Phase II randomized double-masked crossover test of rhPRG4 (450 µg/mL) eye drops versus vehicle for ocular GVHD dry eye

A PHASE II PROSPECTIVE RANDOMIZED DOUBLE-MASKED CROSSOVER STUDY ASSESSING THE SAFETY & EFFICACY OF RHPRG4 (450 μG/ML RECOMBINANT HUMAN PROTEOGLYCAN 4) COMPARED TO VEHICLE FOR THE TREATMENT OF OCULAR GRAFT-VERSUS-HOST DISEASE (OGVHD)

This 84-day crossover study will try rhPRG4 eye drops versus a vehicle drop in adults with moderate to severe ocular graft‑versus‑host disease to see if eye symptoms and corneal staining get better.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Lubris Bio Pty Ltd (Industry-sponsored)
• Locations: 3 sites (Sydney, New South Wales and 2 other locations)
• Trial ID: NCT07118254 on ClinicalTrials.gov

What this trial studies

This is an 84-day, randomized (1:1), double-masked, crossover Phase II trial in adults with moderate to severe ocular graft‑versus‑host disease–related dry eye. Each participant receives both rhPRG4 (450 µg/mL) and matching vehicle eye drops for 28 days each, separated by a 14-day vehicle washout period, with treatment order randomized. Key entry requirements include symptom thresholds on VAS (dryness ≥40 mm and average symptom score ≥25 mm) and an Oxford corneal fluorescein staining score ≥2 in the worse eye. The trial measures safety and efficacy outcomes including symptom scores and corneal staining to compare rhPRG4 against vehicle within the same patients.

Who should consider this trial

Good fit: Adults aged 18–80 with a diagnosis of ocular GVHD for at least 3 months who are using artificial tears and meet the specified symptom (VAS) and corneal staining (Oxford ≥2) thresholds are the intended participants.

Not a fit: Patients with mild or very recent (<3 months) oGVHD, those who do not meet the symptom or corneal staining thresholds, or those unable to self-administer eye drops are unlikely to qualify or benefit from this protocol.

Why it matters

Potential benefit: If successful, rhPRG4 drops could reduce dry eye symptoms and corneal staining in patients with ocular GVHD, improving comfort and visual function.

How similar studies have performed: Preclinical data and small early clinical investigations of lubricin (rhPRG4) for dry eye show promise in improving lubrication and reducing corneal staining, but larger randomized crossover trials are limited.

Eligibility criteria

Inclusion Criteria:

1. Have the ability to comprehend and provide a signed and dated consent form.
2. Are 18-80 years of age at time of consent;
3. Have been diagnosed with oGVHD for at least 3 months prior to giving informed consent to participate in the trial;
4. Current use of artificial tears for the treatment of oGVHD related dry eye;
5. Have been stably using systemic medications for at least 14 days prior to Visit 1;
6. VAS Eye Dryness (100-point scale) score ≥ 40 mm;
7. Average VAS score for all symptoms of dry eye (dryness, foreign body sensation, burning/stinging, itching, pain, stick feeling, blurred vision and photophobia) ≥ 25 mm, none \< 5 mm;
8. Have Oxford corneal fluorescein staining grade of ≥ 2 using the Oxford scale in the worst performing eye
9. Stated willingness to comply with all study procedures, attend all scheduled clinic visits, and continue participation for the duration of the study;
10. Ability to self-administer study medication and willingness to adhere to the medication regimen.

Exclusion Criteria:

Are currently or have a history of any ocular or systemic disorder or condition other than dry eye that based on investigator judgment will interfere with the interpretation of the study results. Examples of ocular or systemic disorders or conditions include active ocular infection, conjunctivochalasis, superior limbic keratoconjunctivitis, limbal stem cell deficiency, allergic conjunctivitis, giant papillary conjunctivitis, atopic keratoconjunctivitis, anterior basement membrane dystrophies, neurotrophic keratitis, corneal dystrophy, exposure keratitis, moderate to severe blepharitis, ocular trauma, progressive or degenerative corneal conditions, uveitis, and systemic infection; 2. History of any ocular surgery (including laser or refractive surgical procedures) or therapeutic medical devices in either eye within 30 days before study enrollment. Therapeutic medical devices include trigeminal stimulation, meibomian glad warming (excepting at home masks) or expression, intense pulsed light, low level light therapy, etc. Ocular surgeries include laser or refractive surgical procedures, insertion of punctal or punctal cauterization; Ocular surgery will not be allowed during study participation; 3. Initiation of new therapeutic modalities within 14 days of Visit 1; 4. Have a known hypersensitivity to one of the components of the study or procedural medications; 5. Have participated in another clinical study at the same time as the present study or within 30 days of the Visit 1; 6. Have a history of drug, medication or alcohol abuse or addiction; 7. Are females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) who meet any one of the following conditions:

1. are currently pregnant or,
2. have a positive result on the urine pregnancy test at the Screening/Baseline Visit or,
3. intend to become pregnant during the entire course of and 30 days after the study treatment periods, or,
4. are breast-feeding or,
5. not willing to use highly effective birth control measures, such as: hormonal contraceptives - oral, implanted, transdermal, or injected and/or mechanical barrier methods, during the entire course of and 30 days after the study treatment periods; 8. Per the discretion of the investigator or designee, history of a serious physical or mental disorder that prevents the subject from attending study visits, complying with study-related procedures, and/or prevents the subject's ability to make decisions on their own; 9. Any other surgical or medical condition or finding that in the opinion of the investigator would compromise the subject's safety or participation in the study.

Where this trial is running

Sydney, New South Wales and 2 other locations

• Sydney Eye Hospital — Sydney, New South Wales, Australia (Not_yet_recruiting)
• Ota — Brisbane, Queensland, Australia (Recruiting)
• Royal Melbourne Hospital — Melbourne, Victoria, Australia (Recruiting)

Study contacts

• Study coordinator: Edward CEO

Conditions Ocular Graft Versus Host Disease; oGVHD; Dry Eye; Graft Versus Host Disease