Trial Outcomes & Findings for Trial of CF101 to Treat Patients With Dry Eye Disease (NCT NCT01235234)
NCT ID: NCT01235234
Last Updated: 2022-06-30
Results Overview
Complete clearing of corneal staining by fluorescein staining (FS). The primary efficacy analysis was performed for 1eye (target eye), defined as the eye with the larger corneal FS value at Baseline. If both eyes had the same corneal FS value at baseline, the target eye was considered the eye with the larger central corneal staining value at Baseline. Corneal FS defined as a corneal punctate fluorescein staining score of ≥4 in either eye by the National Eye Institute evaluation scale summed over 5 areas each with a 0-3 scoring scale
COMPLETED
PHASE3
236 participants
24 weeks
2022-06-30
Participant Flow
Participant milestones
| Measure |
CF101 0.1 mg
CF101: orally q12h
|
CF101 1 mg
CF101: orally q12h
|
Placebo
CF101: orally q12h
|
|---|---|---|---|
|
Overall Study
STARTED
|
77
|
78
|
81
|
|
Overall Study
COMPLETED
|
75
|
76
|
80
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
1
|
Reasons for withdrawal
| Measure |
CF101 0.1 mg
CF101: orally q12h
|
CF101 1 mg
CF101: orally q12h
|
Placebo
CF101: orally q12h
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
2
|
2
|
1
|
Baseline Characteristics
Trial of CF101 to Treat Patients With Dry Eye Disease
Baseline characteristics by cohort
| Measure |
CF101 0.1 mg
n=77 Participants
CF101: orally q12h
|
CF101 1 mg
n=78 Participants
CF101: orally q12h
|
Placebo
n=81 Participants
CF101: orally q12h
|
Total
n=236 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
57 Participants
n=5 Participants
|
56 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
172 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
20 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
64 Participants
n=4 Participants
|
|
Age, Continuous
|
56.8 years
n=5 Participants
|
54.5 years
n=7 Participants
|
55.1 years
n=5 Participants
|
55.5 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
55 Participants
n=5 Participants
|
62 Participants
n=7 Participants
|
71 Participants
n=5 Participants
|
188 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
48 Participants
n=4 Participants
|
|
Region of Enrollment
Israel
|
77 participants
n=5 Participants
|
78 participants
n=7 Participants
|
81 participants
n=5 Participants
|
236 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: The Number of Subjects Who Achieved Complete Clearing of Corneal Staining in Target Eye Using NRI at 24 weeks (ITT Population)
Complete clearing of corneal staining by fluorescein staining (FS). The primary efficacy analysis was performed for 1eye (target eye), defined as the eye with the larger corneal FS value at Baseline. If both eyes had the same corneal FS value at baseline, the target eye was considered the eye with the larger central corneal staining value at Baseline. Corneal FS defined as a corneal punctate fluorescein staining score of ≥4 in either eye by the National Eye Institute evaluation scale summed over 5 areas each with a 0-3 scoring scale
Outcome measures
| Measure |
CF101 0.1 mg
n=76 Participants
CF101: orally q12h
|
CF101 1 mg
n=77 Participants
CF101: orally q12h
|
Placebo
n=76 Participants
CF101: orally q12h
|
|---|---|---|---|
|
Number of Subjects Who Achieved Complete Clearing of Corneal Staining (i.e., Total Corneal FS Score = 0) at Week 24
Complete clearing of corneal staining in target eye using NRI - YES
|
15 participants
|
13 participants
|
21 participants
|
|
Number of Subjects Who Achieved Complete Clearing of Corneal Staining (i.e., Total Corneal FS Score = 0) at Week 24
Complete clearing of corneal staining in target eye using NRI - NO
|
61 participants
|
64 participants
|
55 participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Safety Population - Adverse Events
Determine the safety of oral CF 101 in this subject population. Adverse Events (AEs) and changes in vital signs, physical examination, clinical laboratory tests (liver, kidney, hematology, chemistry and urinalysis), electrocardiogram (ECG) findings, slit lamp and ophthalmic examination, visual acuity, and intraocular pressure measurements.
Outcome measures
| Measure |
CF101 0.1 mg
n=77 Participants
CF101: orally q12h
|
CF101 1 mg
n=78 Participants
CF101: orally q12h
|
Placebo
n=81 Participants
CF101: orally q12h
|
|---|---|---|---|
|
Number of Subjects With Adverse Events
Any AEs
|
29 Participants
|
37 Participants
|
29 Participants
|
|
Number of Subjects With Adverse Events
Blood and lymphatic system disorders
|
1 Participants
|
0 Participants
|
3 Participants
|
|
Number of Subjects With Adverse Events
Cardiac disorders
|
4 Participants
|
6 Participants
|
6 Participants
|
|
Number of Subjects With Adverse Events
Ear and labyrinth disorders
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Subjects With Adverse Events
Endocrine disorders
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Subjects With Adverse Events
Eye disorders
|
6 Participants
|
8 Participants
|
3 Participants
|
|
Number of Subjects With Adverse Events
Gastrointestinal disorders
|
3 Participants
|
5 Participants
|
4 Participants
|
|
Number of Subjects With Adverse Events
General disorders and administration site conditions
|
1 Participants
|
4 Participants
|
2 Participants
|
|
Number of Subjects With Adverse Events
Immune system disorders
|
1 Participants
|
0 Participants
|
2 Participants
|
|
Number of Subjects With Adverse Events
Infections and infestations
|
3 Participants
|
2 Participants
|
0 Participants
|
|
Number of Subjects With Adverse Events
Investigations
|
4 Participants
|
9 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events
Musculoskeletal and connective tissue disorders
|
2 Participants
|
5 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events
Nervous system disorders
|
4 Participants
|
6 Participants
|
4 Participants
|
|
Number of Subjects With Adverse Events
Psychiatric disorders
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Subjects With Adverse Events
Renal and urinary disorders
|
10 Participants
|
16 Participants
|
11 Participants
|
|
Number of Subjects With Adverse Events
Reproductive system and breast disorders
|
2 Participants
|
3 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events
Respiratory, thoracic and mediastinal disorders
|
2 Participants
|
1 Participants
|
7 Participants
|
|
Number of Subjects With Adverse Events
Skin and subcutaneous tissue disorders
|
2 Participants
|
5 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events
Vascular disorders
|
2 Participants
|
4 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: ITT Population
ST wetting increase over Baseline of ≥10 mm with or without anesthesia in either eye at Week 24, using a strip of filter paper placed inside the lower eyelid
Outcome measures
| Measure |
CF101 0.1 mg
n=76 Participants
CF101: orally q12h
|
CF101 1 mg
n=77 Participants
CF101: orally q12h
|
Placebo
n=76 Participants
CF101: orally q12h
|
|---|---|---|---|
|
Number of Subjects Who Achieve an Increase in Schirmer Test Tearing by >9mm Over Baseline
Number of subjects with ST wetting increase over baseline of >=10mm at 24 weeks
|
15 Participants
|
13 Participants
|
13 Participants
|
|
Number of Subjects Who Achieve an Increase in Schirmer Test Tearing by >9mm Over Baseline
Number of subjects without ST wetting increase over baseline of >=10mm at 24 weeks
|
61 Participants
|
64 Participants
|
63 Participants
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: ITT Population
The Ocular Surface Disease Index is assessed on a scale of 0 to 100, with higher scores representing greater disability. The subtotal score is based on 12 questions with answers ranging from 0 - "none the time," to 4 - "all of the time." The number of questions answered is totaled (0 - 12). The OSDI is calculated using the sum of subtotal scores (0-48) multiplied by 25, divided by number of questions answered (0-12). (i.e., if a participant answers all 12 questions with a severe outcome of 4, their respected OSDI = \[(4 x 12) x 25\] / 12 = \[48 x 25\] / 12 = 100) The outcome measured is the Mean Change from Baseline in Ocular Surface Disease Index at Week 24
Outcome measures
| Measure |
CF101 0.1 mg
n=76 Participants
CF101: orally q12h
|
CF101 1 mg
n=77 Participants
CF101: orally q12h
|
Placebo
n=76 Participants
CF101: orally q12h
|
|---|---|---|---|
|
Ocular Surface Disease Index
|
-24.27 Score on a Scale - OSDI
Standard Deviation 17.99
|
-25.63 Score on a Scale - OSDI
Standard Deviation 16.77
|
-28.70 Score on a Scale - OSDI
Standard Deviation 19.08
|
Adverse Events
CF101 0.1 mg
CF101 1 mg
Placebo
Serious adverse events
| Measure |
CF101 0.1 mg
n=77 participants at risk
CF101: orally q12h
|
CF101 1 mg
n=78 participants at risk
CF101: orally q12h
|
Placebo
n=81 participants at risk
CF101: orally q12h
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Nervous system disorders
Facial nerve disorder
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Nervous system disorders
Cerebral aneurysm ruptured syphilitic
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Reproductive system and breast disorders
Endometrial cancer
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Vascular disorders
Cerebrovascular accident
|
0.00%
0/77
|
0.00%
0/78
|
1.2%
1/81
|
|
Endocrine disorders
Type 2 diabetes mellitus
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Vascular disorders
Hypertension
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
|
Cardiac disorders
Angina unstable
|
1.3%
1/77
|
0.00%
0/78
|
0.00%
0/81
|
Other adverse events
| Measure |
CF101 0.1 mg
n=77 participants at risk
CF101: orally q12h
|
CF101 1 mg
n=78 participants at risk
CF101: orally q12h
|
Placebo
n=81 participants at risk
CF101: orally q12h
|
|---|---|---|---|
|
Renal and urinary disorders
Urinary tract infection
|
11.7%
9/77
|
16.7%
13/78
|
11.1%
9/81
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place