Trial Outcomes & Findings for Late Phase II Confirmatory Study of DE-089 Ophthalmic Solution in Patients With Dry Eye (NCT NCT01189032)

NCT ID: NCT01189032

Last Updated: 2014-08-13

Results Overview

Fluorescein staining was scored according to the protocol by Shimmura et al. The cornea was divided into 3 equal zones: upper, middle, and lower. Each zone had a staining score ranging between 0 and 3 points, with minimum and maximum total staining scores ranging between 0 and 9 points. 0 is better. The degree of staining with Fluorescein dyes was scored as follows: 0 = no staining, 1= staining of less than half of the area, 2= staining of more than half of the area, 3= staining in the whole area.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 320 participants
• Primary outcome timeframe: Baseline and 4-week (discontinued(LOCF))
• Results posted on: 2014-08-13

Participant Flow

Excluded (n=34)

Participant milestones

Measure	Placebo Placebo ophthalmic solution	Low Concentration 1% DE-089 ophthalmic solution	High Concentration 3% DE-089 ophthalmic solution
Overall Study STARTED	94	96	96
Overall Study COMPLETED	89	89	90
Overall Study NOT COMPLETED	5	7	6

Reasons for withdrawal

Measure	Placebo Placebo ophthalmic solution	Low Concentration 1% DE-089 ophthalmic solution	High Concentration 3% DE-089 ophthalmic solution
Overall Study Adverse Event	1	3	4
Overall Study Lack of Efficacy	2	3	0
Overall Study Withdrawal by Subject	1	0	1
Overall Study Protocol Violation	1	1	1

Baseline Characteristics

Late Phase II Confirmatory Study of DE-089 Ophthalmic Solution in Patients With Dry Eye

Baseline characteristics by cohort

Measure	Placebo n=94 Participants Placebo ophthalmic solution	Low Concentration n=96 Participants 1% DE-089 ophthalmic solution	High Concentration n=96 Participants 3% DE-089 ophthalmic solution	Total n=286 Participants Total of all reporting groups
Age, Continuous	56.1 years STANDARD_DEVIATION 17.3 • n=5 Participants	57.9 years STANDARD_DEVIATION 16.3 • n=7 Participants	56.4 years STANDARD_DEVIATION 17.8 • n=5 Participants	56.6 years STANDARD_DEVIATION 17.1 • n=4 Participants
Sex: Female, Male Female	76 Participants n=5 Participants	85 Participants n=7 Participants	77 Participants n=5 Participants	238 Participants n=4 Participants
Sex: Female, Male Male	18 Participants n=5 Participants	11 Participants n=7 Participants	19 Participants n=5 Participants	48 Participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline and 4-week (discontinued(LOCF))

Population: Efficacy analysis was performed per protocol set (PPS). Excluded cases were 7 subjects (3 subjects in 3% group, 3 subjects in 1% group, and 1 subject in Placebo group). The reason of exclusion: used the prohibited concomitant drug, dosing period shortage, number of doses non-compliance, or had no available efficacy data.

Fluorescein staining was scored according to the protocol by Shimmura et al. The cornea was divided into 3 equal zones: upper, middle, and lower. Each zone had a staining score ranging between 0 and 3 points, with minimum and maximum total staining scores ranging between 0 and 9 points. 0 is better.

The degree of staining with Fluorescein dyes was scored as follows: 0 = no staining, 1= staining of less than half of the area, 2= staining of more than half of the area, 3= staining in the whole area.

Outcome measures

Measure	Placebo n=93 Participants Placebo ophthalmic solution	Low Concentration n=93 Participants 1% DE-089 ophthalmic solution	High Concentration n=93 Participants 3% DE-089 ophthalmic solution
Mean Change in Fluorescein Staining Score From Baseline	-0.95 points Standard Deviation 1.33	-1.34 points Standard Deviation 1.43	-1.55 points Standard Deviation 1.30

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 17 other events

Deaths: 0 deaths

Low Concentration

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

High Concentration

Serious events: 1 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=94 participants at risk Placebo ophthalmic solution	Low Concentration n=96 participants at risk 1% DE-089 ophthalmic solution	High Concentration n=96 participants at risk 3% DE-089 ophthalmic solution
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lung adenocarcinoma	0.00% 0/94	0.00% 0/96	1.0% 1/96

Other adverse events

Measure	Placebo n=94 participants at risk Placebo ophthalmic solution	Low Concentration n=96 participants at risk 1% DE-089 ophthalmic solution	High Concentration n=96 participants at risk 3% DE-089 ophthalmic solution
Eye disorders Eye irritation	3.2% 3/94	7.3% 7/96	13.5% 13/96
Eye disorders Eye pain	4.3% 4/94	2.1% 2/96	6.2% 6/96
Infections and infestations Nasopharyngitis	10.6% 10/94	5.2% 5/96	9.4% 9/96

Additional Information

General Manager of Clinical Development Group

Santen Pharmaceutical Co., Ltd.

Phone: +81-6-4802-9341

Email: clinical@santen.co.jp

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place