Safety and Efficacy Study of CF101 to Treat Keratoconjunctivitis Sicca
Condition: Keratoconjunctivitis Sicca
MeSH terms: Keratoconjunctivitis; Keratoconjunctivitis Sicca; Dry Eye Syndromes
Intervention: CF101; Placebo
Intervention description: Orally CF101 1mg; Orally matching Placebo
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 80.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Keratoconjunctivitis Sicca; KCS; Dry Eye

Summary:

      This is a Phase 2, randomized, double-masked, placebo-controlled, parallel-group study in
      adult males and females, aged 18 years and over, with a diagnosis of moderate-to-severe
      keratoconjunctivitis sicca (KCS). Patients will be randomized to receive either CF101 1 mg or
      matching placebo, given orally every 12 hours (q12h) for 12 weeks. A Screening Period of up
      to 4 weeks that includes a 2-week run-in period will precede a 12-week treatment period,
      followed by a 2-week follow-up period.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female, 18 years of age and over;

          -  Have a diagnosis of moderate-to-severe KCS as defined by: (1) Schirmer Test (ST)
             (without anesthesia) < 7 mm/5 min in either eye; AND (2) Positive FS, defined as a
             corneal punctate fluorescein staining score of ≥1 in either eye, where 0 = none and 3=
             severe; AND (3) At least 1 of the following ocular symptoms scored at ≥2, where 0 =
             none and 4 = very severe/interferes with normal activities: photophobia, blurred
             vision, foreign body sensation, soreness or pain, itching, burning, dryness;

          -  Willing to use no topical ocular treatments except for the unpreserved artificial
             tears;

          -  Doses of unpreserved artificial tears have been stable for >2 weeks prior to Screening
             Visit;

          -  Females of child-bearing potential must have a negative urine pregnancy test at
             screening and throughout the study, to be eligible for, and continue participation in,
             the study;

          -  Females of child-bearing potential must be willing to use 2 methods of contraception
             deemed adequate by the Investigator (for example oral contraceptive pills plus a
             barrier method) to be eligible for, and continue participation in, the study;

          -  Ability to complete the study in compliance with the protocol; and

          -  Ability to understand and provide written informed consent.

        Exclusion Criteria:

          -  Has Sjögren's Syndrome with significant systemic non-exocrine gland involvement;

          -  Has Stevens-Johnson Syndrome;

          -  If KCS is due to rheumatoid arthritis or other autoimmune diseases, patient may not be
             receiving disease-modifying drugs, including methotrexate and biological agents;

          -  Use of systemic immunosuppressive drugs;

          -  Use of oral corticosteroids >10 mg prednisone, or equivalent, per day;

          -  Use of topical steroids within 2 weeks prior to the Screening Visit and for the
             duration of the study;

          -  Receipt of topical cyclosporine eye drops within 3 months prior to the Screening Visit
             and for the duration of the trial;

          -  Presence of chronic ocular disease other than KCS requiring topical treatment;

          -  Presence of post-burn ocular injury;

          -  Ocular herpes simplex virus infection;

          -  Concomitant use of contact lenses;

          -  Persistent intraocular inflammation or infection;

          -  Active blepharitis;

          -  Recent surgical occlusion of the lacrimal puncta;

          -  Subepithelial corneal scarring;

          -  Anesthetic or neurotrophic corneas;

          -  Hemoglobin level <9.0 gm/L;

          -  Platelet count <125,000/mm^3;

          -  White blood cell count <3500/mm^3;

          -  Serum creatinine level outside the laboratory's normal limits;

          -  Liver aminotransferase levels greater than 2 times the laboratory's upper limit of
             normal;

          -  Pregnancy, planned pregnancy, lactation, or inadequate contraception as judged by the
             Investigator;

          -  History of malignancy within the past 5 years (excluding basal cell carcinoma of the
             skin);

          -  Significant acute or chronic medical, ophthalmic, or psychiatric illness that, in the
             judgment of the Investigator, could compromise patient safety, limit the patient's
             ability to complete the study, and/or compromise the objectives of the study;

          -  Participation in another investigational drug or vaccine trial concurrently or within
             30 days; or

          -  Other conditions which would confound the study evaluations or endanger the safety of
             the patient.