Clinical trial NCT05204329: Safety of Topical Mesenchymal Stromal Cell Secretome for Ocular Surface Disease
Official title: Safety of Topical Mesenchymal Stromal Cell Secretome for Ocular Surface Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Mesenchymal Stromal Cells
Cornea
Corneal Defect
Corneal Epithelium Defect
Keywords: Mesenchymal Stromal Cells
Corneal Defect
Corneal Epithelium Defect
Lead sponsor: University of Illinois at Chicago
Sponsor class: OTHER
Collaborators: name: National Eye Institute (NEI)
name: National Institutes of Health (NIH)
First posted: 2022-01-24
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: A longitudinal assessment using a classic dose-escalation study design to evaluate the safety and maximal tolerated dose of topical MSC Secretome eye drops
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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:

1. Incidence of treatment emergent adverse events (TEAE) assessed at 28 days following treatment initiation
2. Proportion of patients with improved corneal epithelial barrier at 28 days compared to baseline
3. Final visual acuity, corneal epithelial thickness, corneal stromal haze, corneal sensation, and treatment tolerability

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.

Eligibility criteria: Inclusion Criteria:

* Patients 18 years of age or older
* Chronic corneal epithelial disease with fluorescein staining score ≥ 6 by NEI grading scale
* Reduced corneal sensation (≤ 4 cm measured by Cochet Bonnet esthesiometry) in at least one corneal quadrant
* A stable ocular surface with no objective clinical evidence of significant (\> 50%) improvement/worsening of the epithelial disease in the last 30 days
* Epithelial disease refractory to conventional non-surgical treatments (e.g., preservative-free artificial tears, gels or ointments; discontinuation of preserved topical drops; anti-inflammatory therapy)

Exclusion Criteria:

* Any active or suspected ocular infection (bacterial, viral, fungal or protozoal).
* Evidence of corneal ulceration with stromal loss \> 10%
* Presence of an epithelial defect ≥1.0 mm in the largest diameter in the affected eye
* Presence of any size epithelial defect that has been persistent for more than 30 days
* Patients unable to discontinue or intermittently remove therapeutic contact lens in the study eye (to apply drops) during the 4-week study period
* History of any ocular surgery (including laser or refractive surgical procedures) in the affected eye within the 3 months prior to study enrollment
* History of chemical injury within the last 6 months prior to study enrollment Known hypersensitivity to one of the components of the study or procedural medications (e.g.,fluorescein)
* History of drug, medication or alcohol abuse or addiction
* Use of any investigational agent within 4 weeks of screening visit
* History of previous enrollment in the MSC Secretome Study at a lower dose
* Participation in another clinical study at the same time as the present study
* Participants who are pregnant at the time of study enrollment

Primary outcomes:
measure: Epithelial Status Assessment
timeFrame: Day 28
Secondary outcomes:
measure: Visual Acuity
timeFrame: Baseline, Days 7, 14, 28, 56, 90
measure: Corneal Epithelial thickness
timeFrame: Baseline, Days 14, 28, 56, 90
measure: Corneal Scarring / Haze
timeFrame: Baseline, Days 14, 28, 56, 90
measure: Tolerability of MSC secretome drops
timeFrame: Baseline, Days 7, 14, 28, 56, 90
measure: Durability of Corneal Epithelial Status Improvement
timeFrame: Baseline, Days 7, 14, 28, 56, 90
measure: Time to Improvement of Corneal Epithelial Barrier
timeFrame: Baseline, Days 7, 14, 28, 56, 90
Other outcomes:
Locations:
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
Study officials:
affiliation: University of Illinois at Chicago
name: Ali R Djalilian, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Illinois at Chicago
name: Charlotte E Joslin, OD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Illinois at Chicago
name: Elmer Y Tu, MD
role: PRINCIPAL_INVESTIGATOR
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