Chronic Graft-versus-host Disease
Conditions
Keywords
Chronic Graft versus Host Disease, Keratoconjunctivitis Sicca
Brief summary
* To assess the efficacy of R348 Ophthalmic Solutions administered for 12 weeks to subjects evaluated by objective and subjective measures. * To investigate the safety and tolerability of R348 Ophthalmic Solutions administered for 12 weeks to subjects.
Interventions
R348 Ophthalmic Solution 0.2% 2 drops per eye twice a day
R348 Ophthalmic Solution, 0.5% 2 drops per eye twice a day
OTHERPlacebo Ophthalmic Solution
Placebo Ophthalmic Solution 2 drops per eye twice a day
Sponsors
Rigel Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Received an Allogeneic hematologic stem cell transplantation at least 3 months prior. * Subjects with post Allogeneic hematologic stem cell transplantation onset or worsening of dry eye symptoms for at least 1 month prior. * Use of over-the-counter and/or prescription eye drops for dry eye symptoms within 1 month. * Corneal fluorescein staining score of ≥ 2 in 1 region and ≥ 1 in at least 1 other region. * Total lissamine green conjunctival staining score (according to a modified National Eye Institute grading system) of ≥ 2.
Exclusion criteria
* Clinically unstable Graft versus Host Disease (requiring a change in immunosuppressive regimen), medical condition, or laboratory abnormality * Used topical ophthalmic cyclosporine within 45 days. * Used any topical ophthalmic steroid within 2 weeks. * Used autologous serum eye drops within 2 weeks.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline (Visit 1) of Total Corneal Fluorescein Staining Score at 12 Weeks. | Baseline to 12 weeks | Change from baseline (Visit 1) of total CFS score at 12 weeks. Total CFS score range 0-20, where '0' represents no fluorescein staining of any region and '20' represents severe staining the entire cornea. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| R348 Ophthalmic Solution, 0.2% R348 Ophthalmic Solution 0.2% 2 drops per eye twice a day
R348 Ophthalmic Solution, 0.2%: R348 Ophthalmic Solution 0.2% 2 drops per eye twice a day | 11 |
| R348 Ophthalmic Solution, 0.5% R348 Ophthalmic Solution, 0.5% 2 drops per eye twice a day
R348 Ophthalmic Solution, 0.5%: R348 Ophthalmic Solution, 0.5% 2 drops per eye twice a day | 12 |
| Placebo Placebo Ophthalmic Solution 2 drops per eye twice a day
Placebo Ophthalmic Solution: Placebo Ophthalmic Solution 2 drops per eye twice a day | 12 |
| Total | 35 |
Baseline characteristics
| Characteristic | R348 Ophthalmic Solution, 0.2% | R348 Ophthalmic Solution, 0.5% | Placebo | Total |
|---|---|---|---|---|
| Age, Continuous | 57.1 years STANDARD_DEVIATION 9.34 | 55.8 years STANDARD_DEVIATION 17.15 | 52.5 years STANDARD_DEVIATION 15.96 | 55.13 years STANDARD_DEVIATION 10.81 |
| Sex: Female, Male Female | 5 Participants | 5 Participants | 6 Participants | 16 Participants |
| Sex: Female, Male Male | 6 Participants | 7 Participants | 6 Participants | 19 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 11 / 11 | 10 / 12 | 9 / 12 |
| serious Total, serious adverse events | 1 / 11 | 2 / 12 | 1 / 12 |
Outcome results
Primary
Change From Baseline (Visit 1) of Total Corneal Fluorescein Staining Score at 12 Weeks.
Change from baseline (Visit 1) of total CFS score at 12 weeks. Total CFS score range 0-20, where '0' represents no fluorescein staining of any region and '20' represents severe staining the entire cornea.
Time frame: Baseline to 12 weeks
Population: The per-protocol (PP) population excludes subjects with significant protocol deviations or with early study termination
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| R348 Ophthalmic Solution, 0.2% | Change From Baseline (Visit 1) of Total Corneal Fluorescein Staining Score at 12 Weeks. | -4.2 units on a scale | Standard Deviation 2.9 |
| R348 Ophthalmic Solution, 0.5% | Change From Baseline (Visit 1) of Total Corneal Fluorescein Staining Score at 12 Weeks. | -6.2 units on a scale | Standard Deviation 3.7 |
| Placebo | Change From Baseline (Visit 1) of Total Corneal Fluorescein Staining Score at 12 Weeks. | -2.1 units on a scale | Standard Deviation 2.6 |