REPARO NGF0212 Phase I/II Neurotrophic Keratitis Trial

• Research type

  Research Study

• Full title

  An 8-week phase I/II, multicenter, randomized, double-masked, vehicle controlled parallel group study with a 48 or 56 week follow-up period to evaluate the safety and efficacy of two doses (10 µg/ml and 20 µg/ml) of recombinant human nerve growth factor eye drops solution versus vehicle in patients with Stage 2 and 3 of Neurotrophic Keratitis

• IRAS ID

  114922

• Contact name

  John Kenneth George Dart

• Sponsor organisation

  Dompé S.p.a

• Eudract number

  2012-002527-15

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  An 8-week phase I/II, multicenter, randomized, double-masked, vehicle controlled parallel group study with a 48 or 56 week follow-up period to evaluate the safety and efficacy of two doses (10 æg/ml and 20 æg/ml) of recombinant human nerve growth factor eye drops solution versus vehicle in patients with Stage 2 and 3 of Neurotrophic Keratitis.Neurotrophic keratitis (also called neurotrophic keratopathy) is a rare disease of the corneal epithelium that occurs when the sensory nerves to the cornea (the clear front part of eye that covers the iris and pupil) do not function properly. In patients with neurotrophic keratitis, the corneal epithelium breaks down over time due to a loss or abnormality in the sensory nerves that help the cornea detect sensations like touch, pain or temperature. Since the protective barrier is broken down, this disease can cause the cornea to be prone to injury that can lead to corneal ulcers, perforation, infection, and scarring.As of today there are no medical treatments that can induce corneal healing in NK patients outside of discontinuation of topical or systemic medications that may be toxic to the cornea or may reduce corneal sensitivity and the administration of preservative-free lubricants. Additionally, there are no medications available that can restore decreased corneal sensitivity, the principal underlying impairment in NK patients.Nerve Growth Factor (NGF) is a small protein that is produced by the body naturally and it plays an important role in the growth and survival of nerve cells. Recombinant human Nerve Growth Factor (rhNGF) is a manufactured drug (protein) that is very similar to the body??s natural NGF. Since a loss or abnormality of sensory nerves to the cornea is involved in causing neurotrophic keratitis, it is thought that treatment with rhNGF may help patients with this disease. This study will recruit approximately 174 patients (58 per arm).To participate in the study, patients must be adults with stage 2 or stage 3 neurotrophic keratitis. The purpose of this study is to find out the safety and effectiveness of rhNGF eye drops solution in healing the corneal epithelium or corneal ulcers in patients with neurotrophic keratitis. It will also assess the duration of complete healing, improvement in visual acuity (sharp sight) and improvement in corneal sensitivity following treatment with rhNGF eye drops solution.The study will run for 64 weeks (approximately 16 months) and there will be two periods in this study:1) an 8 week PHASE 1/2 double-masked, randomized, controlled treatment period and2) a 48 or 56 week follow-up period.The design of the PHASE 1 & PHASE 2 segments of the study are identical with the exception that in the phase I segment of the study the randomisation scheme is different and patients will be followed with additional safety assessments and blood samples.The duration of the follow-up period (48 weeks or 56 weeks) will depend on which treatment participants received during the 8-week treatment period and on whether their eyes have healed by the end of the 8-week treatment period.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  13/LO/0049

• Date of REC Opinion

  1 Mar 2013

• REC opinion

  Further Information Favourable Opinion