The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
This is a Phase 1/2 seamless, adaptive, multi-center, sequential study evaluates the safety and efficacy of GEB-101 in adult participants with TGFBI-related corneal dystrophy. The Phase 1 part is a single-arm, open-label, dose-escalation study with three planned cohorts , followed by 2 expansion cohorts at the recommended Phase 2 dose. The Phase 2 transitions to a randomized, double-blind, vehicle-controlled, self-controlled design. After completing the primary 1-year follow-up, a long-term follow-up will be proposed to all the participants who received GEB-101 treatment, with close in site follow-up conducted from Year 2 to Year 5, followed by telephone or questionnaire-based follow-up until Year 15.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Single intrastromal injection
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Fudan University Eye & ENT Hospital
Shanghai, Shanghai Municipality, China
Incidence of DLTs
Number and proportion of dose limited toxicity(DLTs)
Time frame: 30 Days
Incidence of AEs
Number and severity of overall and ocular and systemic adverse events (AEs)
Time frame: 360 Days
Incidence of SAEs
Number and severity of overall and ocular and systemic serious adverse events (SAEs)
Time frame: 360 Days
Change in BCVA from baseline over time
Time frame: 360 Days
Change in corneal haze level from baseline over time
Time frame: 360 Days
Time to recurrence of disease
Time frame: 360 Days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.