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

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: 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 T

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 Summary:
Sponsor: Dompé Farmaceutici S.p.A

Current Primary Outcome: Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: up to 48 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Best Corrected Distance Visual Acuity (BCDVA) (ETDRS chart) [ Time Frame: Day 0, Week 12, 24, 36, 48 ]
    Efficacy as measured by Best Corrected Distance Visual Acuity (BCDVA) test results will be analyzed by means of analysis of variance, including terms for treatment, time (Day 0, Week 12, 24, 36, 48), and treatment by time interaction.
  • Change in ocular tolerability - VAS [ Time Frame: Weeks 1, 2, 6, 12, 24 ]
    Ocular tolerability as measured by the visual analogue scale (VAS), will be analyzed by means of analysis of variance, including terms for treatment, time (Weeks 1, 2, 6, 12, 24), and treatment by time interaction
  • Change in ocular tolerability - Dilated fundus ophthalmoscopy [ Time Frame: Day 0, Weeks 12, 24 and 48 ]
    Ocular tolerability as measured by fundus appearance, will be analyzed by means of analysis of variance, including terms for treatment, time (Day 0, Weeks 12, 24 and 48), and treatment by time interaction
  • Presence of Anti-NGF antibodies [ Time Frame: At day 0 and at week 24 ]
    Anti-NGF antibodies and laboratory tests are performed at screening and at the end of treatment and shift tables will be used to present shifts from within/outside the normal range between screening and end of treatment


Original Secondary Outcome: Same as current

Information By: Dompé Farmaceutici S.p.A

Dates:
Date Received: April 8, 2014
Date Started: January 2014
Date Completion: November 2015
Last Updated: March 16, 2015
Last Verified: March 2015