The Effects and Safety of 5% Lifitegrast Ophthalmic Solution in Subjects With Dry Eye Disease in Ocular Graft-versus-Host Disease

NCT04792580 | Completed Early Phase 1 FDA Drug

• 1 other identifier: CLIF606A1US09T
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• Change from baseline in Symptoms questionnaire (SANDE) scores to Week 4

  The SANDE score is calculated by taking the square root of the product of the severity of symptoms scores and the frequency of symptoms score. The SANDE scale ranges from 0 to 100 with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount or absence of dry eye symptoms.

  Baseline to Week 4

Secondary Outcomes (1)

• Change from baseline in Schirmer I test (without anesthesia) to Week 4

  Baseline to Week 4

Other Outcomes (5)

• Change from baseline in Symptoms questionnaire (SANDE) scores for frequency to Week 4

  Baseline to Week 4

• Change from baseline in conjunctiva vital staining with lissamine green to Week 4

  Baseline to Week 4

• Change from baseline in Tear Film Break-Up Time (TFBUT) to Week 4

  Baseline to Week 4

Study Arms (2)

Treatment

EXPERIMENTAL

Subjects receive lifitegrast 5% ophthalmic solution twice a day for 4 weeks after a 2 week washout.

Drug: Lifitegrast 5% Ophthalmic Solution

Placebo

PLACEBO COMPARATOR

Subjects receive the lifitegrast vehicle as placebo twice a day for 4 weeks after a 2 week washout.

Drug: Placebo

Interventions

Lifitegrast 5% Ophthalmic Solution DRUG

Used twice a day in both eyes for 4 weeks after a 2 week washout.

Treatment

Placebo DRUG

Used twice a day in both eyes for 4 weeks after a 2 week washout with the same drops.

Placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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:
• are currently pregnant or,
• have a positive result at the urine pregnancy test (Baseline/Day 0) or,
• intend to become pregnant during the study treatment period or,
• are breast-feeding or,
• 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

Sponsors & Collaborators

• Richard W Yee, MD: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Richard W Yee, MD PLLC

Bellaire, Texas, 77401, United States

Location

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

lifitegrast; Ophthalmic Solutions

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Pharmaceutical Solutions; Solutions; Pharmaceutical Preparations; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: March 8, 2021
• First Posted: March 11, 2021
• Study Start: October 22, 2022
• Primary Completion: April 30, 2024
• Study Completion: April 30, 2024
• Last Updated: July 15, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)