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
• Genders Eligible for Study: All

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
Ocular pain from Glia OSD Symptoms Questionnaire	Change in ocular pain score	1 hour
Ocular discomfort	Change in ocular discomfort score	1 hour
Ocular discomfort	Change in ocular discomfort score	2 weeks
Corneal fluorescein staining	Change in fluorescein staining score	10 weeks
Conjunctival staining	Change in conjunctival staining score	10 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modified SANDE	Change in SANDE score	2 weeks
Modified SANDE	Change in SANDE score	6, 10 weeks
Modified SANDE	Change in SANDE score	10 weeks
Glia OSD Symptoms Questionnaire	Change in total score	1 hour
Glia OSD Symptoms Questionnaire	Change in total score	2 weeks
Glia OSD Symptoms Questionnaire	Change in total score	6 weeks
Glia OSD Symptoms Questionnaire	Change in total score	10 weeks
Tear Film	Change in tear film score	1 hour
Tear Film	Change in tear film score	2 weeks
Tear Film	Change in tear film score	6 weeks
Tear Film	Change in tear film score	10 weeks
Conjunctival staining	Change in conjunctival staining score	6
Lid margin	Change in lid margin abnormality score	6 weeks
Lid margin	Change in lid margin abnormality score	10 weeks
Ocular pain from Glia OSD Symptoms Questionnaire	Change in ocular pain score	2 weeks
Ocular pain from Glia OSD Symptoms Questionnaire	Change in ocular pain score	10 weeks
Ocular pain from Glia OSD Symptoms Questionnaire	Change in ocular pain score	6 weeks
Ocular discomfort	Change in ocular discomfort score	6 weeks
Ocular discomfort	Change in ocular discomfort score	10 weeks
Corneal fluorescein staining	Change in fluorescein staining score	6 weeks
NIH Chronic GvHD Eye Score	Change in eye score	2 weeks
NIH Chronic GvHD Eye Score	Change in eye score	6 weeks
NIH Chronic GvHD Eye Score	Change in eye score	10 weeks

Collaborators and Investigators

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

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): March 24, 2023
• Last Update Submitted That Met QC Criteria: March 23, 2023
• Last Verified: March 1, 2023

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 if Phase 3 study follows.

Drug and device information, study documents

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