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Data Monitoring Committee Recommends Continuing Phase 2 Trial of Gene Therapy in Cystic Fibrosis
SEATTLE, June 23 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation today announced that an independent data monitoring committee (DMC) met for its scheduled interim analysis of a Phase IIb clinical trial of the Company's product candidate, tgAAVCF, to treat patients with cystic fibrosis (CF). Based upon its review, the DMC recommended continuation of the study as planned. The DMC provided its recommendation based upon an analysis of whether or not there was a chance that, upon full patient enrollment, the study could show a statistically significant positive impact on lung function measurements in patients treated with tgAAVCF compared to placebo.
"Targeted Genetics' ongoing Phase IIb clinical trial has advanced further than any other CF gene therapy program to date, and we are pleased to continue this study based on the DMC's recommendation," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "This recommendation allows us to continue to generate clinical results and assess the ability of this product candidate to improve lung function in patients with CF. There remains a significant unmet need in the CF patient community for treatments that address the underlying cause of this chronic disease. We are optimistic about this trial and hope it will lead to a true advance in the treatment of CF."
This Phase IIb, double-blind, randomized, placebo-controlled study is partially funded by Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the drug discovery and development affiliate of the Cystic Fibrosis Foundation, and is being conducted through CFFT's Therapeutics Development Network. The trial includes bi-monthly evaluation of lung function after repeat dosing of tgAAVCF. Researchers also are assessing the impact of tgAAVCF on inflammation and biologic markers over time when compared to placebo. The study continues to monitor the safety and tolerability profile of tgAAVCF. A total of 100 patients, 12 years of age and older, are being evaluated, 50 in the treatment group and 50 in the placebo group. Study participants receive two doses of 1x1013 DNAse resistant particles (DRP) of tgAAVCF or placebo, delivered via nebulizer at day 0 and day 30 of the study and are evaluated for efficacy every two weeks for a total of 90 days. Study participants are being monitored for safety for seven months.
The planned interim analysis was conducted after 53 patients were dosed with either the product candidate or placebo. The DMC did not raise any safety concerns and recommended continuation of the trial. In order to protect the integrity of the trial, the complete, unblinded results will not be made available to Targeted Genetics until the study is completed. The DMC is an independent group of experts that was established by CFFT to monitor the safety and efficacy data. The primary role of the DMC is to safeguard the interests of current and future patients in the trial and act in an advisory capacity to Targeted Genetics and the Cystic Fibrosis Foundation.
Source: Targeted Genetics Corporation