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    The EU Clinical Trials Register currently displays   43883   clinical trials with a EudraCT protocol, of which   7296   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2007-007129-38
    Sponsor's Protocol Code Number:CE1226_3001
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-12-22
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedCzechia - SUKL
    A.2EudraCT number2007-007129-38
    A.3Full title of the trial
    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
    Otevřená, nekontrolovaná, multicentrická studie ke zhodnocení účinnosti a bezpečnosti podávání přípravku Zemaira® ke chronické rozšiřovací a udržovací terapii u pacientů s emfyzémem v důsledku nedostatečnosti alfa1-proteázového inhibitoru, kteří dokončili klinickou studii CE1226_4001
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical research study to assess the safety and effectiveness of alpha1-proteinase inhibitor (A1-PI) administered by intravenous application to patients with alpha1-proteinase deficiency and emphysema who have completed the 2-year treatment and observation period in the Phase III/IV Zemaira® CE1226_4001.
    A.4.1Sponsor's protocol code numberCE1226_3001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCSL Behring GmbH
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring GmbH
    B.5.2Functional name of contact pointGlobal Project Lead
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Str. 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.4Telephone number+49 6421 39-2929
    B.5.5Fax number+49 6421 39-3172
    B.5.6E-mailanja.braeunlich@cslbehring.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAlpha1-Proteinase Inhibitor / Zemaira®
    D.3.2Product code CE1226
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNThere is no recommended INN.
    D.3.9.2Current sponsor codeCE1226
    D.3.9.3Other descriptive nameAlpha1-Proteinase Inhibitor (Human)
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chronic augmentation and maintenance therapy in individuals with alpha1-proteinase inhibitor (A1-PI) deficiency and clinical evidence of emphysema.
    E.1.1.1Medical condition in easily understood language
    Chronic hereditary, genetic disorder in which there is not enough of this protein to prevent lung damage and is associated with progressive, severe emphysema.
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level LLT
    E.1.2Classification code 10001811
    E.1.2Term Alpha-1 proteinase inhibitor deficiency
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the long-term verification of a disease-modifying benefit of Zemaira® on the progression of emphysema, assessed by volume-adjusted lung density, and measured by yearly computed tomography (CT).
    E.2.2Secondary objectives of the trial
    The secondary objectives are to assess the effect of treatment with A1-PI on:
    1. change in symptoms score as assessed by Quality of Life (QoL) using the St. George’s Respiratory Questionnaire (SGRQ);
    2. the assessment of pulmonary function (ie, forced expiratory volume in 1 second [FEV1] and [FEV1/forced vital capacity{FVC}]);
    3. reduction in risk of pulmonary exacerbations assessed by the number and annual rate of exacerbations over the study duration;
    4. antigenic and functional serum A1-PI levels in relation to disease progression, body mass index (BMI), safety, and mortality.
    5. the safety profile of subjects will be assessed throughout the study.
    The safety of Zemaira® will be assessed by the occurrence of adverse events (AEs).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226_4001 study and are willing to sign informed consent.

    2. Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator.
    E.4Principal exclusion criteria
    1. Individuals residing in the US.

    2. Current evidence of alcohol abuse, or abuse of drugs, such as barbiturates, benzodiazepines, amphetamines, cocaine, opioids, and cannabinoids.

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

    4. Current tobacco smoker (i.e. smoking must be discontinued for at least 6 months prior to study participation).

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

    6. History of non-compliance.

    7. Administration of any other experimental new drug or participation in an investigation of a marketed product.

    8. Inability to perform necessary study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Variable: Annual Rate of Change in adjusted P15 from CT Scans

    The exploratory efficacy variable of primary interest is the annual rate of change, measured by a slope of time and treatment interaction from a mixed effects model, in volume-adjusted lung density. This is assessed by the 15th percentile (P15) of the lung density distribution. The variable of interest is called adjusted P15 measured in g/L. Scans are obtained at total lung capacity (TLC) and functional residual capacity (FRC) inspiration state, and lung volume is assessed for the right, the left, and for both lungs. During the extension study, the CT scans are taken at Month 12 and Month 24 (ie, Month 36 [Visit 5] and Month 48 [Visit 9] when consideration is given to the start of study CE1226_4001). The primary interest of the study will be comparing the annual rate of change in adjusted P15 from CT scans between subjects who had an early start with Zemaira® treatment and subjects who had a late start with treatment over 4 years.
    E.5.1.1Timepoint(s) of evaluation of this end point
    See E.5.1
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Variable: Absolute and % Change in Adjusted P15 from Baseline to Month 48

    The absolute and % change from baseline to the Month 48 will be derived using the values of adjusted P15 of the last assessment of CT scans from study CE1226_3001 and the first assessment of CT scans from study CE1226_4001.
    E.5.2.1Timepoint(s) of evaluation of this end point
    See E.5.2
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    Czech Republic
    Denmark
    Estonia
    Finland
    Germany
    Ireland
    Poland
    Romania
    Sweden
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 125
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 98
    F.4.2.2In the whole clinical trial 140
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Supply of Zemaira® on named-patient basis from the U.S. or, alternatively, use of competitor products
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2010-01-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-04-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-09-09
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