Trial Outcomes & Findings for Safety and Efficacy Study of CF101 to Treat Keratoconjunctivitis Sicca (NCT NCT00349466)

Results Overview

Involved placing a standardized paper tear strip inside the lower eyelid for 5 minutes. The tear strip was then removed and the length of the strip that was wet from tears was measured in millimeters. Change from baseline is calculated. Scores range 0-35 mm, with higher scores indicating more (better) tear production.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

80 participants

Primary outcome timeframe

12 weeks

Results posted on

2022-05-09

Participant Flow

Participant milestones

Participant milestones
Measure	CF101 1 mg CF101 1 mg q12 hours	Placebo Placebo tablets q12 hours for 12 weeks
Overall Study STARTED	42	38
Overall Study COMPLETED	33	35
Overall Study NOT COMPLETED	9	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Efficacy Study of CF101 to Treat Keratoconjunctivitis Sicca

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CF101 1 mg n=42 Participants CF101 1 mg q12 hours	Placebo n=38 Participants Placebo tablets q12 hours for 12 weeks	Total n=80 Participants Total of all reporting groups
Age, Continuous	56.2 Years STANDARD_DEVIATION 14.4 • n=5 Participants	61.4 Years STANDARD_DEVIATION 1311.7 • n=7 Participants	58.7 Years STANDARD_DEVIATION 13.4 • n=5 Participants
Sex: Female, Male Female	24 Participants n=5 Participants	28 Participants n=7 Participants	52 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants	10 Participants n=7 Participants	28 Participants n=5 Participants
Region of Enrollment Israel	42 participants n=5 Participants	38 participants n=7 Participants	80 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: ITT

Involved placing a standardized paper tear strip inside the lower eyelid for 5 minutes. The tear strip was then removed and the length of the strip that was wet from tears was measured in millimeters. Change from baseline is calculated. Scores range 0-35 mm, with higher scores indicating more (better) tear production.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Change From Baseline to Week 12 in Schirmer Test (ST) Score in Millimeters	1.25 Millimeters Standard Error 0.765	3.81 Millimeters Standard Error 0.814

PRIMARY outcome

Timeframe: 12 weeks

Population: ITT

Time elapsed between a complete blink and the development of the first random dry spot on the tear film as seen under fluorescent light. Change from baseline is calculated. Scores range 0-30 seconds, with higher scores indicating better tear composition and function.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Change From Baseline to Week 12 in Tear Break-Up Time (BUT) in Seconds	2.83 seconds Standard Error 0.906	0.48 seconds Standard Error 0.083

PRIMARY outcome

Timeframe: 12 weeks

Population: ITT

Severity of corneal epithelial loss as graded by Fluorescein Staining of the cornea, assessed on a 0-4+ scale with 0 = none and 4+ = severe de-epithelialization (in other words, higher scores indicate worse disease), expressed as number of participants with \>25% improvement at Week 12 relative to baseline.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Number of Subjects With >25% Improvement in Fluorescein Staining of the Cornea (FS) at Week 12	22 participants	12 participants

SECONDARY outcome

Timeframe: 12 weeks

Population: ITT

Proportion of subjects experiencing improvement of ≥25% over baseline at Week 12 in (a) tear breakup time (BUT), (b) superficial punctate keratitis as assessed by fluorescein staining (FS), or Schirmer Test (ST)

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Number of Subjects Experiencing Clinical Success at Week 12	24 Participants	20 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: ITT

DESS consists of 12 questions designed to assess the symptoms of ocular irritation, covering three areas: ocular symptoms, environmental triggers and vision-related function. Each of the 12 symptoms can be graded 0-4, with 0 indicating no symptoms over the past week, and 4 meaning constant symptoms over the past week. Scores from all answered questions are summed. The final score is multiplied by (25/number of questions answered) to give an outcome between 0 (no symptoms) to 100 (worst possible symptoms). Change from baseline is calculated.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Change From Baseline to 12 Weeks in Dry Eye Symptom Score (DESS)	34.44 score on a scale Standard Deviation 25.393	34.53 score on a scale Standard Deviation 20.605

SECONDARY outcome

Timeframe: 12 weeks

Population: ITT

Indicator of tear volume. TM was recorded on a scale from 0-3, with 0=none, 1=trace, 2=normal, and 3=high, with higher scores meaning better tear production and integrity. Change from baseline.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Change From Baseline to Week 12 in Tear Meniscus (TM) Height	0.38 units on a scale Standard Deviation 0.637	0.13 units on a scale Standard Deviation 0.694

SECONDARY outcome

Timeframe: 12 weeks

Population: ITT

Daily use of REFRESH TEARS® Lubricant Eye Drops artificial tears supplied to each patient was recorded in diaries provided to patients. Percent change from baseline is calculated.

Outcome measures

Outcome measures
Measure	CF101 1 mg n=26 Participants Oral tablets given every 12 hours for 12 weeks	Placebo n=23 Participants Oral tablets given every 12 hours for 12 weeks
Percent Change From Baseline to Week 12 in the Use of Artificial Tears	355625.0 percentage change from baseline Standard Error 410162.00	187883.1 percentage change from baseline Standard Error 477720.08

Adverse Events

CF101 1 mg

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	CF101 1 mg n=39 participants at risk CF101 1 mg q12 hours	Placebo n=37 participants at risk Placebo tablets q12 hours for 12 weeks
Gastrointestinal disorders Constipation	7.7% 3/39 • Number of events 3 • Starting on week 2 till week 12 and continue till follow up visit on week 14	5.4% 2/37 • Number of events 2 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Nervous system disorders Headache	7.7% 3/39 • Number of events 3 • Starting on week 2 till week 12 and continue till follow up visit on week 14	10.8% 4/37 • Number of events 4 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Cardiac disorders Palpitations	7.7% 3/39 • Number of events 3 • Starting on week 2 till week 12 and continue till follow up visit on week 14	0.00% 0/37 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Skin and subcutaneous tissue disorders Itching	7.7% 3/39 • Number of events 3 • Starting on week 2 till week 12 and continue till follow up visit on week 14	2.7% 1/37 • Number of events 1 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Gastrointestinal disorders Abdominal pain	5.1% 2/39 • Number of events 2 • Starting on week 2 till week 12 and continue till follow up visit on week 14	0.00% 0/37 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Musculoskeletal and connective tissue disorders Arthralgia	5.1% 2/39 • Number of events 2 • Starting on week 2 till week 12 and continue till follow up visit on week 14	2.7% 1/37 • Number of events 1 • Starting on week 2 till week 12 and continue till follow up visit on week 14
Musculoskeletal and connective tissue disorders Myalgia	5.1% 2/39 • Number of events 2 • Starting on week 2 till week 12 and continue till follow up visit on week 14	0.00% 0/37 • Starting on week 2 till week 12 and continue till follow up visit on week 14
General disorders Fatigue	5.1% 2/39 • Number of events 2 • Starting on week 2 till week 12 and continue till follow up visit on week 14	0.00% 0/37 • Starting on week 2 till week 12 and continue till follow up visit on week 14

Additional Information

Pnina Fishman, PhD

Can-Fite Biopharma

Phone: 011972 3 924 1114

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place