An Early Phase 1 interventional study of MSC Secretome Eye Drops in Mesenchymal Stromal Cells, Cornea and Corneal Defect, sponsored by University of Illinois at Chicago. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-07.
Sponsored by University of Illinois at Chicago · Early Phase 1, Interventional, and Treatment
This study is a longitudinal assessment using a classic dose-escalation study design to assess the safety and maximal tolerated dose (MTD) of topical MSC Secretome eye drops. The study will be conducted at Illinois Eye and Ear Infirmary located at University of Illinois at Chicago. The study will use anterior segment Optical Coherence Tomography (OCT)/Scheimpflug Imaging, esthesiometry, and visual analogue scale (VAS) to assess treatment tolerability.
The "Safety of Topical Mesenchymal Stromal Cell Secretome for Ocular Surface Disease" study is designed to evaluate the safety and maximal tolerated dose (MTD) of topical MSC Secretome eye drops in patients with chronic ocular surface disease through a dose-escalation study under a 28-day topical application protocol, and also obtain a preliminary observation on the following:
The objective is to determine the dose of MSC Secretome through a first-in-human study through a dose-escalation strategy targeting a toxicity rate of 33% or less.
130 studies on the registry are indexed under Corneal Diseases; 43 are open to participants now.
This study's enrollment of 9 is below the median of 32 across 78 interventional studies indexed under Corneal Diseases.
Browse Corneal Diseases studies →University of Illinois at Chicago is the lead sponsor of 515 studies on the registry; 133 are open to participants now.
Of its 31 completed or terminated interventional studies of FDA-regulated products, 18 (58%) have results posted.
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Exclusion Criteria:
Escalating doses of allogenic MSC eye drops will be assigned at the lowest dose level.
Biological: MSC Secretome Eye Drops
Escalating doses of allogenic MSC eye drops will be assigned at the medium dose level.
Biological: MSC Secretome Eye Drops
Escalating doses of allogenic MSC eye drops will be assigned at the high dose level.
Biological: MSC Secretome Eye Drops
MSC Secretome eye drop will be dispensed.
The Number of Participants In Each Dose Frequency Group With No Dose Limiting Toxicities Until Day 28 From The Start of Topical Allogeneic BM-MSC Secretome
Safety was defined with the absence of dose-limiting toxicity in the study eye until Day 28. Adverse events were defined using National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v5). Ocular adverse events included: Grade 2: vision loss ≤ 3 lines * increased corneal fluorescein staining by \>50% * new conjunctivitis, scleritis, or uveitis 1-2+ cells in AC * increased corneal haze \> 50%, stromal loss 10-30% Grade 3: loss of vision by \> 3 lines up to 20/200 * new mild to moderate infectious/inflammatory keratitis with vision loss up to 20/200 * new corneal ulceration with \> 30% stromal loss * new uveitis with 3-4+ cells in AC Grade 4: loss of vision by \> 3 lines worse than 20/200 * new severe infectious/inflammatory keratitis * corneal perforation * Anaphylactic reaction, New SJS Note: Any grade 2 adverse event that lasted longer than 2 weeks or any adverse event of grades 3 or 4 was considered DLT. We also recorded all adverse events at each visit.
Time frame: Day 28
Corneal Epithelial Integrity
The primary efficacy outcome measure was improved corneal epithelial barrier at 28 days compared to baseline - defined as ≥ 50% reduction in corneal fluorescein staining score using National Eye Institute (NEI) grading scale as measured by the investigators via slit lamp examination and assessment of slit lamp images.
Time frame: Day 28
Visual Acuity
Mean Change in best-corrected distance visual acuity (BCVA) in ETDRS letters on day 28 relative to baseline for each dose frequency group. The ETDRS letter score ranges from 0 to 100, where higher scores indicate better visual acuity.
Time frame: Day 28
Change in Corneal Scarring / Haze Compared to Baseline
Corneal scarring/haze was assessed as the size of the hyperreflective area on AS OCT imaging (Cirrus 6000; HD cornea, a-scans) to assess the treatment effect on DAY #28 relative to baseline.
Time frame: Day 28
Time to Improvement of Corneal Epithelial Barrier
The time required for an improved epithelial barrier function was assessed at each visit throughout the trial.
Time frame: Baseline, Days 7, 14, 28, 56, 90
Tolerability of MSC Secretome Drops (Change in VAS Score on Day 28 Compared to Baseline)
Mean change in the patients' self-reported severity of discomfort and pain with visual analog scale (VAS; range 0 -100) on Day 28 compared to baseline for each dose frequency group. The higher numbers indicate more severe discomfort and pain.
Time frame: Day 28
Change in Central Corneal Epithelial Thickness
Mean change in Central Corneal Epithelial Thickness on Day 28 from baseline.
Time frame: Day 28
Durability of Corneal Epithelial Status Improvement
In patients with an improvement in corneal epithelial integrity defined as ≥50% improvement in corneal fluorescein staining with NEI scale on Day 28 from baseline, was the improvement maintained on Days 42, 56 and 90.
Time frame: Days 42, 56, 90
Nine participants were enrolled and received study treatment between January 2024 and November 2024.
| Milestone | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Started | 3 | 3 | 3 |
| Completed | 3 | 3 | 1 |
| Not completed | 0 | 0 | 2 |
Safety was defined with the absence of dose-limiting toxicity in the study eye until Day 28. Adverse events were defined using National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v5). Ocular adverse events included: Grade 2: vision loss ≤ 3 lines * increased corneal fluorescein staining by \>50% * new conjunctivitis, scleritis, or uveitis 1-2+ cells in AC * increased corneal haze \> 50%, stromal loss 10-30% Grade 3: loss of vision by \> 3 lines up to 20/200 * new mild to moderate infectious/inflammatory keratitis with vision loss up to 20/200 * new corneal ulceration with \> 30% stromal loss * new uveitis with 3-4+ cells in AC Grade 4: loss of vision by \> 3 lines worse than 20/200 * new severe infectious/inflammatory keratitis * corneal perforation * Anaphylactic reaction, New SJS Note: Any grade 2 adverse event that lasted longer than 2 weeks or any adverse event of grades 3 or 4 was considered DLT. We also recorded all adverse events at each visit.
| participants | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| The Number of Participants In Each Dose Frequency Group With No Dose Limiting Toxicities Until Day 28 From The Start of Topical Allogeneic BM-MSC Secretome | 3 | 3 | 1 |
The primary efficacy outcome measure was improved corneal epithelial barrier at 28 days compared to baseline - defined as ≥ 50% reduction in corneal fluorescein staining score using National Eye Institute (NEI) grading scale as measured by the investigators via slit lamp examination and assessment of slit lamp images.
| participants | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Corneal Epithelial Integrity | 3 | 3 | 1 |
Mean Change in best-corrected distance visual acuity (BCVA) in ETDRS letters on day 28 relative to baseline for each dose frequency group. The ETDRS letter score ranges from 0 to 100, where higher scores indicate better visual acuity.
| ETDRS Letter Scores | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Visual Acuity | 15 ± 5 | 12.7 ± 10.8 | -10.7 ± 13 |
Corneal scarring/haze was assessed as the size of the hyperreflective area on AS OCT imaging (Cirrus 6000; HD cornea, a-scans) to assess the treatment effect on DAY #28 relative to baseline.
| (µm)² | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Change in Corneal Scarring / Haze Compared to Baseline | 0 ± 0 | 0 ± 0 | 0 ± 0 |
The time required for an improved epithelial barrier function was assessed at each visit throughout the trial.
| participants | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Time to Improvement of Corneal Epithelial Barrier | 0 | 0 | 0 |
Mean change in the patients' self-reported severity of discomfort and pain with visual analog scale (VAS; range 0 -100) on Day 28 compared to baseline for each dose frequency group. The higher numbers indicate more severe discomfort and pain.
| VAS Score | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| Tolerability of MSC Secretome Drops (Change in VAS Score on Day 28 Compared to Baseline) | -7 ± 11.3 | 13.3 ± 32 | 4.7 ± 31 |
Mean change in Central Corneal Epithelial Thickness on Day 28 from baseline.
| micrometers | Low Dose of Allogenic MSC Drops | Medium Dose of Allogenic MSC Drops | High Dose of Allogenic MSC Drops |
|---|---|---|---|
| Change in Central Corneal Epithelial Thickness | 7 ± 8 | -1.6 ± 2.9 | 6 ± 0 |
In patients with an improvement in corneal epithelial integrity defined as ≥50% improvement in corneal fluorescein staining with NEI scale on Day 28 from baseline, was the improvement maintained on Days 42, 56 and 90.
| participants | Low Dose of Allogenic MSC Drops | Medium Dose of Allogenic MSC Drops | High Dose of Allogenic MSC Drops |
|---|---|---|---|
| Durability of Corneal Epithelial Status Improvement | 0 | 0 | 0 |
Collected over From first dose (Day 0) through Day 90 follow-up (90 days after start of treatment).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Low Dose of Allogeneic MSC Drops | 0/3 (0%) | 0/3 (0%) | 0/3 (0%) |
| Medium Dose of Allogeneic MSC Drops | 0/3 (0%) | 0/3 (0%) | 0/3 (0%) |
| High Dose of Allogeneic MSC Drops | 0/3 (0%) | 0/3 (0%) | 2/3 (66.7%) |
| Event | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops |
|---|---|---|---|
| New corneal epithelial defectEye disorders | 0/3 | 0/3 | 1/3 |
| Decreased visual acuity (met DLT criteria)Eye disorders | 0/3 | 0/3 | 1/3 |
Note: In the high-dose group, 2 participants discontinued study drops after 7 days due to adverse events and had some missing data in the later follow-up visits.
| Age, Continuous(Years) | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops | Total |
|---|---|---|---|---|
| Mean | 42 ± 21.3 | 67.7 ± 3.5 | 64 ± 14.1 | 57.9 ± 17.6 |
| Sex: Female, Male(Participants) | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops | Total |
|---|---|---|---|---|
| Female | 2 | 1 | 1 | 4 |
| Male | 1 | 2 | 0 | 3 |
| Ethnicity (NIH/OMB)(Participants) | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops | Total |
|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 1 | 1 |
| Not Hispanic or Latino | 3 | 3 | 0 | 6 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Etiology of persistent corneal epithelial disease(Eyes) | Low Dose of Allogeneic MSC Drops | Medium Dose of Allogeneic MSC Drops | High Dose of Allogeneic MSC Drops | Total |
|---|---|---|---|---|
| Number | 3 | 3 | 1 | 7 |
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University of Illinois at Chicago