ClinicalTrials.gov record NCT07750678

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 23, 2026

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

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).

Age18 years old to 70 years old

SexAll

LocationChina, United States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1/2 study tests a single intrastromal injection of genome-editing GEB-101 in adults with TGFBI-related corneal dystrophy. Phase 1 is an open-label, dose-escalation study with planned cohorts and expansion at a recommended Phase 2 dose. Phase 2 transitions to a randomized, double-blind, vehicle-controlled, self-controlled design. Primary follow-up is one year, with a proposed long-term follow-up extending up to 15 years for treated participants.

What it is studying. The study is testing the safety, tolerability, and effects of a single intrastromal injection of GEB-101 (a genome-editing product) in people with TGFBI-related corneal dystrophy. Key outcomes include dose-limiting toxicities, overall and serious adverse events, changes in best-corrected visual acuity, corneal haze, and time to disease recurrence.

What the researchers hope to learn. To evaluate safety and tolerability (including dose-limiting toxicities and adverse events) and to measure effects on vision, corneal haze, and time to disease recurrence in people with TGFBI-related corneal dystrophy.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Genetic: GEB-101 Injection

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive a single intrastromal injection of GEB-101; Phase 1 is a dose-escalation (several dose cohorts) and Phase 2 transitions to a randomized, double‑blind, vehicle‑controlled, self‑controlled design. You will have scheduled eye assessments (including measurements of vision/BCVA and corneal haze) and safety checks to record any side effects; dose‑limiting toxicities are assessed in the first 30 days and adverse events are followed through 360 days. After the primary one‑year follow‑up, people who received GEB-101 will be invited to long‑term follow‑up with in‑person visits from Year 2 to Year 5, then telephone or questionnaire follow‑up through Year 15.

How the study is structured. This is a seamless Phase 1/2, multicenter interventional study. Phase 1 is single-arm and open-label with dose-escalation cohorts and two expansion cohorts at the recommended Phase 2 dose. Phase 2 is described in the record as randomized, double-blind, vehicle-controlled and self-controlled. The intervention is a single intrastromal injection of GEB-101. The record describes a primary follow-up period of 1 year and a proposed long-term follow-up with in-site visits from Year 2 to Year 5, then telephone or questionnaire follow-up through Year 15 for those who received GEB-101.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry lists this study under Hereditary Corneal Dystrophy; Corneal Abnormality; Corneal Dystrophies; Corneal Dystrophies, Hereditary. Does it apply to my diagnosis?
This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The registry says recruitment has not started. When is it expected to open, and can I register interest now?
The listed sites are in China, United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

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Important dates

Study start: October 2026 (estimated)
Primary completion: August 2027 (estimated)
Study completion: December 2028 (estimated)
Target enrollment: 30 participants (estimated)
Last updated on registry: Sep 10, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: GenEditBio Limited

Participating sites (2)

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

Terms used on this page · explanations by MedicalUpdates.ai

informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
investigational product: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Phase 1: An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
single-arm: All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose-escalation: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
recommended Phase 2 dose: The dose chosen at the end of a phase 1 study for testing in later studies, based on safety and other measurements.
Phase 2: A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
randomized: Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind: Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
follow-up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
long-term follow-up: Observation that continues for years after the study treatment, often required for gene and cell therapies.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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