Summary

Eligibility
for people ages 18-70 (full criteria)
Location
at Los Angeles, California and other locations
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