Study of d-MAPPS™ Ophthalmic Solution in Adults With Chronic Ocular Graft-Versus-Host Disease (oGVHD)

September 3, 2026 updated by: Regenerative Ocular Immunobiologics LLC

A Randomized, Double-Masked, Vehicle-Controlled, Adequate and Well-Controlled Pivotal Study With Co-Primary Endpoints to Evaluate the Safety and Efficacy of d-MAPPS™ Ophthalmic Solution in Subjects With Chronic Ocular Graft-Versus-Host Disease (oGVHD)

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 Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Beverly Hills, California, United States, 90210
        • Recruiting
        • Beverly Hills Institute of Ophthalmology
        • Contact:
        • Principal Investigator:
          • Jillian Chong, MD
      • Beverly Hills, California, United States, 90211
        • Recruiting
        • Beverly Hills Optometry
        • Contact:
          • Kambiz Silani, OD
          • Phone Number: 310-659-2020
        • Contact:
        • Principal Investigator:
          • Kambiz Silani, OD
    • Florida
      • Palm Harbor, Florida, United States, 34684
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Eyewell, LLC.
        • Contact:
        • Principal Investigator:
          • Kristen Brown, OD
      • Boston, Massachusetts, United States, 02494
        • Recruiting
        • BostonSight
        • Contact:
        • Principal Investigator:
          • Daniel C. Brocks, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age 18 years or older.
  • Documented chronic ocular graft-versus-host disease (oGVHD) following allogeneic hematopoietic stem cell transplantation.
  • Investigator determines the participant is medically stable and appropriate for study participation.
  • Ocular Surface Disease Index (OSDI) Total Score of at least 33 at Baseline.
  • UNC Dry Eye Management Scale Visual Analog Scale (UNC DEMS VAS) score of at least 3 at Baseline.
  • Modified Oxford Corneal Staining Score of at least 1 in at least one eye.
  • Snellen Corrected Distance Visual Acuity sufficient to complete protocol-defined assessments.
  • Written informed consent obtained before study procedures.
  • Willing and able to comply with study visits, study treatment, and protocol requirements.
  • Background therapies stable for at least 30 days before Baseline.

Exclusion Criteria:

  • Acute ocular graft-versus-host disease or ocular disease that may interfere with efficacy assessment.
  • Active ocular or periocular infection, ocular malignancy, or active herpetic keratitis.
  • Intraocular surgery, refractive surgery, or ocular laser procedure within 6 weeks before Baseline or planned during the study.
  • Change in systemic immunosuppressive therapy, topical glaucoma therapy, or oral tetracycline therapy within 30 days before Baseline.
  • Initiation within 30 days before Baseline or during the trial of topical cyclosporine, lifitegrast, tacrolimus, autologous serum eye drops, platelet-rich plasma eye drops, amniotic membrane products, or another prescription therapy intended to treat ocular surface disease.
  • Participation in another investigational study within 30 days before Screening or concurrent enrollment.
  • Pregnancy or breastfeeding.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Vehicle Controlled
Biological: Matching vehicle control
Matching vehicle control supplied in identical masked containers and administered as two drops into each eye four times daily for 90 consecutive days.
Experimental: Active Study Drug
Biological: d-MAPPS™ Ophthalmic Solution
Sterile, preservative-free ophthalmic solution administered as two drops into each eye four times daily for 90 consecutive days.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to Day 90 in Modified Oxford Corneal Staining Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in Ocular Surface Disease Index (OSDI) Total Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants With a ≥1-Grade Decrease in Modified Oxford Corneal Staining Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥13-Point Decrease in OSDI Total Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in Tear Break-Up Time (TBUT)
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants Receiving Protocol-Defined Rescue Therapy
Time Frame: Randomization through Day 90
The proportion of participants receiving at least one administration of protocol-defined rescue therapy during the randomized treatment period.
Randomization through Day 90
Time to First Administration of Protocol-Defined Rescue Therapy
Time Frame: Randomization through Day 90
Time from randomization to the first administration of protocol-defined rescue therapy.
Randomization through Day 90
Percentage of Participants With Protocol-Defined Treatment Failure
Time Frame: Randomization through Day 90
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.
Randomization through Day 90
Change From Baseline to Day 90 in Supportive Efficacy Measures in the Fellow Eye
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With Modified Oxford Corneal Staining Score Grade 0 at Day 90
Time Frame: Day 90 (±5 days)
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.
Day 90 (±5 days)
Change From Baseline to Day 90 in Superficial Punctate Keratitis (SPK) Severity
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥1-Grade Decrease in Superficial Punctate Keratitis (SPK) Severity
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With Superficial Punctate Keratitis (SPK) Severity of 0 at Day 90
Time Frame: Day 90 (±5 days)
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.
Day 90 (±5 days)
Change From Baseline to Day 90 in NIH Eye Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With a ≥1-Grade Decrease in NIH Eye Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Percentage of Participants With No Worsening in NIH Eye Score at Day 90
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)
Change From Baseline to Day 90 in UNC Dry Eye Management Scale Visual Analog Scale Score
Time Frame: Baseline to Day 90 (±5 days)
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.
Baseline to Day 90 (±5 days)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regenerative Ocular Immunobiologics LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

September 1, 2026

Primary Completion (Estimated)

March 31, 2027

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

August 17, 2026

First Submitted That Met QC Criteria

August 17, 2026

First Posted (Actual)

August 20, 2026

Study Record Updates

Last Update Posted (Actual)

September 4, 2026

Last Update Submitted That Met QC Criteria

September 3, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • IND 30066-PIVOT

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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