R-Tech RU-101 Ophthalmic Solution in Patients with severe dry eye
|Principal Investigator: NickMamalis|
|Keywords: Dry eye||Department: Ophthalmology-Services|
|IRB Number: 00066755||Co Investigator:|
|Specialty: Ophthalmology, Ophthalmology|
|Sub Specialties: Comprehensive Ophthalmology, Cornea|
This is a Phase 1/2, double-masked, dose-escalating study of RU-101 ophthalmic solution in
patients with severe dry eye. The primary goal is to evaluate safety and tolerability of repeated
instillation of RU-101, but effects on signs and symptoms of dry eye will also be evaluated.
Stage I of the study is designed to determine the maximum tolerated dose (MTD). Stage II will
better define the safety, tolerability, and efficacy of the dose selected in Stage I.
To determine the safety and tolerability of escalating doses of RU-101 for 4 weeks in patients with
severe dry eye
To determine the maximum tolerated dose (MTD)
To explore the efficacy of RU-101
To determine the safety and tolerability of RU-101 for 12 weeks in patients with severe dry eye
To explore the efficacy of RU-101
To explore optimal endpoints for future studies
Each patient must meet the following criteria at Screening to be enrolled in the study:
1. Male or female patients aged 18 years old and older.
2. Patients have had dry eye symptoms at least 4 months.
3. Patients have the following signs in the worst eye:
a. Corneal staining score with fluorescein staining ≥ 6/15 with at least one in the
b. Conjunctival staining score with Lissamine Green ≥ 2/18.
4. Patients who had been receiving treatment with eye drops of artificial tears, Restasis, or
topical steroids but have had insufficient efficacy or have found these products to be
intolerable. If using Restasis or topical steroids, patients must agree to a 30-day washout
period prior to the first IP dose (Day 0).
5. Female patients of child bearing potential must agree to have a urine pregnancy test
performed at Screening (must be negative) and agree to use a medically acceptable form of
birth control (e.g., intrauterine device, birth control pill, patch or subcutaneous implant,
condoms, diaphragm, or abstinence) throughout the duration of IP instillation. Women
considered capable of becoming pregnant include all females who have experienced
menarche and who have not experienced menopause (as defined by amenorrhea for
> 12 consecutive months) or have not undergone successful surgical sterilization
(hysterectomy, bilateral tubal ligation, or bilateral oophorectomy).
Patients who meet any of the following criteria at Screening will be excluded from the study:
1. Presence of anterior eye diseases except dry eye.
2. Intraocular pressure ≥ 22 mmHg at Screening.
3. Use of any ophthalmic solutions (including over-the-counter [OTC] solutions), serum eye
drops, or contact lenses during the Treatment Phase. Artificial tears are allowed during the
Screening Phase but must not be used from the first dose (Day 0) through the Treatment
4. Use of eye drops of Restasis or topical steroids within 30 days of the first dose (Day 0).
5. Use of antihistamines or tricyclic antidepressants during the Treatment Phase. (Topical antihistamines for dermatological use are allowed during the study and systemic beta blockers and antidepressants (as long as stable doses during the study) other than tricyclic antidepressants are allowed in Stage II.)
6. Patients with previous corneal transplantation, kerato-refractive surgery, or laser-assisted in
situ keratomileusis (LASIK).
7. Presence of graft-versus-host disease (GVHD).
8. Patients who have had other ocular surgery within 3 months prior to the first dose.
9. Patients with punctal plugs or punctal cautery < 3 months prior to the first dose.
10. Patients with severe dry eye due to Stevens-Johnson disease or ocular cicatricial
11. History of allergy to human serum protein products and/or any history of allergy to yeast.
12. History of allergies to recombinant products, ophthalmic solutions, any constituents of
RU-101, or any solutions planned for use in this study.
13. Any significant chronic illness that, in the opinion of the Principal Investigator (PI), could
interfere with the study parameters.
14. Use of any investigational product or device within 30 days prior to the Screening Visit or
during the study.
15. Known history of alcohol and/or drug abuse within the past 12 months, which in the opinion
of the PI could interfere with study compliance, outcome measures including safety
parameters, and/or the general medical condition of the patient.
16. Those unable in the opinion of the PI to comply fully with the study requirements.