Clinical trial NCT04792580: The Effects and Safety of 5% Lifitegrast Ophthalmic Solution in Subjects With Dry Eye Disease in Ocular Graft-versus-Host Disease
Official title: A Single-Center Randomized Double-Masked Placebo-Controlled Parallel-Group, Study of the Effects and Safety of 5% Lifitegrast Ophthalmic Solution in Subjects With Dry Eye Disease in Ocular Graft-versus-Host Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Graft-versus-host-disease
Ocular Graft-versus-host Disease
Keywords: Ocular surface
Bone marrow transplant
stem cell transplant
ocular Graft-versus-Host Disease
GVHD
Lead sponsor: Yee, Richard W., M.D.
Sponsor class: OTHER
Collaborators: name: Novartis Pharmaceuticals
First posted: 2021-03-11
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of this study is to evaluate the clinical efficacy of 5% lifitegrast ophthalmic solution in subjects with dry eye disease secondary to ocular Graft-versus-Host Disease compared to placebo.


Eligibility criteria: Inclusion Criteria:

* SANDE questionnaire \>40 mm
* Schirmer test without anesthesia \>2 mm and \<10mm across 5 minutes
* Tear film break-up time (TFBUT) \< 10 seconds in the worse eye
* The same eye (eligible eye) must fulfill all the above criteria
* Best corrected distance visual acuity (BCDVA) score of ≥ 0.1 decimal units (20/200 Snellen value) in both eyes at the time of study enrollment.
* If a female of childbearing potential, have a negative pregnancy test.
* Only patients who satisfy all Informed Consent requirements may be included in the study. The patient must read, sign and date the Informed Consent document before any study-related procedures are performed. The Informed Consent form signed by patients must have been approved by the Institutional Review Board (IRB) / Independent Ethics Committee (IEC) for the current study.
* Patients must have the ability and willingness to comply with study procedures.
* Patients must meet the internationally established criteria for a "probable" or "definite" diagnosis of oGVHD. As an inclusion criterion, a diagnosis of oGVHD is established from the Schirmer's 1 tear test, corneal fluorescein staining, OSDI scores, and conjunctival injection. Severity scores are assigned according to the panels provided by the International Chronic Ocular Graft-versus-Host Disease Consensus Group.
* Ability to speak and understand the English language sufficiently to understand the nature of the study, to provide written informed consent, and to allow the completion of all study assessments.

Exclusion Criteria:

* Evidence of an active ocular infection, in either eye
* Presence of any other ocular disorder or condition requiring topical medication during the entire duration of study
* History of severe systemic allergy or of ocular allergy (including seasonal conjunctivitis) or chronic conjunctivitis and/or keratitis other than dry eye
* Intraocular inflammation defined as Tyndall score \>0
* Systemic disease (excluding GVHD) not stabilized within 1 month before Screening Visit (e.g. diabetes with glycemia out of range, thyroid malfunction..) or judged by the investigator to be incompatible with the study (e.g. current systemic infections) or with a condition incompatible with the frequent assessment required by the study
* Patient had a serious adverse reaction or significant hypersensitivity to any drug or chemically-related compounds or had a clinically significant allergy to drugs, foods, amide local anesthetics or other materials including commercial artificial tears (in the opinion of the investigator)
* Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study if they meet any one of the following conditions:

  1. are currently pregnant or,
  2. have a positive result at the urine pregnancy test (Baseline/Day 0) or,
  3. intend to become pregnant during the study treatment period or,
  4. are breast-feeding or,
  5. are not willing to use highly effective birth control measures, such as: hormonal contraceptives - oral, implanted, transdermal, or injected - and/or mechanical barrier methods - spermicide in conjunction with a barrier such as a condom or diaphragm or Intra Uterine Device (IUD) - during the entire course of, and 30 days after, the study treatment periods
* Any concurrent medical condition, that in the judgment of the PI, might interfere with the conduct of the study, confound the interpretation of the study results, or endanger the patient's well-being
* Use of topical cyclosporine, topical corticosteroids or any other topical drug for the treatment of dry eye in either eye within 30 days prior to study enrollment.
* Contact lenses or punctum plug use at any time 30 days prior to or during the study
* History of drug addiction or alcohol abuse
* Any prior ocular surgery (including refractive palpebral and cataract surgery) if within 90 days before the screening visit
* Participation in a trial with a new active substance during the past 6 months
* Participation in another trial study at the same time as the present study.
* Previous use of lifitegrast, 5%

Primary outcomes:
measure: Change from baseline in Symptoms questionnaire (SANDE) scores to Week 4
timeFrame: Baseline to Week 4
Secondary outcomes:
measure: Change from baseline in Schirmer I test (without anesthesia) to Week 4
timeFrame: Baseline to Week 4
Other outcomes:
measure: Change from baseline in Symptoms questionnaire (SANDE) scores for frequency to Week 4
timeFrame: Baseline to Week 4
measure: Change from baseline in conjunctiva vital staining with lissamine green to Week 4
timeFrame: Baseline to Week 4
measure: Change from baseline in Tear Film Break-Up Time (TFBUT) to Week 4
timeFrame: Baseline to Week 4
measure: Change in OSDI score from baseline to Week 4
timeFrame: Baseline to Week 4
measure: Change in Corneal Staining Score from baseline to Week 4
timeFrame: Baseline to Week 4
Locations:
city: Bellaire
country: United States
facility: Richard W Yee, MD PLLC
state: Texas
Study officials:
References: