Alpha-1 Augmentation Therapy Clinical trial:

This study is being conducted in Beaumont Hospital by Professor McElvaney and his team. This is a placebo-controlled, double-blinded, multicentre phase III / IV study to compare the efficacy and safety of the drug Zemaira ® in patients with Emphysema due to Alpha-1 antitrypsin deficiency. The duration for each patient is 2 years.

We have recruited 16 patients so far and they are all at various stages in the trial. The trial involves having weekly intravenous infusions of Zemaira®, an alpha-1 antitrypsin product or a placebo (which is a mock treatment that looks like the real thing but has none of the activity).

As the study is double-blinded, neither the participating patients nor our study staff knows which therapy has been assigned to them. There is an equal chance of receiving either treatment. As of March 2008 we have had 6 patients graduate onto the extension phase of the study. This is where each patient receives Zemaira for up to another two years.

The infusions are given either in Beaumont Hospital or in the patient’s own home and take on average 20 minutes. Every three months patients are required to attend Beaumont Hospital so that routine tests can be carried out. These include: Monitoring of vital signs, i.e. blood pressure, weight etc.

• Blood tests.
• Pulmonary Function tests.
• Physical Examination by physician.
• Cotinine test (urine test that detects nicotine) is required.

At certain visits a Quality of Life questionnaire and CT scan are performed. These help to investigate the effect of Zemaira® on the development and progression of emphysema within the patients. The main inclusion criteria for all patients that enter onto the study are: · Diagnosis of alpha-1 antitrypsin deficiency (ZZ phenotype).

• Non smokers or Ex-Smokers who have stopped at least 6 months prior to screening.
• Age range of 18 – 65 years of age, male and female.
• Emphysema with an FEV1 of 35-70% predicted range.

As with other alpha-1 therapies, Zemaira® may not be appropriate for the following adults:

• Individuals with a known hypersensitivity and/or history of anaphylaxis or severe systemic reaction to alpha-1 antitrypsin products or their components.
• Individuals with selective IgA deficiencies who have known antibodies against IgA. This is due to Zemaira® being derived from human plasma.

All patients however will be assessed on an individual basis. In previous clinical studies, Zemaira® has been shown to be generally well tolerated and provides patients with half or less the infusion time of other available alpha-1 augmentation therapies. If you would like any further information on Zemaira® or you are interested in taking part in the trial please feel free to contact:

Máire Stack
Tel: 01 8093864/01 8093876
Research Nurse, Study Co-Ordinator