Treatment Safety and Efficacy of Pro-ocular™ 1% for Chronic Ocular Graft Following Allogeneic HSCT.

Treatment Safety and Efficacy Using Pro-ocular™ 1% for Chronic Ocular Graft-versus-host Disease (GvHD) Following Allogeneic Hematopoietic Stem Cell Transplantation.

To evaluate the safety and efficacy of Pro-ocular™ 1% topical gel administered twice daily for 70 days in reducing or eliminating symptoms and signs of chronic ocular GvHD.

Study Overview

• Status: Completed
• Conditions: Chronic Ocular Graft-versus-host Disease
• Intervention / Treatment: Drug: Pro-ocular™ topical gel; Drug: Placebo topical gel

Detailed Description

Ocular graft versus host disease presents a most severe form of ocular surface disease that compromises quality of life of afflicted allogeneic hematopoietic transplantation patients.

A controlled study is necessary to verify findings obtained to date in isolated patients. Currently available ophthalmic solutions and suspensions, drugs and devices largely have not been effective in alleviating the suffering of chronic ocular GvHD patients. Thus, this is an unmet need that Pro-ocular™ topical gel can fulfill.

Potential benefits: Decrease or cessation of adverse ocular symptoms and signs of chronic ocular GvHD, reduction or elimination of the need for other topical ocular therapeutic treatments for chronic ocular GvHD, and restoration of quality of life.

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Massachusetts Eye and Ear Longwood

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female of any race, at least 18 years of age at Visit 1 Screening.
2. Has the diagnosis of chronic ocular GvHD.
3. Has an NIH Consensus Eye Score of at least 2.
4. On the GLIA Ocular Surface Disease Symptoms Questionnaire at Screening, has ocular discomfort at a severity score of moderate or more, and at least one other symptom at a severity of moderate or more.
5. One or more signs from the list of chronic ocular GvHD signs below
6. Has provided verbal and written informed consent.
7. Be able and willing to follow oral and written instructions, including participation in all study assessments and visits.

Exclusion Criteria:

1. Wearing scleral or contact lenses within the last month, or those who plan to start wearing scleral or contact lenses during the study.
2. Anticipate major changes in systemic GvHD management during study period.
3. Comorbidity with other severe or chronic eye conditions that in the judgment of the investigator will interfere with study assessments, such as but not limited to retinal detachment, recent ocular surgery, Bell's palsy, active trigeminal neuritis or trigeminal neuralgia.
4. Anticipate change of vision correction or anticipate any ocular procedures during study period.
5. A woman who is pregnant, nursing an infant, or planning a pregnancy.
6. A woman of childbearing potential who has a positive urine pregnancy test at Visit 1, or who does not use an adequate method of birth control throughout the study period.
7. Has a known adverse reaction and/or sensitivity to the study drug or its components.
8. Unwilling to cease the use of sunscreen on the forehead or eye area.
9. Intraocular pressure >22 mm Hg at screening visit with or without ongoing glaucoma treatment.
10. Currently enrolled in an investigational drug or device study for chronic ocular GvHD.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pro-ocular™; Pro-ocular™ 1% topical gel applied dermally to forehead twice daily, morning and before bedtime.	Drug: Pro-ocular™ topical gel; Topical gel for forehead dermal application
Placebo Comparator: Placebo; Vehicle topical gel without active ingredient applied dermally to forehead twice daily, morning and before bedtime.	Drug: Placebo topical gel; Topical gel for forehead dermal application

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modified SANDE Questionnaire - Frequency, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated?	2 weeks
Corneal Fluorescein Stain - Central, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in central corneal region recorded on visual analog scale 0-10, higher values represent worse outcomes.	2 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modified SANDE Questionnaire - Frequency, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated?	6 weeks
Modified SANDE Questionnaire - Frequency, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated?	10 weeks
Corneal Fluorescein Stain - Central, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in central corneal region recorded on visual analog scale 0-10, higher values represent worse outcomes.	6 weeks
Corneal Fluorescein Stain - Central, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in central corneal region recorded on visual analog scale 0-10, higher values represent worse outcomes.	10 weeks
Modified SANDE Questionnaire - Global, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated? Global score is sqrt of Frequency x Severity.	2 weeks
Modified SANDE Questionnaire - Global, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated? Global score is sqrt of Frequency x Severity.	6 weeks
Modified SANDE Questionnaire - Global, Change From Baseline	Change in score from Baseline for worse eye. Score is 0-100 on a visual analog scale, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated? Global score is sqrt of Frequency x Severity.	10 weeks
Corneal Fluorescein Stain - Total, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in Total comprise Central, Inferior and Superior regions, sum score 0-30, higher values represent worse outcomes.	2 weeks
Corneal Fluorescein Stain - Total, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in Total comprise Central, Inferior and Superior regions, sum score 0-30, higher values represent worse outcomes.	6 weeks
Corneal Fluorescein Stain - Total, Change From Baseline	Change in score from Baseline for worse eye. Fluorescein staining score in Total comprise Central, Inferior and Superior regions, sum score 0-30, higher values represent worse outcomes.	10 weeks
Blurred Vision, Change From Baseline	Change in score from Baseline for worse eye. Score measured from visual analog scale 0-10, higher values represent worse outcomes. Blurred vision symptom is reported by participant.	2 weeks
Blurred Vision, Change From Baseline	Change in score from Baseline for worse eye. Score measured from visual analog scale 0-10, higher values represent worse outcomes. Blurred vision symptom is reported by participant.	6 weeks
Blurred Vision, Change From Baseline	Change in score from Baseline for worse eye. Score measured from visual analog scale 0-10, higher values represent worse outcomes. Blurred vision symptom is reported by participant.	10 weeks
Photophobia, Change From Baseline	Change in score from Baseline for worse eye. Score is recorded on a visual analog scale 0-10, higher values represent worse outcomes. Sensitivity to light symptom is reported by participant.	2 weeks
Photophobia, Change From Baseline	Change in score from Baseline for worse eye. Score is recorded on a visual analog scale 0-10, higher values represent worse outcomes. Sensitivity to light symptom is reported by participant.	6 weeks
Photophobia, Change From Baseline	Change in score from Baseline for worse eye. Score is recorded on a visual analog scale 0-10, higher values represent worse outcomes. Sensitivity to light symptom is reported by participant.	10 weeks
Lid Edema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid edema is viewed by slit lamp without stain.	2 weeks
Lid Edema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid edema is viewed by slit lamp without stain.	6 weeks
Lid Edema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid edema is viewed by slit lamp without stain.	10 weeks
Lid Erythema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is viewed by slit lamp without stain.	2 weeks
Lid Erythema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is viewed by slit lamp without stain.	6 weeks
Lid Erythema, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is viewed by slit lamp without stain.	10 weeks
Lid Margin Ulceration, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid margin ulceration is viewed by slit lamp without stain.	2 weeks
Lid Margin Ulceration, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid margin ulceration is viewed by slit lamp without stain.	6 weeks
Lid Margin Ulceration, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Lid margin ulceration is viewed by slit lamp without stain.	10 weeks
Conjunctival Hyperemia, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Conjunctival hyperemia is viewed by slit lamp without stain.	2 weeks
Conjunctival Hyperemia, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Conjunctival hyperemia is viewed by slit lamp without stain.	6 weeks
Conjunctival Hyperemia, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Conjunctival hyperemia is viewed by slit lamp without stain.	10 weeks
Dryness, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Dryness symptom is reported by participant.	2 weeks
Dryness, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Dryness symptom is reported by participant.	6 weeks
Dryness, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Dryness symptom is reported by participant.	10 weeks
Ocular Pain, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Ocular pain symptom is reported by participant.	2 weeks
Ocular Pain, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Ocular pain symptom is reported by participant.	6 weeks
Ocular Pain, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Ocular pain symptom is reported by participant.	10 weeks
Airflow Sensitivity, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Airflow sensitivity symptom is reported by participant.	2 weeks
Airflow Sensitivity, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Airflow sensitivity symptom is reported by participant.	6 weeks
Airflow Sensitivity, Change From Baseline	Change in score from Baseline for worse eye. Score on a visual analog scale 0-10, higher values represent worse outcomes. Airflow sensitivity symptom is reported by participant.	10 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modified SANDE Questionnaire - Frequency	Score on a visual analog scale 0-100, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated?	1 year
Modified SANDE Questionnaire - Frequency	Score on a visual analog scale 0-100, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. How often do eyes feel dry and/or irritated?	2 years
Modified SANDE Questionnaire - Global	Score on a visual analog scale 0-100, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. Global score is square root of how often do eyes feel dry and/or irritated x how severe are your symptoms of dryness and/or irritation?	1 year
Modified SANDE Questionnaire - Global	Score on a visual analog scale 0-100, higher values represent worse outcomes. Symptom Assessment iN Dry Eye Questionnaire. Global score is square root of how often do eyes feel dry and/or irritated x how severe are your symptoms of dryness and/or irritation?	2 years
Corneal Fluorescein Stain - Central	Fluorescein staining score in central corneal region recorded on visual analog scale 0-10, higher values represent worse outcomes.	1 year
Corneal Fluorescein Stain - Central	Fluorescein staining score in central corneal region recorded on visual analog scale 0-10, higher values represent worse outcomes.	2 years
Corneal Fluorescein Stain - Total	Fluorescein staining score Total for worse eye. Total comprise Central, Inferior and Superior regions, sum score 0-30, higher values represent worse outcomes.	1 year
Corneal Fluorescein Stain - Total	Fluorescein staining score Total for worse eye. Total comprises Central, Inferior and Superior regions, sum score 0-30, higher values represent worse outcomes.	2 years
Blurred Vision	Score for worse eye, recorded on a visual analog scale 0-10, higher values represent worse outcomes. Blurred vision symptom is reported by participant.	1 year
Blurred Vision	Score for worse eye, recorded on a visual analog scale 0-10, higher values represent worse outcomes. Blurred vision symptom is reported by participant.	2 years
Lid Edema	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid edema is scored by ophthalmologist using slit lamp biomicroscopy without staining.	1 year
Lid Edema	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid edema is scored by ophthalmologist using slit lamp biomicroscopy without staining.	2 years
Lid Erythema	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is scored by ophthalmologist using slit lamp biomicroscopy without staining.	1 year
Lid Erythema	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is scored by ophthalmologist using slit lamp biomicroscopy without staining.	2 years
Lid Ulceration	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid ulceration is scored by ophthalmologist using slit lamp biomicroscopy without staining.	1 year
Lid Ulceration	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Lid erythema is scored by ophthalmologist using slit lamp biomicroscopy without staining.	2 years
Conjunctival Hyperemia	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Conjunctival hyperemia is scored by ophthalmologist using slit lamp biomicroscopy without staining.	1 year
Conjunctival Hyperemia	Score for worse eye on a visual analog scale 0-10, higher values represent worse outcomes. Conjunctival hyperemia is scored by ophthalmologist using slit lamp biomicroscopy without staining.	2 years

Collaborators and Investigators

• Sponsor: Glia, LLC
• Collaborators: ORA, Inc.
• Investigators: Principal Investigator: Zhonghui K Luo, MD, PhD, Massachusetts Eye and Ear, Longwood

Publications and helpful links

General Publications

• Luo ZK, Domenech-Estarellas EA, Han A, Lee D, Khatri R, Wahl JL, Cutler C, Armand P, Antin JH, Koreth J, Gooptu M, Alyea EP, Soiffer RJ, Ho VT. Efficacy and Safety of 1% Progesterone Gel to the Forehead for Ocular Chronic Graft-versus-Host Disease. Transplant Cell Ther. 2021 May;27(5):433.e1-433.e8. doi: 10.1016/j.jtct.2021.02.008. Epub 2021 Feb 14.

Study record dates

Study Major Dates

• Study Start (Actual): October 17, 2019
• Primary Completion (Actual): October 31, 2021
• Study Completion (Actual): November 19, 2021

Study Registration Dates

• First Submitted: June 11, 2019
• First Submitted That Met QC Criteria: June 15, 2019
• First Posted (Actual): June 18, 2019

Study Record Updates

• Last Update Posted (Actual): May 6, 2025
• Last Update Submitted That Met QC Criteria: April 16, 2025
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: oGvHD-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Data may be shared with other investigators.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No