Intravenous Therapy for Patients with Alpha1-Proteinase Inhibitor Deficiency

Study Summary

The University of Chicago and Dr. Strek are conducting a research study to assess the effectiveness of an intravenous study drug for people who have emphysema due to alpha-1 antitrypsin deficiency. This study drug is approved by the FDA.

This is a randomized (like a flip of a coin), placebo (inactive product) controlled, double blind study. Neither you nor your doctor will know if you get study drug or placebo during the study.

This is a 24-month study that includes a screening and observation period of up to four weeks, followed by a 23-month study drug or placebo period. You will be screened to see if you qualify for participation. If you do, you will be randomized to study drug or placebo. Some of the procedures that may be done during the study include physical examinations, medical history, blood and urine testing, chest CT scans, breathing and walking tests, and questionnaires.

You will be reimbursed for travel, lodging and food expenses.

Benefits

You may not receive any direct health benefit from participating in this study. However, you may benefit by being followed for your alpha1-proteinase inhibitor deficiency and your general health status. The knowledge gained from this study could provide valuable information about alpha1-proteinase inhibitor deficiency and its treatments and, additionally, help other people with alpha1-proteinase inhibitor deficiency.

Participation Details

Initial selection criteria include:

• Must be male or non-pregnant, non-lactating female between the ages of 18 and 65
• Diagnosis of alpha-1 proteinase inhibitor deficiency
• Currently not smoking tobacco

Study Director: Dr. Mary Strek
Department: Pulmonary

For more information contact:
smalecka@medicine.bsd.uchicago.edu
(773) 834-4053

IRB: #16411A