Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy (CLARITY)

GenEditBio Limited

Status and phase

Begins enrollment this month

Phase 2

Phase 1

Conditions

Corneal Dystrophies, Hereditary

Hereditary Corneal Dystrophy

Corneal Dystrophies

Corneal Abnormality

Treatments

Genetic: GEB-101 Injection

Study type

Interventional

Funder types

Industry

Identifiers

NCT07750678

GEB-101-101

Details and patient eligibility

About

The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy

Full 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 follow-up conducted from Year 2 to Year 5, followed by telephone or questionnaire-based follow-up until Year 15.

Enrollment

30 estimated patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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 disorders that may hinder therapeutic surgery or interfere with the interpretation of study results.
Prior gene therapy administration in either eye.
Active infection occurring or suspected within 4 weeks prior to the investigational product, requiring systemic anti-infective therapy.
Subjects with a history of major cardiovascular events within 6 months, the presence of any clinically significant condition, a diagnosis of malignancy within 5 years (with specified exceptions), or active infection with HIV, syphilis, hepatitis C, or hepatitis B.
Female subjects who are pregnant, breastfeeding, or planning to become pregnant during the study period.
Disease, condition, or disorder that in the judgement of Investigator could confound study assessments or limit compliance to study protocol.