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Clinical Trial
. 2020 Jan;104(1):127-135.
doi: 10.1136/bjophthalmol-2018-312470. Epub 2019 Apr 3.

Effect of recombinant human nerve growth factor eye drops in patients with dry eye: a phase IIa, open label, multiple-dose study

Marta Sacchetti  1 , Alessandro Lambiase  2 , Doreen Schmidl  3 , Leopold Schmetterer  3 , Mauro Ferrari  4 , Flavio Mantelli  4 , Marcello Allegretti  4 , Gerhard Garhoefer  3
Affiliations
Clinical Trial

Effect of recombinant human nerve growth factor eye drops in patients with dry eye: a phase IIa, open label, multiple-dose study

Marta Sacchetti et al. Br J Ophthalmol. 2020 Jan.

Abstract

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.

Keywords: clinical trial; ocular surface; pharmacology; tears.

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Conflict of interest statement

Competing interests: MS and AL: Consultant—Dompé Farmaceutici SpA. MA, FM and MF: Employee—Dompé Farmaceutici SpA. MS, AL: report personal fees from Dompé Farmaceutici SpA, Italy, outside the submitted work. MA, MS, MF, GG: report personal fees from Dompe Farmaceutici spa, during the conduct of the study.

Figures

Figure 1
Figure 1
Study design. AE, adverse events; rhNGF, recombinant human nerve growth factor.
Figure 2
Figure 2
Both high and low doses of rhNGF eye drops treatments showed a significant improvement of DED symptoms evaluated by SANDE and OSDI scales (*p<0.05). DED, dry eye disease; rhNGF, recombinant human nerve growth factor; OSDI, Ocular Surface Disease Index; SANDE, Symptoms Assessment in Dry Eye.
Figure 3
Figure 3
Both high and low doses of rhNGF eye drops treatments showed a significant improvement of ocular surface damage evaluated by lissamine green staining (NEI score) (*p<0.05). NIE, National Eye Institute; rhNGF, recombinant human nerve growth factor.
Figure 4
Figure 4
The higher dose of rhNGF at 20 µg/mL concentration showed improvement of lacrimal function at each time point (*p<0.05). rhNGF, recombinant human nerve growth factor; TBUT, tear film break-up time.
See this image and copyright information in PMC

References

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