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news.For the design of a clinical trial to assess the safety and efficacy of Gencaro
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ARCA biopharma (ARCA) has submitted a study protocol for review under the FDA’s Special Protocol Assessment (SPA) process for the design of a clinical trial. The study is to assess the safety and efficacy of Gencaro in approximately 3,000 patients with chronic heart failure who have the genotype that appears to respond most favorably to Gencaro.
Gencaro is the company’s investigational, pharmacologically beta-blocker and mild vasodilator. The FDA has previously designated the investigation of Gencaro for the reduction of cardiovascular mortality and cardiovascular hospitalisations in a genotype-defined heart failure population as a fast-track development program.
The company said that an SPA is an agreement with the FDA that the proposed trial protocol design, clinical endpoints and statistical analyses are acceptable to support regulatory approval. Reportedly, the proposed trial protocol includes a superiority comparison to the beta-blocker metoprolol CR/XL, which is approved for heart failure and other indications.
Additionally, the trial protocol also includes an interim data analysis at a pre-specified number of primary endpoints, the results of which could allow ARCA to formally submit a response to the FDA’s Complete Response Letter. It could serve as the clinical effectiveness basis for FDA approval, if the results meet certain predefined criteria agreed upon with the FDA during the SPA process.
The company also said that if the proposed protocol and criteria are agreed to by the FDA, it anticipates that the proposed trial could reach the specified number of endpoint events in approximately two years after the trial begins.
The SPA submission proposes that a composite of cardiovascular mortality and cardiovascular hospitalisation serve as the primary endpoint of the trial. Any proposed trial protocol must be reviewed and agreed on by the FDA and the final trial protocol may be significantly different from the company’s initial SPA submission.
Subject to the timing and outcome of the FDA’s review of the SPA submission, and subject to the company’s ability to obtain sufficient funding, ARCA expects it could begin the proposed trial in late 2010 or the first half of 2011.
Michael Bristow, president and CEO of ARCA, said: “Submission of our trial protocol for review under the FDA’s SPA process is an important milestone for Arca and Gencaro. We believe that Gencaro has the potential to provide a needed advancement in the treatment of patients with chronic heart failure.”