Trial Outcomes & Findings for A Phase 3 Study to Evaluate the Efficacy of Lifitegrast in Subjects With Dry Eye (NCT NCT01743729)

NCT ID: NCT01743729

Last Updated: 2021-06-23

Results Overview

Corneal staining was performed to grade the degree of corneal epithelial cell injury as measured by fluorescence using slit-lamp examination. The corneal surface is divided into three regions: superior, central and inferior. The scores for each of these 3 regions ranged from 0 to 4 (0=no staining; 1=few/rare punctate lesions; 2=discrete and countable lesions; 3=lesions too numerous to count, but not coalescent; 4=coalescent) with 0.5 point increments, and lower scores indicate improvement. Inferior corneal fluorescein staining scores from the study eye only were reported. Study eye is the 'worse eye', defined as the eye with worse (higher) score at baseline.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

720 participants

Primary outcome timeframe

Baseline to Day 84

Results posted on

2021-06-23

Participant Flow

Two of 720 participants were excluded from data analysis due to duplication. Therefore, 718 participants were randomized and treated. One participant from placebo arm (N=360) has taken study drug by mistake and considered for "Lifitegrast" arm (N=358). Therefore, Placebo (N=359), Lifitegrast (N=359) were considered for safety analysis.

Participant milestones

Participant milestones
Measure
Lifitegrast
Placebo
Overall Study
STARTED
358
360
Overall Study
COMPLETED
321
348
Overall Study
NOT COMPLETED
37
12

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase 3 Study to Evaluate the Efficacy of Lifitegrast in Subjects With Dry Eye

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Lifitegrast
n=358 Participants
Placebo
n=360 Participants
Total
n=718 Participants
Total of all reporting groups
Age, Continuous
58.7 years
STANDARD_DEVIATION 13.93 • n=5 Participants
58.9 years
STANDARD_DEVIATION 14.26 • n=7 Participants
58.8 years
STANDARD_DEVIATION 14.09 • n=5 Participants
Sex: Female, Male
Female
285 Participants
n=5 Participants
265 Participants
n=7 Participants
550 Participants
n=5 Participants
Sex: Female, Male
Male
73 Participants
n=5 Participants
95 Participants
n=7 Participants
168 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Day 84

Population: Intent-to-treat (ITT) set included all randomized participants who received at least 1 dose of study drug.

Corneal staining was performed to grade the degree of corneal epithelial cell injury as measured by fluorescence using slit-lamp examination. The corneal surface is divided into three regions: superior, central and inferior. The scores for each of these 3 regions ranged from 0 to 4 (0=no staining; 1=few/rare punctate lesions; 2=discrete and countable lesions; 3=lesions too numerous to count, but not coalescent; 4=coalescent) with 0.5 point increments, and lower scores indicate improvement. Inferior corneal fluorescein staining scores from the study eye only were reported. Study eye is the 'worse eye', defined as the eye with worse (higher) score at baseline.

Outcome measures

Outcome measures
Measure
Lifitegrast
n=358 Participants
Placebo
n=360 Participants
Change From Baseline in Inferior Corneal Fluorescein Staining Score to Day 84
Baseline
2.39 units on a scale
Standard Deviation 0.763
2.40 units on a scale
Standard Deviation 0.722
Change From Baseline in Inferior Corneal Fluorescein Staining Score to Day 84
Change from Baseline to Day 84
-0.73 units on a scale
Standard Deviation 0.926
-0.71 units on a scale
Standard Deviation 0.943

PRIMARY outcome

Timeframe: Baseline to Day 84

Population: ITT population with last observation carried forward (LOCF).

Eye dryness score was assessed on a visual analogue scale (a 7-item \[burning/stinging, itching, foreign body sensation, eye discomfort, eye dryness, photophobia, and pain\], participant-reported, symptom index) with scores ranging from 0 to 100 (0=no discomfort; 100=maximal discomfort) and lower scores indicate a better outcome.

Outcome measures

Outcome measures
Measure
Lifitegrast
n=358 Participants
Placebo
n=360 Participants
Change From Baseline in Eye Dryness Score (Visual Analogue Scale) to Day 84
Baseline
69.68 units on a scale
Standard Deviation 16.954
69.22 units on a scale
Standard Deviation 16.761
Change From Baseline in Eye Dryness Score (Visual Analogue Scale) to Day 84
Change from Baseline to Day 84
-35.30 units on a scale
Standard Deviation 28.400
-22.75 units on a scale
Standard Deviation 28.600

Adverse Events

Lifitegrast

Serious events: 3 serious events
Other events: 102 other events
Deaths: 0 deaths

Placebo

Serious events: 4 serious events
Other events: 30 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Lifitegrast
n=359 participants at risk
Placebo
n=359 participants at risk
Ear and labyrinth disorders
Vertigo
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Endocrine disorders
Thyrotoxic crisis
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Gastrointestinal disorders
Colitis ischaemic
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Musculoskeletal and connective tissue disorders
Osteoarthritis
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder cancer
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal cancer
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Nervous system disorders
Cerebrovascular accident
0.00%
0/359
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.

Other adverse events

Other adverse events
Measure
Lifitegrast
n=359 participants at risk
Placebo
n=359 participants at risk
Eye disorders
Visual acuity reduced
5.0%
18/359 • Number of events 23
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
6.4%
23/359 • Number of events 28
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
General disorders
Instillation site irritation
7.8%
28/359 • Number of events 29
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
1.4%
5/359 • Number of events 5
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
General disorders
Instillation site reaction
7.0%
25/359 • Number of events 29
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
1.1%
4/359 • Number of events 4
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
Nervous system disorders
Dysgeusia
16.2%
58/359 • Number of events 58
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.
0.28%
1/359 • Number of events 1
One participant from placebo group (N=360) has taken study drug by mistake and considered for "Lifitegrast" group (N=358). Therefore, Placebo (N)= 359, Lifitegrast (N)=359 were considered for safety analysis.

Additional Information

Study Director

Shire (Note: Lifitegrast was divested to Novartis in 2019)

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee If a multicentre publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicentre Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
  • Publication restrictions are in place

Restriction type: OTHER