Open-Label Phase 1 Study of CSB-001 Ophthalmic Solution 0.1% to Treat Limbal Stem Cell Deficiency

What this Trial Is About

Researchers are evaluating the safety and effectiveness of CSB-001 Ophthalmic Solution 0.1% in adults with limbal stem cell deficiency LSCD, a condition affecting the cornea. This phase 1, open-label study involves subjects with confirmed LSCD who will receive the investigational drug in one or both eyes. The study includes multiple dosing and observation periods to assess treatment impact and safety over an extended time. Participants will receive CSB-001 eye drops four times daily during two separate 8-week dosing phases, with a 31 to 40-day break dosing holiday between them. The study is divided into phases including initial observation, dosing periods, and a non-interventional observation phase lasting up to 48 weeks. An optional extension phase allows further non-interventional observation for up to 36 months, extending total participation to about three years. Throughout the study, participants will undergo regular safety assessments including adverse event monitoring, slit-lamp biomicroscopy exams, and vision tests best-corrected distance visual acuity. Researchers will also evaluate changes in LSCD severity and vision. The study involves ongoing monitoring during dosing and observation periods, with data collected up to week 144. Participants will have several visits during these phases to assess treatment effects and safety under medical supervision.

Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency