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

NCT03990051 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: oGvHD-1
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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.

Conditions

Chronic Ocular Graft-versus-host Disease

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (37)

• Modified SANDE Questionnaire - Frequency, Change From Baseline

  6 weeks

• Modified SANDE Questionnaire - Frequency, Change From Baseline

  10 weeks

• Corneal Fluorescein Stain - Central, Change From Baseline

  6 weeks

• Corneal Fluorescein Stain - Central, Change From Baseline

  10 weeks

• Modified SANDE Questionnaire - Global, Change From Baseline

  2 weeks

Other Outcomes (18)

• Modified SANDE Questionnaire - Frequency

  1 year

• Modified SANDE Questionnaire - Frequency

  2 years

• Modified SANDE Questionnaire - Global

  1 year

Study Arms (2)

Pro-ocular™

EXPERIMENTAL

Pro-ocular™ 1% topical gel applied dermally to forehead twice daily, morning and before bedtime.

Drug: Pro-ocular™ topical gel

Placebo

PLACEBO COMPARATOR

Vehicle topical gel without active ingredient applied dermally to forehead twice daily, morning and before bedtime.

Drug: Placebo topical gel

Interventions

Pro-ocular™ topical gel DRUG

Topical gel for forehead dermal application

Pro-ocular™

Placebo topical gel DRUG

Topical gel for forehead dermal application

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Glia, LLC: lead
• ORA, Inc.: collaborator

Study Sites (1)

Massachusetts Eye and Ear Longwood

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• 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.

  PMID: 33942724 RESULT

Results Point of Contact

• Title: Wei-wei Chang, PhD; President
• Organization: Glia, LLC

chang@gliacell.com

(617) 794-8922

Study Officials

• Zhonghui K Luo, MD, PhD

  Massachusetts Eye and Ear, Longwood

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Randomized double-masked parallel treatment assignment will be made using a block design at the ratio of 2:1, study drug:placebo, (22 subjects:11 subjects). This 70-day double-masked phase will be followed by 6 weeks open-label active drug treatment of the subjects previously on placebo.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 11, 2019
• First Posted: June 18, 2019
• Study Start: October 17, 2019
• Primary Completion: October 31, 2021
• Study Completion: November 19, 2021
• Last Updated: May 6, 2025
• Results First Posted: May 6, 2025

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)