Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00670007
First received: April 29, 2008
Last updated: August 6, 2014
Last verified: July 2014
  Purpose

This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® i.v. administration in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The long-term verification of a disease-modifying benefit of Zemaira® on the progression of emphysema will be assessed by volume-adjusted lung density, measured yearly by computed tomography (CT).


Condition Intervention Phase
Emphysema
Alpha 1-proteinase Inhibitor Deficiency
Biological: Alpha1- proteinase inhibitor [human]
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Non-controlled, Multicenter, Multinational Study to Evaluate the Efficacy and Safety of Zemaira® Administration in Chronic Augmentation and Maintenance Therapy in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency Who Completed Clinical Study CE1226_4001

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Rate of change of lung density [ Time Frame: Yearly, for up to 2 years ] [ Designated as safety issue: No ]
    The annualized rate of change of lung density measured by volume-adjusted CT.


Secondary Outcome Measures:
  • Number of pulmonary exacerbations [ Time Frame: For the duration of the study, up to 2 years ] [ Designated as safety issue: No ]
  • Lung function as measured by forced expiratory volume in 1 second (FEV1) [ Time Frame: Once every 3 months for the duration of the study, up to 2 years. ] [ Designated as safety issue: No ]
  • Change in adjusted lung density [ Time Frame: Baseline, and completion visit (up to 2 years) ] [ Designated as safety issue: No ]
    Absolute change from baseline to the completion visit. The baseline value will be the last assessment from the preceding study CE1226_4001.

  • Percent change in adjusted lung density [ Time Frame: Baseline, and completion visit (up to 2 years) ] [ Designated as safety issue: No ]
    Percent change in adjusted lung density from baseline to the completion visit. The baseline value will be the last assessment from the preceding study CE1226_4001.

  • Quality of Life (QoL) [ Time Frame: At 12 and 24 months ] [ Designated as safety issue: No ]
    QoL will be assessed using the disease-specific St. George's Respiratory Questionnaire (SGRQ)

  • Lung function as measured by ratio of FEV1/FVC (forced vital capacity) [ Time Frame: Once every 3 months for the duration of the study, up to 2 years. ] [ Designated as safety issue: No ]
  • Annual rate of pulmonary exacerbations [ Time Frame: For the duration of the study, up to 2 years ] [ Designated as safety issue: No ]
  • Time to first onset of pulmonary exacerbation [ Time Frame: For the duration of the study, up to 2 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 140
Study Start Date: April 2008
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Zemaira Biological: Alpha1- proteinase inhibitor [human]
Lyophilized preparation of 60 mg/kg body weight intravenously once per week
Other Name: Zemaira®

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226_4001 study (NCT00261833) and are 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

Exclusion Criteria:

  • Individuals residing in the US
  • Current evidence of alcohol abuse or abuse of 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
  • Current tobacco smoker (smoking must be discontinued for at least 6 months prior to study participation)
  • 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
  • Inability to perform necessary study procedures
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00670007

  Show 21 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director, Clinical R&D CSL Behring
  More Information

Additional Information:
No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00670007     History of Changes
Other Study ID Numbers: CE1226_3001, 1466, 2007-007129-38
Study First Received: April 29, 2008
Last Updated: August 6, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Canada: Ethics Review Committee
Ireland: Irish Medicines Board
Ireland: Medical Ethics Research Committee
Finland: Ethics Committee
Australia: Department of Health and Ageing Therapeutic Goods Administration
Finland: Finnish Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Germany: Paul-Ehrlich-Institut
Czech Republic: Ethics Committee
Czech Republic: State Institute for Drug Control
Denmark: Danish Medicines Agency
Estonia: The State Agency of Medicine
Romania: National Medicines Agency
Sweden: Medical Products Agency

Keywords provided by CSL Behring:
Alpha1-proteinase inhibitor deficiency
Chronic augmentation and maintenance therapy
Emphysema
Emphysema due to Alpha 1-proteinase inhibitor deficiency

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Emphysema
Pulmonary Emphysema
Pathologic Processes
Pulmonary Disease, Chronic Obstructive
Lung Diseases, Obstructive
Lung Diseases
Respiratory Tract Diseases
Liver Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Protease Inhibitors
Alpha 1-Antitrypsin
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Trypsin Inhibitors
Serine Proteinase Inhibitors

ClinicalTrials.gov processed this record on September 18, 2014