Clinical trial NCT03990051: Treatment Safety and Efficacy of Pro-ocular™ 1% for Chronic Ocular Graft Following Allogeneic HSCT.
Official title: Treatment Safety and Efficacy Using Pro-ocular™ 1% for Chronic Ocular Graft-versus-host Disease (GvHD) Following Allogeneic Hematopoietic Stem Cell Transplantation.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Chronic Ocular Graft-versus-host Disease
Lead sponsor: Glia, LLC
Sponsor class: INDUSTRY
Collaborators: name: ORA, Inc.
First posted: 2019-06-18
Enrollment: 33
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The double-masked phase involves placebo controlled administration of study drug for 70 days or 10 weeks. Subsequently the placebo group will receive the active study drug of 42 days or 6 weeks. Long term open label phase to 27 months.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Modified SANDE Questionnaire - Frequency, Change From Baseline
timeFrame: 2 weeks
measure: Corneal Fluorescein Stain - Central, Change From Baseline
timeFrame: 2 weeks
Secondary outcomes:
measure: Modified SANDE Questionnaire - Frequency, Change From Baseline
timeFrame: 6 weeks
measure: Modified SANDE Questionnaire - Frequency, Change From Baseline
timeFrame: 10 weeks
measure: Corneal Fluorescein Stain - Central, Change From Baseline
timeFrame: 6 weeks
measure: Corneal Fluorescein Stain - Central, Change From Baseline
timeFrame: 10 weeks
measure: Modified SANDE Questionnaire - Global, Change From Baseline
timeFrame: 2 weeks
measure: Modified SANDE Questionnaire - Global, Change From Baseline
timeFrame: 6 weeks
measure: Modified SANDE Questionnaire - Global, Change From Baseline
timeFrame: 10 weeks
measure: Corneal Fluorescein Stain - Total, Change From Baseline
timeFrame: 2 weeks
measure: Corneal Fluorescein Stain - Total, Change From Baseline
timeFrame: 6 weeks
measure: Corneal Fluorescein Stain - Total, Change From Baseline
timeFrame: 10 weeks
measure: Blurred Vision, Change From Baseline
timeFrame: 2 weeks
measure: Blurred Vision, Change From Baseline
timeFrame: 6 weeks
measure: Blurred Vision, Change From Baseline
timeFrame: 10 weeks
measure: Photophobia, Change From Baseline
timeFrame: 2 weeks
measure: Photophobia, Change From Baseline
timeFrame: 6 weeks
measure: Photophobia, Change From Baseline
timeFrame: 10 weeks
measure: Lid Edema, Change From Baseline
timeFrame: 2 weeks
measure: Lid Edema, Change From Baseline
timeFrame: 6 weeks
measure: Lid Edema, Change From Baseline
timeFrame: 10 weeks
measure: Lid Erythema, Change From Baseline
timeFrame: 2 weeks
measure: Lid Erythema, Change From Baseline
timeFrame: 6 weeks
measure: Lid Erythema, Change From Baseline
timeFrame: 10 weeks
measure: Lid Margin Ulceration, Change From Baseline
timeFrame: 2 weeks
measure: Lid Margin Ulceration, Change From Baseline
timeFrame: 6 weeks
measure: Lid Margin Ulceration, Change From Baseline
timeFrame: 10 weeks
measure: Conjunctival Hyperemia, Change From Baseline
timeFrame: 2 weeks
measure: Conjunctival Hyperemia, Change From Baseline
timeFrame: 6 weeks
measure: Conjunctival Hyperemia, Change From Baseline
timeFrame: 10 weeks
measure: Dryness, Change From Baseline
timeFrame: 2 weeks
measure: Dryness, Change From Baseline
timeFrame: 6 weeks
measure: Dryness, Change From Baseline
timeFrame: 10 weeks
measure: Ocular Pain, Change From Baseline
timeFrame: 2 weeks
measure: Ocular Pain, Change From Baseline
timeFrame: 6 weeks
measure: Ocular Pain, Change From Baseline
timeFrame: 10 weeks
measure: Airflow Sensitivity, Change From Baseline
timeFrame: 2 weeks
measure: Airflow Sensitivity, Change From Baseline
timeFrame: 6 weeks
measure: Airflow Sensitivity, Change From Baseline
timeFrame: 10 weeks
Other outcomes:
measure: Modified SANDE Questionnaire - Frequency
timeFrame: 1 year
measure: Modified SANDE Questionnaire - Frequency
timeFrame: 2 years
measure: Modified SANDE Questionnaire - Global
timeFrame: 1 year
measure: Modified SANDE Questionnaire - Global
timeFrame: 2 years
measure: Corneal Fluorescein Stain - Central
timeFrame: 1 year
measure: Corneal Fluorescein Stain - Central
timeFrame: 2 years
measure: Corneal Fluorescein Stain - Total
timeFrame: 1 year
measure: Corneal Fluorescein Stain - Total
timeFrame: 2 years
measure: Blurred Vision
timeFrame: 1 year
measure: Blurred Vision
timeFrame: 2 years
measure: Lid Edema
timeFrame: 1 year
measure: Lid Edema
timeFrame: 2 years
measure: Lid Erythema
timeFrame: 1 year
measure: Lid Erythema
timeFrame: 2 years
measure: Lid Ulceration
timeFrame: 1 year
measure: Lid Ulceration
timeFrame: 2 years
measure: Conjunctival Hyperemia
timeFrame: 1 year
measure: Conjunctival Hyperemia
timeFrame: 2 years
Locations:
city: Boston
country: United States
facility: Massachusetts Eye and Ear Longwood
state: Massachusetts
Study officials:
affiliation: Massachusetts Eye and Ear, Longwood
name: Zhonghui K Luo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.