Clinical trial NCT02101281: Safety and Efficacy of rhNGF Eye Drops at Different Doses in Patients With Dry Eye
Official title: An Open-label Study Evaluating Safety and Efficacy of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drops at Different Doses in Patients With Dry Eye
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dry Eye Syndrome
Keywords: Dry Eye
Lead sponsor: Dompé Farmaceutici S.p.A
Sponsor class: INDUSTRY
Collaborators: name: Cross Research S.A.
First posted: 2014-04-02
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study was to assess the efficacy and safety of different doses of rhNGF when administered as eye drops to patients with dry eye.

Detailed description: This is an open-label study evaluating safety and efficacy of recombinant human nerve growth factor (rhNGF) eye drops at different doses in patients with Dry Eye

Eligibility criteria: Inclusion Criteria:

1. Male or female patients, ≥ 18 years old;
2. Required use of artificial tears for the treatment of Dry Eye within the 3 months prior to study enrolment;
3. Current use or recommended use of artificial tears for the treatment of Dry Eye;
4. Average VAS score for typical symptoms of Dry Eye (foreign body sensation, burning/stinging, itching, pain, stick feeling, blurred vision and photophobia) ≥ 25 mm;
5. Corneal staining score with lissamine green \> 3 using the NEI corneal grading system in the worse eye (study eye);
6. Conjunctival staining score \> 3 using the NEI conjunctival grading system in the worse eye (study eye);
7. Schirmer test without anaesthesia ≤ 10 mm/5 minutes in the worse eye (study eye);
8. Tear film break-up time (TBUT) ≤ 10 seconds in the worse eye (study eye);
9. A negative urine pregnancy test if female of childbearing potential and must use adequate birth control throughout the study period

Exclusion Criteria:

1. Patient not suitable to participate in the study in the opinion of the investigator;
2. Patient with a mild or moderate Dry Eye condition (severity level less than 3 according to the Report of the International Dry Eye Workshop -DEWS, 2007) if fourteen (14) patients with mild or moderate dry eye condition have been already enrolled in the current treatment group (Group 1 and Group 2 separately);
3. Patient has had a serious adverse reaction or significant hypersensitivity to any drug or chemically related compounds or has a clinically significant allergy to drugs, foods, amide local anaesthetics or other materials including commercial artificial tears containing Hypromellose (in the opinion of the investigator);
4. Use of topical cyclosporine, topical corticosteroids or any other topical medication for the treatment of dry eye in either eye within 30 days of study enrolment. Use of own artificial tears is allowed until Visit 2;
5. Any ocular disease other than Dry Eye requiring treatment with topical medications in either eye within 30 days of study enrolment;
6. Any active ocular infection or active inflammation in either eye unrelated to Dry Eye;
7. Presence or history of any systemic or ocular disorder, condition or disease that could possibly interfere with the conduct of the required study procedures or the interpretation of the study results;
8. Use of therapeutic or Refractive Contact lenses in either eye within 30 days of study enrolment;
9. History of ocular surgery in the study eye, including corneal refractive procedures, within 90 days of study enrolment;
10. Participation in another clinical study at the same time as the present and within 30 days of study enrolment;
11. History of drug, medication or alcohol abuse or addiction.

Primary outcomes:
measure: Change From Baseline in Frequency of Dry Eye Symptoms (SANDE)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Severity of Dry Eye Symptoms (SANDE)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Ocular Surface Vital Staining (National Eye Institute [NEI] Scale)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Tear Wetting Distance as Determined by Schirmer Tear Test I - Study Eye
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs),
timeFrame: Throughout the study up to day 56
Secondary outcomes:
measure: Change From Baseline in Ocular Tolerability (Visual Analogue Scale, VAS)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Slit Lamp Examination
timeFrame: Baseline, Day 1, Day 8, Da 29 and Day 56
measure: Change From Baseline in Tear Wetting Distance as Determined by Schirmer Tear Test II - Study Eye
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Tear Film Break-Up Time (TFBUT)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Corneal Fluorescein Staining
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Corneal Sensitivity to Contact (Cochet-Bonnet Aesthesiometry)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Intraocular Pressure (IOP)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Ocular Surface Disease Index (OSDI)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Visual Acuity (BCDVA)
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Number of Participants With a Change in Fundus Ophthalmoscopy
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Change From Baseline in Tear Film Osmolarity
timeFrame: Changes from baseline up to day 56±4
measure: Change From Baseline in Conjunctival Impression Cytology for Goblet Cells' Count
timeFrame: Baseline, Day 1, Day 8, Day 29 and Day 56
measure: Mean Frequency of Artificial Tears Use
timeFrame: Day 1-Day 8, Day 9-Day 29, Day 30-Day 56 intervals
Other outcomes:
Locations:
city: Wien
country: Austria
facility: Department of Clinical Pharmacology
Study officials:
affiliation: Medical University of Vienna, Vienna General Hospital, AKH
name: Gerhard Garhöfer, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sacchetti M, Lambiase A, Schmidl D, Schmetterer L, Ferrari M, Mantelli F, Allegretti M, Garhoefer G. Effect of recombinant human nerve growth factor eye drops in patients with dry eye: a phase IIa, open label, multiple-dose study. Br J Ophthalmol. 2020 Jan;104(1):127-135. doi: 10.1136/bjophthalmol-2018-312470. Epub 2019 Apr 3.