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

Tracking Information

Start Date  ICMJEMarch 2006
Estimated Primary Completion DateAugust 2012   (final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 3, 2008)
Lung density as measured by CT [ Time Frame: 5 times during study ] [ Designated as safety issue: No ]
Original Primary Outcome Measures ICMJE 
 (submitted: December 2, 2005)
Lung density as measured by CT
Change HistoryComplete list of historical versions of study NCT00261833 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE 
 (submitted: June 11, 2009)
  • Number, severity, and duration of pulmonary exacerbations [ Time Frame: For the duration of the study ] [ Designated as safety issue: No ]
  • Lung function as measured by forced expiratory volume in 1 second (FEV1) and lung diffusing capacity for carbon monoxide (DLco) [ Time Frame: Once every 3 months during the study ] [ Designated as safety issue: No ]
  • Antigenic and functional serum A1 - PI Levels [ Time Frame: Once every 3 months ] [ Designated as safety issue: No ]
  • Body mass index [ Time Frame: Once every 3 months ] [ Designated as safety issue: No ]
  • Exercise capacity [ Time Frame: Once every 3 months ] [ Designated as safety issue: No ]
  • Quality of Life [ Time Frame: Yearly ] [ Designated as safety issue: No ]
  • Mortality [ Time Frame: Duration of the study ] [ Designated as safety issue: No ]
Original Secondary Outcome Measures ICMJE 
 (submitted: December 2, 2005)
  • Number, severity and duration of exacerbations
  • Lung function as measured by FEV1 and DLco

Descriptive Information

Brief Title  ICMJEZemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor (API) Deficiency
Official Title  ICMJEA 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
Brief Summary

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).

Detailed Description 
Study PhasePhase IV
Study Type  ICMJEInterventional
Study Design  ICMJETreatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Condition  ICMJE
  • Alpha1-proteinase Inhibitor Deficiency
  • Emphysema
Intervention  ICMJE
  • Biological: Alpha1-proteinase inhibitor
    60 mg/kg b.w. i.v. weekly
    Other Name: Zemaira®
  • Other: Placebo
    Lyophilized preparation / 60 mg/kg body weight / weekly
Study Arms / Comparison Groups
  • Zemaira®: Active Comparator
    Intervention: Biological: Alpha1-proteinase inhibitor
  • Placebo: Placebo Comparator
    Intervention: Other: Placebo

Recruitment Information

Estimated Enrollment  ICMJE180
Estimated Completion DateAugust 2012
Estimated Primary Completion DateAugust 2012   (final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Willing to sign informed consent.
  • Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive 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 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.
Ages18 Years to 65 Years
Accepts Healthy VolunteersNo
Contacts  ICMJE
Contact: Central Contact: Clinical Trials Registration Coordinatorclinicaltrials@cslbehring.com
Contact: Xiang Ma(+1) 610-878-4546xiang.ma@cslbehring.com
Location Countries  ICMJEUnited States,   Australia,   Bulgaria,   Canada,   Czech Republic,   Denmark,   Estonia,   Finland,   Germany,   Ireland,   Poland,   Romania,   Russian Federation,   Sweden

Administrative Information

Responsible PartyGlobal Head Clinical Research & Development, CSL Behring
Study ID Numbers  ICMJECE1226_4001, 1449
Study Sponsor  ICMJECSL Behring
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Program Director, Clinical R&DCSL Behring
Information Provided ByCSL Behring