Clinical trial NCT01977781: Safety and Efficacy of Topical Tacrolimus 0.05% in the Treatment of Ocular Graft-Versus-Host Disease
Official title: Safety and Efficacy of Topical Tacrolimus 0.05% in the Treatment of Ocular Graft-Versus-Host Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Ocular GVHD
Keywords: GVHD
Ocular GVHD
Tacrolimus
Methylprednisolone
Lead sponsor: Massachusetts Eye and Ear Infirmary
Sponsor class: OTHER
First posted: 2013-11-07
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and tolerability of topical tacrolimus 0.05% twice a day in the treatment of ocular graft versus host disease (GVHD).

Detailed description: The objective of this study is to compare the safety and efficacy of topical tacrolimus 0.05% drops compared with topical methylprednisolone sodium succinate 0.5% drops in patients with ocular GVHD. 40 patients with ocular GVHD who meet the inclusion criteria of the study as determined by a screening visit will be divided into two equal groups. In addition to their current medication, one group will receive the topical tacrolimus 0.05% drops and the other group will receive the topical methylprednisolone sodium succinate 0.5% drops. The participants will then have two follow-up visits at the week 5 and week 10 markers after their screening visit.

To evaluate the purpose of the study as well as the patient's safety, the following procedures will be performed at each visit: a comprehensive eye examination, tear break-up time (TBUT), Schirmer's test, intraocular pressure (IOP), fundoscopy and grading scores of lid margin and corneal fluorescein staining. The following questionnaires will be administered at each visit: Ocular Surface Disease Index (OSDI) and Symptom Assessment in Dry Eye (SANDE). Impression cytology specimens will be taken only at the screening visit and at the week 10 visit.

Eligibility criteria: Inclusion Criteria:

* Age ≥18 years.
* Willing and able to provide written informed consent.
* Willing and able to comply with study assessments for the full duration of the study.
* Diagnosis of ocular GVHD.
* Minimum corneal fluorescein staining of 4 (NEI grading scheme, 0-15) in at least one eye.
* Ocular Surface Disease Index score \>22.
* In good stable overall health.

Exclusion Criteria:

* History of immune disease other than GVHD.
* Ocular or periocular malignancy.
* Significant change, as judged by the principal investigator, in systemic immunosuppressive regimen within 2 weeks of study entry.
* Any history of topical tacrolimus use.
* Any change in dosage of tetracycline compounds (tetracycline, doxycycline, and minocycline) within the last month.
* Any change in frequency of preserved anti-glaucoma medications within 2 weeks of study entry.
* Current use of topical steroids more than twice a day.
* Change in frequency of serum tears, topical cyclosporine and/or topical kineret within the last month.
* Corneal epithelial defect \>1mm2.
* Any history of herpetic keratitis.
* Participation in another simultaneous medical research study.
* Signs of current infection, including fever and current treatment with antibiotics.
* Intra-ocular surgery or ocular laser surgery within 3 months.
* Pregnancy (positive pregnancy test) or lactating
* Has worn contact lenses, except for bandage contact lens or rigid gas permeable lens, for the last 2 weeks prior to the study or would be unable to stay off contact lenses for the study duration.
* Any condition (including language barrier) that precludes patient's ability to comply with study requirements including completion of study.

Primary outcomes:
measure: Ocular Burning Sensation, Ocular Discharge, Ocular Redness, Ocular Itching, Foreign Body Sensation
timeFrame: 10 weeks
Secondary outcomes:
measure: Ocular Surface Disease Index (OSDI) Questionnaire
timeFrame: 10 weeks
measure: Corneal Epitheliopathy (Corneal Fluorescein Staining Using the NEI Grading Scheme)
timeFrame: 10 weeks
measure: Schirmer Tear Test (mm)
timeFrame: 10 weeks
measure: Tear Film Break-Up Time
timeFrame: 10 weeks
measure: Visual Acuity
timeFrame: 10 weeks
measure: Intraocular Pressure
timeFrame: 10 weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts Eye and Ear Infirmary
state: Massachusetts
Study officials:
affiliation: Massachusetts Eye and Ear Infirmary
name: Reza Dana, MD, MPH, MSc
role: PRINCIPAL_INVESTIGATOR
References: