Clinical Trial: RST-001 Phase I/II Trial for Retinitis Pigmentosa

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Uniocular Intravitreal RST-001 in Patients With Retinitis Pigmentosa (RP)

Brief Summary:

RST-001 is a gene therapy given as an injection into the eye and delivers a gene encoding a photo switch, channelrhodopsin-2, (optogenetics) to cells in the retina of the eye. When expressed, the channelrhodopsin-2 protein can depolarize in response to light thus generating a signal that is transmitted to the brain.

The study is composed of two parts. An initial dose-ranging study (part 1) is proposed whereby three dose levels of RST-001 will be studied in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent which is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies.


Detailed Summary:
Sponsor: RetroSense Therapeutics

Current Primary Outcome: Safety [ Time Frame: 6 months ]

Any Grade 3 (National Cancer Institute [NCI] grading system) or greater adverse event considered related to injected test article


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: RetroSense Therapeutics

Dates:
Date Received: September 21, 2015
Date Started: November 2015
Date Completion: April 2017
Last Updated: October 7, 2016
Last Verified: October 2016