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Phase 1 N=40 Randomized Single-blind Treatment

Safety and Efficacy of Topical Tacrolimus 0.05% in the Treatment of Ocular Graft-Versus-Host Disease

Ocular GVHD

Enrolled (actual)
40
Serious AEs
0.0%
Results posted
May 2017
Primary outcome: Primary: Ocular Burning Sensation, Ocular Discharge, Ocular Redness, Ocular Itching, Foreign Body Sensation — 3.50; 2.23; 0.16; 0.26 units on a scale — p=0.0019

Study Design & Population

Study type
Interventional
Phase
Phase 1
Interventions
Tacrolimus (Drug); Methylprednisolone Sodium Succinate (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Massachusetts Eye and Ear Infirmary
Primary completion
Sep 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
Ocular Burning Sensation, Ocular Discharge, Ocular Redness, Ocular Itching, Foreign Body Sensation
3.50; 2.23; 0.16; 0.26; 1.25; 0.43 0.0019 sig
SECONDARY
Ocular Surface Disease Index (OSDI) Questionnaire
42; 28
SECONDARY
Corneal Epitheliopathy (Corneal Fluorescein Staining Using the NEI Grading Scheme)
3.7; 6.6
SECONDARY
Schirmer Tear Test (mm)
3.5; 3.5
SECONDARY
Tear Film Break-Up Time
2.6; 1.0
SECONDARY
Visual Acuity
0.13; 0.13
SECONDARY
Intraocular Pressure
16.5; 17.1

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).

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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01977781). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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