Current Research Opportunities

The most current research opportunities can be found at www.clinicaltrials.gov. Choose "Search for Clinical Trials," and enter "Alpha-1 Antitrypsin Deficiency" in the search box.

Investigation of an Aerosolized, Recombinant Alpha-1 Antitrypsin in Subjects with Alpha-1 Antitrypsin Deficiency

Individuals with ZZ Alpha-1 Antitrypsin Deficiency (or alpha-1 protein level <11um) and FEV1 greater than 50% predicted after bronchodilator therapy are invited to consider participation in a clinical trial to study the effects of an inhaled, recombinant, alpha-1 antitrypsin therapy. The study objective is to determine short-term safety and tolerability of the product at three different doses. Participants may receive treatment or placebo (inactive substance). The study site is located at the University of Florida in Gainesville. For more information please contact Pam Schreck, RN, MSN at 352-294-0512 or toll free at 1-866-284-2708, ext. 3.

Alpha-1 Gene Therapy Study

The University of Florida in collaboration with the Alpha-1 Foundation and NIH is conducting a study to determine the safety of an intramuscular injection of a recombinant Adeno-Associated Virus Alpha-1 Antitrypsin Gene Vector for adults. To be eligible you must have a confirmed severe type Alpha-1 Antitrypsin Deficiency, be at least 18 years of age, and have FEV1 > 30% of predicted among other criteria. For more information or to inquire about participation, please contact Margaret Humphries toll-free at 1-800-749-7424 or 352-846-2286.

Alpha-1 Antitrypsin and Macrophage Function Study

The University of Florida is conducting a study to evaluate the differences in inflammatory markers in the blood, pulmonary function testing, and gene expression of alpha-1 antitrypsin deficiency in individuals who have normal vs. abnormal levels of alpha-1 antitrypsin.  Participants may be of any genotype.  Participation in this research will require about a half-day outpatient stay in the General Clinical Research Center at the University of Florida.  For more information, please call 1-800-559-5706, or (352) 273-6541.

Last updated: January 28, 2009