Phase 2 randomized, double-masked, vehicle-controlled trial of recombinant human nerve growth factor for neurotrophic keratitis

2018

OPHTHALMOLOGY

Phase 2 randomized, double-masked, vehicle-controlled trial of recombinant human nerve growth factor for neurotrophic keratitis

01 Pubblicazione su rivista

Bonini Stefano, Lambiase Alessandro, Rama Paolo, Sinigaglia Francesco, Allegretti Marcello, Chao Wendy, Mantelli Flavio, Bonini Stefano, Lambiase Alessandro, Rama Paolo, Messmer Elisabeth, Aragona Pasquale, Geerling Gerd, Mastropasqua Leonardo, Mencucci Rita, Dart John, Leonardi Andrea, Montero Jesus, Rolando Maurizio, Reinhard Thomas, Cursiefen Claus, Etxebarria Jaime, Gabison Eric, Szaflik Jacek P., Borderie Vincent, De La Paz Maria, Sainz de la Maza Maite, Wylegala Edward, Figueiredo Francisco, Fogagnolo Paolo, Hossain Parwez, Lorenz Katrin, Robert Pierre-Yves, Benitez del Castillo José, Creuzot-Garcher Catherine, Kruse Friedrich, Malecaze François, Merayo-Lloves Jesús, Rauz Saaeha, Alio Jorge, Carley Fiona, Kanna Ramaesh, Koppen Carina, Nemeth Janos, Murta Joaquim Neto, Torrao Luis

DOI: 10.1016/j.ophtha.2018.02.022

ISSN: 0161-6420

Purpose: To evaluate the safety and efficacy of topical recombinant human nerve growth factor (rhNGF) for treating moderate-to-severe neurotrophic keratitis (NK), a rare degenerative corneal disease resulting from impaired corneal innervation. Design: Phase 2 multicenter, randomized, double-masked, vehicle-controlled trial. Participants: Patients with stage 2 (moderate) or stage 3 (severe) NK in 1 eye. Methods: The REPARO phase 2 study assessed safety and efficacy in 156 patients randomized 1:1:1 to rhNGF 10 μg/ml, 20 μg/ml, or vehicle. Treatment was administered 6 drops per day for 8 weeks. Patients then entered a 48- or 56-week follow-up period. Safety was assessed in all patients who received study treatment, whereas efficacy was by intention to treat. Main Outcome Measures: Corneal healing (defined as

OPHTHALMOLOGY