A Randomized, Placebo-Controlled, Double-Blind, Multicenter Phase III/IV Study to compare the Efficacy and Safety of 60 mg/kg body weight of Zemaira weekly i.v. administration with Placebo weekly i.v. administration in Chronic Augmentation and Maintenance Therapy in Subjects with Emphysema due to Alpha1-Proteinase Inhibitor Deficiency

Investigator: Michael Campos

Institutional Protocol #: 20071125

National Clinical Trials Identifier: NCT00261833

Funding Agency/Sponsor: CSL Behring

Division: Pulmonary and Critical Care

Therapeutic Area: Other

Phase: Phase IV

Enrolling Sites: University of Miami Medical Group

Enrolling Since: 7/30/2013


Purpose/Abstract:


This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to
compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to
alpha1-proteinase inhibitor deficiency. The effect of Zemaira® on the progression of
emphysema will be assessed by the decline of lung density, measured by computed tomography
(CT).




Eligibility Criteria:


Inclusion Criteria:

- 18 to 65 years of age and willing to sign informed consent.

- Males, and non-pregnant, non-lactating females, whose screening pregnancy test is
negative and who are using contraceptives methods deemed reliable by the
investigator.

- Diagnosis of alpha1-proteinase inhibitor deficiency (serum A1-PI levels < 11 μM or <
80 mg/dL). This includes newly diagnosed subjects, previously untreated subjects,
currently treated subjects, and subjects currently not on treatment therapy but on
treatment in the past.

- Subjects with emphysema and FEV1 ≥ 35% and ≤ 70% (predicted).

- No signs of chronic or acute Hepatitis A, Hepatitis B, Hepatitis C or HIV infection
(negative serologies for HIV and viral hepatitis). In case of positive serologies for
viral hepatitis, vaccination status or negative IgM should be available.

Exclusion Criteria:

- Any relevant chronic diseases or history of relevant diseases (e.g., severe renal
insufficiency) except respiratory or liver disease secondary to alpha1-proteinase
inhibitor deficiency. Subjects with well-controlled, chronic diseases may be included
after consultation with the treating physician and the sponsor.

- Current evidence of alcohol abuse or history of abuse of illegal and/or legally
prescribed drugs such as barbiturates, benzodiazepines, amphetamines, cocaine,
opioids, and cannabinoids.

- History of allergy, anaphylactic reaction, or severe systemic response to human
plasma derived products, or known mannitol hypersensitivity, or history of prior
adverse reaction to mannitol.

- History of transfusion reactions.

- Selective IgA deficiency.

- Acute illness within one week prior to the first administration of the
investigational medicinal product (IMP). Start of treatment after recovery is
possible.

- Current tobacco smoker (smoking has to be ceased at least 6 months prior study
inclusion). Subjects with a positive cotinine test due to nicotine replacement
therapy (e.g. patches, chewing gum) or snuff are eligible.

- Conditions or behaviors that interfere with attending scheduled study visits in the
opinion of the investigator.

- History of non-compliance.

- Administration of any other experimental new drug or participation in an
investigation of a marketed product within one month prior to the screening visit
date.

- Inability to perform necessary study procedures.

- Lung transplantation, lung volume reduction surgery or lobectomy or being on a
waiting list for any such surgeries.


Gender: Both

Minimum Age: 18 Years

Maximum Age: 65 Years

Accepts healthy volunteers: No

Please note that this is written for scientific review, and if there are any questions or clarifications needed, please contact

Patricia Rebolledo, Research Assistant
(305) 243 2568
prebolledo@med.miami.edu

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