Effect of recombinant human nerve growth factor eye drops in patients with dry eye: a phase IIa, open label, multiple-dose study.
BACKGROUND
Dry eye disease (DED) affects more than 14% of the elderly population causing decrease of quality of life, high costs and vision impairment. Current treatments for DED aim at lubricating and controlling inflammation of the ocular surface. Development of novel therapies targeting different pathogenic mechanisms is sought-after. The aim of this study is to evaluate safety and efficacy of recombinant human nerve growth factor (rhNGF) eye drops in patients with DED.


METHODS
Forty consecutive patients with moderate to severe DED were included in a phase IIa, prospective, open label, multiple-dose, clinical trial to receive rhNGF eye drops at 20 µg/mL (Group 1: G1) or at 4 µg/mL (Group 2: G2) concentrations, two times a day in both eyes for 28 days (NCT02101281). The primary outcomes measures were treatment-emerged adverse events (AE), Symptoms Assessment in Dry Eye (SANDE) scale, ocular surface staining and Schirmer test.


RESULTS
Of 40 included patients, 39 completed the trial. Both tested rhNGF eye drop concentrations were safe and well tolerated. Twenty-nine patients experienced at least one AE (14 in G1 and 15 in G2), of which 11 had at least 1 related AE (8 in G1 and 3 in G2). Both frequency and severity of DED symptoms and ocular surface damage showed significant improvement in both groups, while tear function improved only in G1.


CONCLUSIONS
The data of this study indicate that rhNGF eye drops in both doses is safe and effective in improving symptoms and signs of DED. Randomised clinical trials are ongoing to confirm the therapeutic benefit of rhNGF in DED.


TRIAL REGISTRATION NUMBER
NCT02101281.
['Sacchetti|Marta|M|', 'Lambiase|Alessandro|A|0000-0002-8974-991X', 'Schmidl|Doreen|D|', 'Schmetterer|Leopold|L|0000-0002-7189-1707', 'Ferrari|Mauro|M|', 'Mantelli|Flavio|F|', 'Allegretti|Marcello|M|', 'Garhoefer|Gerhard|G|']
D000328:Adult D000368:Aged D004305:Dose-Response Relationship, Drug D004334:Drug Administration Schedule D015352:Dry Eye Syndromes D005260:Female D006801:Humans D065346:Lubricant Eye Drops D008297:Male D008875:Middle Aged D009414:Nerve Growth Factors D011994:Recombinant Proteins D012720:Severity of Illness Index D013666:Tears D016896:Treatment Outcome
ocular surface clinical trial tears pharmacology