Safety and Efficacy Study of Single Doses of TT-034 in Patients With Chronic Hepatitis C
The study is a first in man, dose escalation study that will measure the safety and efficacy of TT-034 in the treatment of patients with chronic hepatitis C. The study is divided into 5 dose levels. Subjects will be given a single dose delivered by IV infusion. The subjects will be monitored and the data analyzed. After a set time, between 6 and 10 weeks depending on the dose level, the next set of subjects will be dosed. The study drug is a gene therapy treatment that produces molecules that destroy the Hepatitis C virus (HCV) in infected cells. Once the study drug is given, it cannot be withdrawn. Additionally, once an individual receives a dose, he or she will not be able to receive a second dose, but will remain eligible to receive most other HCV treatments.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I,II Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of Single Doses of TT-034 for Subjects With Chronic Hepatitis C (CHC) Infection|
- The primary objective is to assess the safety and tolerability of single escalating doses of TT-034 administered IV as a single infusion to subjects with CHC. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- To assess the activity of TT-034 on the viral load of subjects with CHC receiving single escalating doses of TT-034 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- To determine the maximum tolerable dose or the optimal efficacy dose (whichever comes first) [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- To determine the viral load [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- To assess the presence of viral escape mutants in subjects with detectable viral load after receiving TT-034 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- To monitor TT-034 vector DNA levels and shRNA expression in the target organ (liver) after dosing with TT-034 [ Time Frame: 3 weeks ] [ Designated as safety issue: No ]
- To monitor TT-034 vector DNA levels and shRNA expression peripherally (in blood) after dosing with TT-034 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||January 2014|
|Estimated Study Completion Date:||January 2016|
|Estimated Primary Completion Date:||September 2015 (Final data collection date for primary outcome measure)|
Experimental: Escalating Dose of TT-034
The study contains one dose escalation arm with active drug.
The study drug will be given as a single dose IV infusion on Day 1. 5 different dose levels corresponding to the 5 cohorts of the study will be given.
This is a first-time use of a method of therapy designed to transfer anti-HCV genetic sequences into the hepatocytes of subjects infected with HCV. The anti-HCV sequences will be comprised of three different short hairpin RNAs (shRNA) that have the ability to directly cleave the RNA genome of HCV by a process known as RNA interference. The transfer of the anti-HCV sequences will be accomplished using a "vector" that was made from an adenovirus-associated virus (AAV) by removing the viral genes and replacing them with a non-replicating genetic sequence that produces three different shRNA that target three different regions within the HCV genes. This type of vector has been used in other clinical trials in order to transduce the hepatocytes of subjects who suffer from hemophilia.
|United States, California|
|UCSD Antiviral Research Center||Not yet recruiting|
|San Diego, California, United States, 92103|
|Principal Investigator: David Wyles, MD|
|United States, North Carolina|
|Duke Clinical Research Institute||Not yet recruiting|
|Durham, North Carolina, United States, 27710|
|Contact: Vicki Robertson 919-613-6246 firstname.lastname@example.org|
|Principal Investigator: Keyur Patel, MD|
|Study Director:||David Suhy, PhD||Tacere Therapeutics, Inc.|
|Principal Investigator:||Keyur Patel, MD||Duke Clinical Research Institute|
|Principal Investigator:||David Wyles, MD||University of California, San Diego|