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Early next year, researchers at the University of North Carolina at Chapel Hill will begin enrolling infants in a clinical trial to test the effectiveness of inhaled saline as a treatment for cystic fibrosis lung disease.

It’s a treatment that was proven to work in adults in two earlier studies – one by researchers at UNC and one funded by the Cystic Fibrosis Foundation in Australia. Both studies were published in The New England Journal of Medicine in January 2006.

The new study will be the first to test the treatment in infants over an extended period of time, said Dr. Stephanie Davis, an associate professor of pediatrics in the UNC School of Medicine and one of three principal investigators in the multicenter clinical trial.

“Our hope is that inhaled saline will be just as effective in treating cystic fibrosis lung disease in infants as it is in adults,” Davis said. “If so, then this therapy may prevent infants from developing the severe lung problems that ultimately lead to death in most people with cystic fibrosis, and the therapy may also lead to an improved quality of life.”

The clinical trial, called “Infant Study of Inhaled Saline” or ISIS, is being funded by Cystic Fibrosis Foundation Therapeutics, Inc., a non-profit affiliate of the Cystic Fibrosis Foundation. UNC will receive approximately $3 million in funding for the study over a five-year period.

“This is an important clinical trial for infants because it has the potential to maintain lung health using a novel approach,” said Dr. Preston W. Campbell, executive vice president of medical affairs for the CF Foundation.

Cystic fibrosis is a life-threatening genetic disease that affects about 30,000 children and adults in the United States. It causes serious lung infections and digestive complications. Respiratory failure is the leading cause of death in CF patients. It appears on average in one of four children of parents who both carry a defective copy of a gene known as cystic fibrosis transmembrane conductance regulator or CFTR. Children born with the disease often develop chronic lung damage, since their lungs cannot effectively clear excessively sticky mucus by sweeping it from the distal airways to the mouth.

The study seeks to enroll 150 infants at a minimum age of 4 months at 15 sites in the U.S. and one site in Canada. By inhaling a saltwater aerosol solution almost twice as salty as the Atlantic Ocean for 15 minutes at least twice a day, it is hoped the infants should be able to avoid a significant part of the damage the disease causes to their lungs. That’s because the aerosolized saltwater restores the thin lubricant layer of water that normally coats airway surfaces. This water layer promotes the clearance of the naturally occurring mucus the body uses to trap harmful bacteria, viruses and other foreign particles.

Each infant in the study will receive the treatment for one year, but it is expected to take five years to complete the entire study, Davis said.
In addition to Davis, the study’s principal investigators are Dr. Margaret Rosenfeld, M.P.H., of Children’s Hospital and Regional Medical Center in Seattle, and Dr. Felix Ratjen of The Hospital for Sick Children in Toronto. Other UNC investigators taking part in the study include Dr. Scott Donaldson and respiratory therapist Robin Johnson.