Summary

Eligibility
for people ages 18-70 (full criteria)
Location
at UCLA
Dates
study started
study ends around

Description

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

Official Title

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

Details

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.

Keywords

Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary, Corneal Dystrophy, TGFBI Corneal Dystrophies, Granular Corneal Dystrophy, Lattice Corneal Dystrophy, Reis-Bücklers Corneal Dystrophy, Thiel-Behnke Corneal Dystrophy, TGFBI-related corneal dystrophies, Gene Therapy, Hereditary Corneal Dystrophies, Familial Amyloid Neuropathies, Thiel-Behnke type Corneal dystrophy, GEB-101 Injection

Eligibility

You can join if…

Open to people ages 18-70

  • 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.

You CAN'T join if...

  • 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.

Locations

  • University of California, Los Angeles (UCLA)
    Los Angeles California 90095 United States
  • Fudan University Eye & ENT Hospital
    Shanghai Shanghai Municipality 200031 China

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
GenEditBio Limited
ID
NCT07750678
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 30 study participants
Last Updated