Current Research Opportunities

Harvard Sibling Pair Study

It is known that there is considerable variation in the time of onset, and clinical symptoms of individuals affected by Alpha-1 Antitrypsin Deficiency. The study goal is to identify genes other than the gene for the alpha-1 antitrypsin protein that may influence the onset and severity of disease. The study is directed by Dr. Ed Silverman at Brigham and Women's Hospital and Harvard Medical School in Boston, Mass. To be eligible for this study you and your brother or sister must be at least 30 years old and both have ZZ type Alpha-1 Antitrypsin Deficiency. The study can be completed in a few hours and a traveling research coordinator can come to your home to do a breathing test, blood draw, and administer questionnaires. For more information contact James Keary in Boston, MA toll free at 866-328-9494.

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 developed by Baxter Healthcare. The study objective is to determine short-term safety and tolerability of the product at three different doses. Participants may recieve 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 866-284-2708.

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 saftety 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 800-749-7424 or 352-846-2286.