Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency

CSL Behring

Status and phase

Completed

Phase 4

Conditions

Emphysema

Alpha1-proteinase Inhibitor Deficiency

Treatments

Biological: Alpha1-proteinase inhibitor

Other: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00261833

2005-003459-12 (EudraCT Number)

CE1226_4001

1449 (Other Identifier)

Details and patient eligibility

About

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).

Enrollment

180 patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

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 (A1-PI) 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 forced expiratory volume in 1 second (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.