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

Phase 2

Completed

Conditions: Chronic Ocular Graft-versus-host Disease

Interventions: Drug: Pro-ocular™ topical gel
Drug: Placebo topical gel

Registration Number: NCT03990051

Lead Sponsor: Glia, LLC

Brief Summary: 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.

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 33

Inclusion Criteria

Male or female of any race, at least 18 years of age at Visit 1 Screening.
Has the diagnosis of chronic ocular GvHD.
Has an NIH Consensus Eye Score of at least 2.
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.
One or more signs from the list of chronic ocular GvHD signs below
Has provided verbal and written informed consent.
Be able and willing to follow oral and written instructions, including participation in all study assessments and visits.

Exclusion Criteria

Wearing scleral or contact lenses within the last month, or those who plan to start wearing scleral or contact lenses during the study.
Anticipate major changes in systemic GvHD management during study period.
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.
Anticipate change of vision correction or anticipate any ocular procedures during study period.
A woman who is pregnant, nursing an infant, or planning a pregnancy.
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.
Has a known adverse reaction and/or sensitivity to the study drug or its components.
Unwilling to cease the use of sunscreen on the forehead or eye area.
Intraocular pressure >22 mm Hg at screening visit with or without ongoing glaucoma treatment.
Currently enrolled in an investigational drug or device study for chronic ocular GvHD.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pro-ocular™	Pro-ocular™ topical gel	Pro-ocular™ 1% topical gel applied dermally to forehead twice daily, morning and before bedtime.
Placebo	Placebo topical gel	Vehicle topical gel without active ingredient applied dermally to forehead twice daily, morning and before bedtime.

Primary Outcome Measures

Name	Time	Method
Modified SANDE Questionnaire - Frequency, Change From Baseline	2 weeks	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?
Corneal Fluorescein Stain - Central, Change From Baseline	2 weeks	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.

Secondary Outcome Measures

Name	Time	Method
Modified SANDE Questionnaire - Frequency, Change From Baseline	10 weeks	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?
Corneal Fluorescein Stain - Central, Change From Baseline	10 weeks	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.
Modified SANDE Questionnaire - Global, Change From Baseline	10 weeks	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.
Corneal Fluorescein Stain - Total, Change From Baseline	10 weeks	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.
Blurred Vision, Change From Baseline	10 weeks	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.
Photophobia, Change From Baseline	10 weeks	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.
Lid Edema, Change From Baseline	10 weeks	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.
Lid Erythema, Change From Baseline	10 weeks	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.
Lid Margin Ulceration, Change From Baseline	10 weeks	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.
Conjunctival Hyperemia, Change From Baseline	10 weeks	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.
Dryness, Change From Baseline	10 weeks	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.
Ocular Pain, Change From Baseline	10 weeks	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.
Airflow Sensitivity, Change From Baseline	10 weeks	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.