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Not yet recruiting NCT07750678

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

Phase I / Phase II Interventional Hereditary Corneal Dystrophy Corneal Abnormality Corneal Dystrophies Corneal Dystrophies, Hereditary

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: GEB-101 Injection.
Who it may be relevant to
Registry conditions: Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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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

Overview

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

Detailed 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.

Interventions

  • Genetic GEB-101 Injection
    Single intrastromal injection

Primary outcome measures

  • Incidence of DLTs [Time frame: 30 Days]
  • Incidence of AEs [Time frame: 360 Days]
  • Incidence of SAEs [Time frame: 360 Days]
Secondary outcome measures (3)
  • 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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • University of California, Los Angeles (UCLA) — Los Angeles
China · 1 center
  • Fudan University Eye & ENT Hospital — Shanghai

Identifiers

NCT: NCT07750678 · GEB-101-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗