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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00261833
• Date of registration: 02/12/2005
• Prospective Registration: Yes
• Primary sponsor: CSL Behring
• Public title: Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
• Scientific title: A Randomized, Placebo-Controlled, Double-Blind, Multicenter Phase III/IV Study to Compare the Efficacy and Safety of 60mg/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
• Date of first enrolment: March 2006
• Target sample size: 180
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00261833
• Study type: Interventional
• Phase: Phase 4

Countries of recruitment

Australia, Bulgaria, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Russian Federation, Sweden, United States

Contacts

• Name: Senior Director Immonology & Pulmonology, Clinical R&D
• Affiliation: CSL Behring

Key inclusion & exclusion 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 (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.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Alpha1-proteinase Inhibitor Deficiency

Emphysema

Intervention(s)

Other: Placebo

Biological: Alpha1-proteinase inhibitor

Primary Outcome(s)

Annual Rate of Change in Lung Density [Time Frame: Over a 2-year period]

Secondary Outcome(s)

Change in Lung Density [Time Frame: From baseline to 2 years]

Change in Patient-reported Symptoms [Time Frame: From baseline to 2 years]

Percent Change in FEV1 [Time Frame: From baseline to 2 years]

Severity of Pulmonary Exacerbations [Time Frame: Over a 2-year period]

Frequency and Intensity of Adverse Events (AEs) [Time Frame: Over a 2-year period]

Change in Exercise Capacity [Time Frame: From baseline to 2 years]

Duration of Pulmonary Exacerbations Relative to Treatment Duration [Time Frame: Over a 2-year period]

Time to First Pulmonary Exacerbation [Time Frame: Over a 2-year period]

Percent Change in DLCO [Time Frame: From baseline to 2 years]

Percent Change in FEV1 Divided by Forced Vital Capacity [Time Frame: From baseline to 2 years]

Annual Rate of Pulmonary Exacerbations [Time Frame: Over a 2-year period]

Percent Change in Percent Predicted FEV1 [Time Frame: From baseline to 2 years]

Secondary ID(s)

1449

2005-003459-12

CE1226_4001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/01/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00261833