Lead Researcher Discusses Combination Trial of Drugs Targeting Basic Defect
Coverage from the 2010 North American CF Conference
November 5, 2010
Shortly before the kickoff of this year’s North American Cystic Fibrosis Conference, Vertex Pharmaceuticals announced the launch of a clinical trial that will evaluate multiple combinations of two oral drugs that target the most common mutation in CF.
Cystic fibrosis is caused by defective or missing CFTR proteins, which interfere with the proper flow of chloride and sodium in and out of the cells lining the lungs and other organs.
The three-part trial will enroll up to 160 people who have two copies of the most common CFTR mutation, known as Delta F508. In individuals with this mutation, CFTR proteins do not reach the cell surface and function abnormally.
The two drugs, VX-770 and VX-809, aim to help fix these underlying problems, but in different ways.
“VX-809 helps to get the Delta F508 CFTR protein to the cell surface, and VX-770 turns it on once the protein is in place,” explained Michael P. Boyle, M.D., director of the Adult Cystic Fibrosis Program at Johns Hopkins University and principal investigator of the combination trial nationwide.
The Phase 2a trial will be carried out at 21 clinical sites, primarily in the United States but also overseas. It is designed primarily to test the safety and tolerability of the two drugs alone and in combination. Both drugs were developed as part of a collaboration with the CF Foundation.
Researchers expect to have interim data from the trial in the first half of 2011.
Hear an interview with Dr. Michael P. Boyle from the 2010 NACFC
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