalation/expansion study (ClinicalTrials.gov Identifier: NCT03252847). METHODS: vg/ml) was administered to the poorer-seeing eye (n = 10). Dose confirmation (intermediate dose) was carried out in 3 pediatric participants. In the dose expansion phase, 36 participants were randomized 1:1:1 to immediate (low or intermediate dose) or deferred (control) treatment. The primary outcome was s
Abstract by Michel Michaelides; Cagri G. Besirli; Yesa Yang; Thales A. C. de Guimarães; Sui Chien Wong; Rachel M. Huckfeldt; Jason Comander; José‐Alain Sahel, American Journal of Ophthalmology (2024) — licensed CC BY 4.0.
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Metadata source: OpenAlex · DOI 10.1016/j.ajo.2024.05.034