A Multicenter, Phase I/II, Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Intrastromal Injection of Genome-editing GEB-101 in Participants With TGFBI-related Corneal Dystrophy
GenEditBio Limited
Summary
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
Description
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…
Eligibility
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Males or females, age 18-70 years (inclusive). * TGFBI gene mutation confirmed. * Patients with corneal dystrophy who are symptomatic. * Willing and able to give signed informed consent and follow study instructions. Exclusion Criteria: * In either eye: Have any active infection, glaucoma, corneal disease, or other ocular conditions that may affect the assessment of efficacy or safety. * In the study eye: Have a history of specific intraocular or corneal surgery, or the presence of active intraocular inflammation or vitreous hemorrhage. * Patient with other corneal dis…
Interventions
- GeneticGEB-101 Injection
Single intrastromal injection
Locations (2)
- University of California, Los Angeles (UCLA)Los Angeles, California
- Fudan University Eye & ENT HospitalShanghai, Shanghai Municipality