Limbal Stem-cell DeficiencyPhase 1NCT03957954
Stem Cell Therapy for Limbal Stem Cell Deficiency
A Phase 1 study testing cLSC and Scleral contact lens (SCL) in people with Limbal Stem-cell Deficiency, run by University of California, Los Angeles at 1 site in United States.
Trial summary
This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Limbal Stem-cell Deficiency
- What is being tested?
- cLSC and Scleral contact lens (SCL)
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2020to 30 Apr 2027
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Main inclusion Criteria:
- Male or female ≥18 years of age.
- Best corrected visual acuity in the affected eye of 20/160 or less.
- Documentation of a LSCD diagnosis and the central cornea is affected.
- Absence of lagophthalmos and eyelid abnormality
- Adequate forniceal depth is ≥ 5 mm.
- LSCD fails to resolve by surgical treatments of the ocular surface during the previous 6 months of screening visit.
- If the etiology of LSCD is due to chemical injury, a minimal interval of 1 year since the initial chemical injury is required.
- A Schirmer test result at 5 minute of ≥5 mm of wetting.
- Absence of active infectious keratitis in either eye at the Enrollment Visit.
- Have a life expectancy ≥ 2 years after enrollment.
Main exclusion Criteria:
- Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Exposure keratopathy or lagophthalmos of the study eye.
- Persistent severe ocular surface inflammation and/or meibomian gland dysfunction
- Chemical injury occurred less than 12 months ago.
- Presence of ocular surface tumor.
- Uncontrolled diabetes with last hemoglobin A1C (HgA1C) >8.5.
- Presence of known allergies to any of the cLSC components.
- Current participation in another simultaneous medical investigation or trial.
- Unable to be compliant with or complete the requirements of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California
Los Angeles, California, 90095
About this study
Official title: Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events (safety).
Read the detailed description
20 patients with severe to total Limbal Stem Cell Deficiency (LSCD) in one eye attributable to injury or ocular surgery. The first 5 eyes will receive the cLSCs to determine the feasibility and safety. Then the subsequent 15 eyes will be randomized into cLSC group and a scleral lens treatment (SCL) control group in a 2:1 ratio in blocks of 3 or 6 using a random number generator.
- Conditions
- Limbal Stem-cell Deficiency
- Being tested
- cLSC, Scleral contact lens (SCL)
- Medical subject headings
- Limbal Stem Cell Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cLSCBiological
Transplantation of cLSC
Scleral contact lens (SCL)Device
Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface.
| Study group | Role | What they receive |
|---|---|---|
| Cultivated Limbal Stem-Cells (cLSC) | Experimental | One dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter. Receives: Biological: cLSC |
| Scleral Contact Lens Device (SCL) | Active comparator | Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface. Receives: Device: Scleral contact lens (SCL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation
Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.
Measured: 12 months for all participants
Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.
Measured: 12 months for all participants
Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination
Feasibility assessed by successful cLSC manufacturing without contamination.
Measured: 12 months for all participants
Secondary outcomes (2)
Changes in the Area of Corneal Epithelial Defect
Evaluate changes in the area of corneal epithelial defect (ED) or lack of ED.
Measured: At 6 months and 12 months in both cLSC and the control groups
Changes in the Clinical Score
Evaluate changes in the clinical score determined by slit lamp examination using fluorescein staining and confocal imaging.
Measured: At 6 months and 12 months in both cLSC and the control groups
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2019
- Study start30 Sep 2020
- Primary completion4 Feb 2027 (estimated)
- Study completion30 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limbal Stem-cell DeficiencyStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- National Eye Institute (NEI), California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Principal investigator, Sophie Deng, MD, PhD, University of California, Los Angeles
- Investigators
- Sophie Deng, MD, PhD, Principal investigator, Stein Eye Institute UCLA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03957954
- Sponsor's protocol ID
- V001
- Other IDs
- R01EY021797
- Status last verified
- Feb 2026
- First submitted
- 21 Feb 2019
- Last updated
- 10 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
- ClinicalTrials.gov ID
- NCT03957954
- Acronym / sponsor ID
- V001
- Conditions
- Limbal Stem-cell Deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- National Eye Institute (NEI), California Institute for Regenerative Medicine (CIRM)
- Study start
- 30 Sep 2020 (actual)
- Primary completion
- 4 Feb 2027 (estimated)
- Study completion
- 30 Apr 2027 (estimated)
- First posted
- 21 May 2019 (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.
This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events (safety).
Detailed description
20 patients with severe to total Limbal Stem Cell Deficiency (LSCD) in one eye attributable to injury or ocular surgery. The first 5 eyes will receive the cLSCs to determine the feasibility and safety. Then the subsequent 15 eyes will be randomized into cLSC group and a scleral lens treatment (SCL) control group in a 2:1 ratio in blocks of 3 or 6 using a random number generator.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cultivated Limbal Stem-Cells (cLSC) | Experimental | Biological: cLSCOne dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter. |
| Scleral Contact Lens Device (SCL) | Active comparator | Device: Scleral contact lens (SCL)Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| cLSCTransplantation of cLSC | Biological | Cultivated Limbal Stem-Cells (cLSC) |
| Scleral contact lens (SCL)Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface. | Device | Scleral Contact Lens Device (SCL) |
Primary outcome
Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation
Time frame: 12 months for all participants
Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.
Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
Time frame: 12 months for all participants
Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.
Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination
Time frame: 12 months for all participants
Feasibility assessed by successful cLSC manufacturing without contamination.
2 secondary outcomes
Changes in the Area of Corneal Epithelial Defect
Time frame: At 6 months and 12 months in both cLSC and the control groups
Changes in the Clinical Score
Time frame: At 6 months and 12 months in both cLSC and the control groups
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 Feb 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Sophie Deng, MD, PhD principal investigator
Stein Eye Institute UCLA
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing cLSC and Scleral contact lens (SCL) in people with Limbal Stem-cell Deficiency, run by University of California, Los Angeles at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: under way, not looking for participants, as of the sponsor's last update on 10 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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.
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?
Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation (time frame: 12 months for all participants). 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 Feb 2026.