This is a Phase III, multicenter, randomized, double-masked, vehicle-controlled, parallel-group clinical trial designed to evaluate the efficacy, safety, and tolerability of d-MAPPS™ Ophthalmic Solution in adult subjects with chronic ocular graft-versus-host disease (oGVHD). Eligible subjects will receive masked study treatment for 90 days, with the primary efficacy assessment performed at the Day 90 study visit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
Sterile, preservative-free ophthalmic solution administered as two drops into each eye four times daily for 90 consecutive days.
Matching vehicle control supplied in identical masked containers and administered as two drops into each eye four times daily for 90 consecutive days.
Beverly Hills Institute of Ophthalmology
Beverly Hills, California, United States
RECRUITINGBeverly Hills Optometry
Beverly Hills, California, United States
RECRUITINGRegenerative Ocular Immunobiologics, LLC.
Palm Harbor, Florida, United States
NOT_YET_RECRUITINGEyewell, LLC.
Boston, Massachusetts, United States
RECRUITINGBostonSight
Boston, Massachusetts, United States
RECRUITINGChange From Baseline to Day 90 in Modified Oxford Corneal Staining Score
Corneal fluorescein staining will be assessed in the prespecified study eye using the Modified Oxford grading scale. Scores range from 0 to 5, where 0 represents no corneal staining and higher scores represent progressively greater staining. A decrease from Baseline indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in Ocular Surface Disease Index (OSDI) Total Score
The Ocular Surface Disease Index (OSDI) will assess ocular symptoms and their effect on vision-related functioning. Total scores range from 0 to 100, with higher scores representing greater symptom burden. A decrease from Baseline indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥1-Grade Decrease in Modified Oxford Corneal Staining Score
The proportion of participants achieving at least a 1-grade decrease from Baseline to Day 90 in the prespecified study eye. Scores range from 0 to 5, with higher scores indicating greater corneal staining. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in Supportive Efficacy Measures in the Fellow Eye
Supportive analyses will evaluate change from Baseline to Day 90 separately for protocol-defined measures collected in the fellow eye, including the Modified Oxford Corneal Staining score, superficial punctate keratitis (SPK) severity, and tear break-up time (TBUT). Modified Oxford scores range from 0 to 5, with higher scores indicating greater corneal staining; a decrease indicates improvement. Lower SPK severity indicates improvement. TBUT is measured in seconds; an increase indicates improved tear-film stability. No composite score will be calculated.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With Modified Oxford Corneal Staining Score Grade 0 at Day 90
The proportion of participants with a Modified Oxford Corneal Staining Score of Grade 0 in the prespecified study eye at Day 90. Scores range from 0 to 5, with higher scores indicating greater corneal staining and a worse outcome. Grade 0 indicates no corneal staining.
Time frame: Day 90 (±5 days)
Change From Baseline to Day 90 in Superficial Punctate Keratitis (SPK) Severity
Change from Baseline to Day 90 in the protocol-defined Superficial Punctate Keratitis (SPK) Severity Grade in the prespecified study eye. Grades range from 0 (no SPK) to 4 (severe SPK), with higher grades indicating greater severity and a worse outcome. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥1-Grade Decrease in Superficial Punctate Keratitis (SPK) Severity
The proportion of participants achieving at least a 1-grade decrease from Baseline to Day 90 in the protocol-defined Superficial Punctate Keratitis (SPK) Severity Grade in the prespecified study eye. Grades range from 0 (no SPK) to 4 (severe SPK), with higher grades indicating greater severity and a worse outcome. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With Superficial Punctate Keratitis (SPK) Severity of 0 at Day 90
The proportion of participants with a Superficial Punctate Keratitis (SPK) Severity Grade of 0 in the prespecified study eye at Day 90. Grades range from 0 (no SPK) to 4 (severe SPK), with higher grades indicating greater severity and a worse outcome. Grade 0 indicates complete resolution of SPK.
Time frame: Day 90 (±5 days)
Percentage of Participants With a ≥13-Point Decrease in OSDI Total Score
The proportion of participants achieving a decrease of at least 13 points from Baseline to Day 90 in Ocular Surface Disease Index (OSDI) Total Score. Scores range from 0 to 100, with higher scores indicating greater symptom burden. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in NIH Eye Score
Change from Baseline to Day 90 in the National Institutes of Health (NIH) Eye Score. Scores range from 0 (no ocular symptoms) to 3 (severe ocular involvement), with higher scores indicating greater ocular graft-versus-host disease severity and a worse outcome. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥1-Grade Decrease in NIH Eye Score
The proportion of participants achieving at least a 1-grade decrease from Baseline to Day 90 in the National Institutes of Health (NIH) Eye Score. Scores range from 0 (no ocular symptoms) to 3 (severe ocular involvement), with higher scores indicating greater ocular graft-versus-host disease severity and a worse outcome. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants With No Worsening in NIH Eye Score at Day 90
The proportion of participants whose National Institutes of Health (NIH) Eye Score at Day 90 is equal to or lower than their Baseline score. Scores range from 0 (no ocular symptoms) to 3 (severe ocular involvement), with higher scores indicating greater ocular graft-versus-host disease severity and a worse outcome. A lower score indicates improvement, while an unchanged score indicates no worsening.
Time frame: Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in UNC Dry Eye Management Scale Visual Analog Scale Score
Change from Baseline to Day 90 in the University of North Carolina Dry Eye Management Scale (UNC DEMS) Visual Analog Scale score. Scores range from 1 to 10, with higher scores indicating more severe dry-eye symptoms, greater effects on daily life, and a worse outcome. A decrease indicates improvement.
Time frame: Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in Tear Break-Up Time (TBUT)
Change from Baseline to Day 90 in tear break-up time, measured in seconds, in the prespecified study eye. An increase indicates improvement in tear-film stability.
Time frame: Baseline to Day 90 (±5 days)
Percentage of Participants Receiving Protocol-Defined Rescue Therapy
The proportion of participants receiving at least one administration of protocol-defined rescue therapy during the randomized treatment period.
Time frame: Randomization through Day 90
Time to First Administration of Protocol-Defined Rescue Therapy
Time from randomization to the first administration of protocol-defined rescue therapy.
Time frame: Randomization through Day 90
Percentage of Participants With Protocol-Defined Treatment Failure
The proportion of participants experiencing treatment failure, defined as initiation of protocol-defined rescue therapy, permanent discontinuation of assigned study treatment because it is not providing adequate benefit, or withdrawal from all further study participation because of worsening ocular GVHD signs or symptoms.
Time frame: Randomization through Day 90
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.