Hereditary Corneal DystrophyPhase 1/2CLARITYNCT07750678
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy
A Phase 1/2 study testing GEB-101 Injection in people with Hereditary Corneal Dystrophy and Corneal Abnormality, run by GenEditBio Limited at 2 sites in 2 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies
- What is being tested?
- GEB-101 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Oct 2026to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
United States1 site
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
China1 site
Fudan University Eye & ENT Hospital
Shanghai, Shanghai Municipality, 200031
About this study
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
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
Read the 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.
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Being tested
- GEB-101 Injection
- Keywords
- Corneal Dystrophies, 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
- Medical subject headings
- Corneal Dystrophies, Hereditary, Amyloid Neuropathies, Familial, Corneal dystrophy, Thiel-Behnke type
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GEB-101 InjectionGenetic
Single intrastromal injection
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 | Experimental | GEB-101 dose 1 GEB-101 dose 2 GEB-101 dose 3 GEB-101 dose 4 GEB-101 dose 5 Receives: Genetic: GEB-101 Injection |
| Phase 2 | Experimental | GEB-101(RP2D) Receives: Genetic: GEB-101 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of DLTs
Number and proportion of dose limited toxicity（DLTs）
Measured: 30 Days
Incidence of AEs
Number and severity of overall and ocular and systemic adverse events (AEs)
Measured: 360 Days
Incidence of SAEs
Number and severity of overall and ocular and systemic serious adverse events (SAEs)
Measured: 360 Days
Secondary outcomes (3)
Change in BCVA from baseline over time
Measured: 360 Days
Change in corneal haze level from baseline over time
Measured: 360 Days
Time to recurrence of disease
Measured: 360 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2026
- Study startOct 2026 (estimated)
- Primary completionAug 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Corneal DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1, single group assignment; Phase 2, parallel assignment
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GenEditBio Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07750678
- Sponsor's protocol ID
- GEB-101-101
- Acronym
- CLARITY
- Status last verified
- Jul 2026
- First submitted
- 2 Aug 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07750678
- Acronym / sponsor ID
- CLARITY / GEB-101-101
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Keywords
- Corneal Dystrophies, Corneal Dystrophy, TGFBI Corneal Dystrophies, Granular Corneal Dystrophy, Lattice Corneal Dystrophy, Reis-Bücklers Corneal Dystrophy, Thiel-Behnke Corneal Dystrophy, TGFBI-related corneal dystrophies
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- GenEditBio LimitedIndustry (pharma, biotech, devices)
- Study start
- Oct 2026 (estimated)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 6 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Phase 1, single group assignment; Phase 2, parallel assignment
| Arm | Type | Receives |
|---|---|---|
| Phase 1 | Experimental | Genetic: GEB-101 InjectionGEB-101 dose 1 GEB-101 dose 2 GEB-101 dose 3 GEB-101 dose 4 GEB-101 dose 5 |
| Phase 2 | Experimental | Genetic: GEB-101 InjectionGEB-101(RP2D) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GEB-101 InjectionSingle intrastromal injection | Genetic | Phase 1, Phase 2 |
Primary outcome
Incidence of DLTs
Time frame: 30 Days
Number and proportion of dose limited toxicity（DLTs）
Incidence of AEs
Time frame: 360 Days
Number and severity of overall and ocular and systemic adverse events (AEs)
Incidence of SAEs
Time frame: 360 Days
Number and severity of overall and ocular and systemic serious adverse events (SAEs)
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GEB-101 Injection in people with Hereditary Corneal Dystrophy and Corneal Abnormality, run by GenEditBio Limited at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of DLTs (time frame: 30 Days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.