Conditions: Retinitis Pigmentosa
Keywords: Cone-Rod Degenerations Cone-Rod Dystrophy Cone-Rod Dystrophy 2 Cone-Rod Retinal Dystrophy Pigmentary Retinopathy Retinal Cone-Rod Dystrophy Rod Cone Dystrophies Rod-Cone Dystrophy Tapetoretinal Degeneration
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 50
Healthy volunteers: no

Brief summary:
The primary objective of the study is to assess the safety and tolerability of two dose regimens of recombinant human nerve growth factor (rhNGF) eye drops solution administered over 6 months versus a vehicle control in patients with typical retinitis pigmentosa. The secondary objective of this study is to attempt to show a dose response by assessing the potential efficacy of the rhNGF dose regimens for improving or slowing the deterioration of visual function outcomes at 3 and 6 months. During a 6 month follow-up period patients will be monitored to determine if there is evidence of a persistent biological effect after discontinuation of the study treatment.

Detailed description:
This is a 24-week phase Ib/II, multicenter, randomized, double-masked, vehicle controlled, parallel-group, dose-ranging study with a 24-week follow-up period to evaluate the safety and potential efficacy of two doses (60 μg/ml and 180 μg/ml) of recombinant human nerve growth factor (rhNGF) eye drops solution versus vehicle in patients with typical retinitis pigmentosa (RP).

Arms:
arm 1: rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes arm 2: rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes. arm 3: Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes.

Interventions: intervention 1: rhNGF 60 µg/ml eye drops solution intervention 2: rhNGF 180 µg/ml eye drops solution intervention 3: Placebo
Intervention descriptions: intervention 1: rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes intervention 2: rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes intervention 3: Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes

Protocol document: