Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
Status:
Completed
Trial end date:
2002-03-01
Target enrollment:
Participant gender:
Summary
This study will evaluate the safety and effectiveness of the drug, interferon gamma, in
treating leukocyte adhesion deficiency type I (LAD I). Patients with this inherited immune
disorder do not have enough proteins called adhesion molecules on their infection-fighting
white blood cells, impairing the ability of these cells to get to the site of infection. As a
result, patients have recurrent infections of soft tissues, such as the skin, gums and
gastrointestinal tract, and poor wound healing. Infants with severe LAD I often die from
multiple infections. Interferon gamma may increase the number of adhesion molecules on white
blood cells, and thus improve their function.
Patients with LAD I who weigh more than 13 kilograms (28.5 pounds) may be eligible for this
study. Candidates will have personal and family medical histories taken, a physical
examination, blood and urine tests and a chest X-ray or computed tomography (CT) scan.
Participants will receive injections of interferon gamma under the skin 3 times a week for 3
months. Adult patients will be taught how to give their own injections (similar to insulin
injections for diabetes) and parents will be taught how to administer the shots to their
child. Blood samples, usually be between 30 to 90 milliliters (2 to 6 tablespoons), will be
drawn just before starting medication and again 1 day, 1 week, 1 month, 3 months and 4 months
after therapy begins. At these same time intervals, patients will provide a salt-water mouth
rinse specimen, which will be tested for changes in the number of white blood cells during
interferon gamma treatment.
Patients will be admitted to the NIH Clinical Center for inpatient evaluations at the start
of therapy and again after 1 week, 1month, 3 months and 4 months. The initial screening visit
will take a few days and subsequent visits will take 1 to 2 days.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)