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    Summary
    EudraCT Number:2008-005326-36
    Sponsor's Protocol Code Number:Kamada-AAT(inhaled)-007LocalAmend2
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-04-28
    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 ConcernedGermany - PEI
    A.2EudraCT number2008-005326-36
    A.3Full title of the trial
    A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema
    A.4.1Sponsor's protocol code numberKamada-AAT(inhaled)-007LocalAmend2
    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 SponsorKamada Limited
    B.1.3.4CountryIsrael
    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 supportKamada Limited
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKamada Limited
    B.5.2Functional name of contact pointSponsor
    B.5.3 Address:
    B.5.3.1Street Address7 Sapir Street Science Park, P.O. Box 4081
    B.5.3.2Town/ cityNess-Ziona
    B.5.3.3Post code74140
    B.5.3.4CountryIsrael
    B.5.4Telephone number00972 8 9406472
    B.5.5Fax number00972 8 9406473
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/04/244
    D.3 Description of the IMP
    D.3.1Product nameAerosolized human Alpha-1 Antitrypsin
    D.3.2Product code Kamada-AAT for inhalation
    D.3.4Pharmaceutical form Nebuliser solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 8000047054
    D.3.9.3Other descriptive nameALPHA-1-ANTITRYPSIN
    D.3.10 Strength
    D.3.10.1Concentration unit g/l gram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboNebuliser solution
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Alpha-1 Antitrypsin deficiency in patients with emphysema.
    E.1.1.1Medical condition in easily understood language
    Alpha-1 antitrypsin (AAT) deficiency is a condition in which the body does not make enough of the normal AAT protein that, among others, protects the lungs from damage.
    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 15.1
    E.1.2Level PT
    E.1.2Classification code 10001806
    E.1.2Term Alpha-1 anti-trypsin deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To explore rate and severity of exacerbations in AAT deficient patients when treated with inhaled AAT or placebo. The objective will be evaluated by the primary endpoint of the time from randomization to the first exacerbation with a severity of moderate or severe.
    E.2.2Secondary objectives of the trial
    Secondary objectives in this study are to evaluate the safety profile of inhaled AAT versus placebo and to explore additional parameters that may demonstrate the effect of inhaled AAT on rate and severity of exacerbations in AAT deficient patients.

    The secondary objectives in this study will be evaluated by the following secondary endpoints:
    1. Time to first exacerbation with a severity of mild, moderate or severe.
    2. Severity of the first exacerbation.
    3. Rate of exacerbation episodes. The number of mild, moderate or severe exacerbations per patient during the treatment period.
    4. Safety and tolerability
    a. Safety endpoints:
    i. Adverse Events
    ii. Vital Signs
    iii. Physical Exam
    iv. ECG
    v. Lung Function
    vi. Laboratory Evaluations
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented diagnosis of emphysema
    2. Male or female patients at least 18 years of age.
    3. Able and willing to sign an informed consent.
    4. Patient with record of congenital AAT deficiency of phenotype PiZZ (homozygote) or other rare phenotypes related to AAT deficiency and/or has AAT serum level lower or equivalent to 11 micromole. For patients receiving IV AAT augmentation therapy the serum AAT level threshold does not apply
    5. FEV1/SVC <70% of predicted value post bronchodilator (SVC is slow VC) and FEV1 < 80% of predicted value post‐bronchodilator
    6. History of at least two moderate or severe exacerbations that required change in treatment (e.g. antibiotics, systemic steroids, hospitalization) in the last 18 months prior to date of screening, with at least one of these occurring within the last 12 months prior to screening
    7. Ability to comply with completion of electronic diary.
    8. Ability to self‐administer inhaled AAT using the eFlow® device.
    9. No significant abnormalities in serum hematology, serum chemistry and serum inflammatory / immunogenic markers according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.
    10. No significant abnormalities in urinalysis according to the Principal Investigator's judgment, taking into considerations the potential effects of the AAT deficiency.
    11. No significant abnormalities in ECG per investigator judgment.
    12. Negative for HBsAg and antibodies to HCV, HIV‐1.
    13. AAT deficient patients who are either naïve (not receiving IV augmentation therapy) or AAT deficient patients receiving IV augmentation therapy, if they have been stable on regular therapy for at least 3 months prior to the screening visit and are willing to continue the same regime throughout this trial. Note that only sites in Germany can recruit patients who are currently being treated with IV‐AAT . Patients who stopped IV augmentation treatment 6 months prior to screening date and will not re‐start this treatment for the course of the study will be considered Naïve.
    14. Non‐pregnant, non‐lactating female patients, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator, or who are at least 2 years post‐menopausal or surgically sterilized.
    E.4Principal exclusion criteria
    1. FEV1 ≥ 80% or FEV1 < 20% of predicted value post‐bronchodilator.
    2. FEV1/SVC≥70%
    3. History of lung transplant.
    4. Any lung surgery within the past two years.
    5. On any thoracic surgery waiting list.
    6. End of last exacerbation less than 6 weeks prior to screening/re‐screening visit.
    7. Clinically significant intercurrent illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal or other) that in the opinion of the investigator, could interfere with the safety, compliance or other aspects of this study. Patients with well‐controlled, chronic diseases could be possibly included after consultation with the treating physician and the sponsor.
    8. Active smoking during the last 12 months from screening date.
    9. Pregnancy or lactation.
    10. Woman of child‐bearing potential not taking adequate contraception deemed reliable by the investigator.
    11. Presence of psychiatric/ mental disorder or any other medical disorder which might impair the patient’s ability to give informed consent or to comply with the requirements of the study protocol.
    12. Evidence of ongoing viral infection with HCV, HBV and/or HIV.
    13. Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally prescribed drugs.
    14. IgA Deficiency
    15. History of life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
    16. Participation in another clinical trial within 30 days prior to baseline visit.
    17. Inability to attend scheduled clinic visits and/or comply with the study protocol.
    18. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.
    E.5 End points
    E.5.1Primary end point(s)
    The time from randomization to the first event‐based exacerbation with a severity of moderate or severe. If patients do not have a moderate or severe exacerbation by their last scheduled dose of study drug (Week 50) the time to exacerbation will be considered censored on that date. If a patient discontinues from the study prior to Week 50 with no moderate or severe exacerbation, his time to first exacerbation will be censored on the date of his last dose of study drug.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Randomisation to week 50.
    E.5.2Secondary end point(s)
    1. Time to first event based exacerbation with a severity of mild, moderate or severe.
    2. Severity of the first event based exacerbation.
    3. Rate of event based exacerbation episodes. The number of mild, moderate or severe event based exacerbations per patient during the treatment period.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Randomisation to week 50.
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA7
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    See protocol.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    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.1.3Elderly (>=65 years) Yes
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 200
    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 Decision2010-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-07-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-11-27
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