Children’s Hospital partakes in trial investigating genetic disorder
Posted on Sunday, August 12, 2007
Arkansas Children’s Hospital has begun treatment of the first patient in the a confirmatory national clinical trial for hereditary angioedema, a rare, life-threatening genetic disorder that causes swelling and inflammation in the hands, feet, face, gastrointestinal tract, respiratory system or throat.
EDEMA 4 is a clinical trial by biopharmaceutical company, Dyax Corp., and is to be used to support the validity of an earlier completed trial phase for a drug candidate to treat acute attacks of hereditary angioedema.
“ Currently there is no treatment on the market for hereditary angioedema, ” says Dr. Stacie Jones, M. D., Chief of Allergy and Immunology at Arkansas Children’s Hospital, researcher at the Arkansas Children’s Hospital Research Institute and associate professor of pediatrics at the University of Arkansas for Medical Sciences College of Medicine. “ We are excited about treating the first patient in the EDEMA 4 trial, and we are pleased to be on the cutting edge in looking for treatments for these patients, and we hope to increase awareness of possible treatments for people who have this disease. ”
The treatment, subcutaneous injections of a plasma kallikrein inhibitor, can reverse attacks of hereditary angioedema by blocking the inflammatory cascade that causes swelling. Attacks can occur spontaneously, or they may be the result of physical trauma or emotional stress.
“ This is acute care therapy, not preventive therapy, and it’s only available in clinical trial format at our center, ” says Jones, principal investigator for the trial at Arkansas Children’s Hospital. “ This has been overwhelmingly changing for this patient’s quality of life, activity level and life-threatening potential. ”
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