This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
- Investigator
- Jorge E Lascano
- Status
- Accepting Candidates
- Ages
- 18 Years - N/A
- Sexes
- All
Cystic fibrosis is a genetic disease that causes thick, sticky mucus to build up in the lungs, digestive tract and other areas of the body. It’s one of the most common chronic lung diseases in children and young adults and is a life-threatening disorder.
CF.
Cystic fibrosis (CF) is a disease that is passed down through families. It’s caused by a defective gene that interrupts or decreases the transport of chloride in the surface of the cells. By doing this, the amount of water on the surface of cells decreases, making the mucus abnormally thick and sticky. This mucus builds up in multiple organs, including the lungs and pancreas.
The buildup of mucus results in bacteria proliferation and destruction of the airways (breathing pipes), leading to life-threatening lung infections and serious digestion problems. The disease may also affect the sweat glands and a man's reproductive system.
Many people carry a CF gene but don’t have symptoms. This is because a person with CF must inherit two defective genes, one from each parent.
Most children with CF are diagnosed by age 2. For a small number, the disease is not detected until later in life. These patients often have a milder version of the disease.
A blood test is done to help detect CF. The test looks for changes in the CF gene. A sweat chloride test is the standard diagnostic test for CF. A high salt level in the person's sweat is a sign of the disease.
An early diagnosis of CF and subsequent treatment plan can improve both survival and quality of life. Follow-up and monitoring are very important. When possible, care should be received at a cystic fibrosis specialty clinic. When children reach adulthood, they should transfer to a cystic fibrosis specialty center for adults.
Another component in the management of CF lung disease is airway clearance. The goal is not only to make the mucus thinner, but to help expectorating the mucus.
Ivacaftor, Ivacaftor/Lumacaftor and Tezacaftor/Ivacaftor are new medicines that treat different patients with CF based on their gene mutations. These medications improve the transport of chloride in the surface of the cell. As a result, there is less buildup of thick mucus in the lungs. Patients taking these medications have an improvement in their lung function that ranges from 4 to 10 percent. Other CF symptoms are improved as well.
You can ease the stress of illness by joining a cystic fibrosis support group. Sharing with others who have common experiences and problems can help your family to not feel alone.
Most subjects with CF stay in good health through adulthood. They are able to take part in most activities and attend school. Many adults with CF finish college or find jobs.
Lung disease can eventually get worse to the point where the person is disabled. Today, the average life span for people with CF who live to adulthood is about 39 years.
Death is most often caused by lung complications.
The most common complication is chronic respiratory infection.
Call your provider if you experience any of the following symptoms:
Call your provider if a person with CF develops new symptoms or if symptoms get worse, particularly severe breathing difficulty or coughing up blood.
CF cannot be prevented. Screening those with a family history of the disease may detect the CF gene in many carriers.
Accurso FJ. Cystic fibrosis. In: Goldman L, Schafer AI, eds. Goldman's Cecil Medicine. 25th ed. Philadelphia, PA: Elsevier Saunders; 2016:chap 89.
Cutting GR. Cystic fibrosis. In: Rimoin DL, Pyeritz RE, Korf BR, eds. Emery and Rimoin's Principles and Practice of Medical Genetics. 6th ed. Philadelphia, PA: Elsevier; 2013:chap 58.
Egan ME, Green DM, Voynow JA. Cystic fibrosis. In: Kliegman RM, Stanton BF, St Geme JW, Schor NF, eds. Nelson Textbook of Pediatrics. 20th ed. Philadelphia, PA: Elsevier; 2016:chap 403.
Review Date
5/24/2018
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