NCT02110225

A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)

A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: Dompé Farmaceutici S.p.A · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	rhNGF 60 µg/ml eye drops solution	rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
DRUG	rhNGF 180 µg/ml eye drops solution	rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
DRUG	Placebo	Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes

Timeline

• Start date: 2014-01-01
• Primary completion: 2015-11-01
• Completion: 2015-11-01
• First posted: 2014-04-10
• Last updated: 2024-04-18
• Results posted: 2019-07-11

Locations

5 sites across 1 country: Italy