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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43881   clinical trials with a EudraCT protocol, of which   7295   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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    EudraCT Number:2008-005326-36
    Sponsor's Protocol Code Number:Kamada-AAT(inhaled)-007
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-08-21
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    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.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial looking at the safety and effectiveness of a new drug for
    inhalation, looking to treat Alpha-1 Antitrypsin Deficient Patients with
    A.4.1Sponsor's protocol code numberKamada-AAT(inhaled)-007
    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.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.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.5.4Telephone number+972-8-9406472
    B.5.5Fax number+972-8-9406473
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 17.1
    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 during a 50-week double-blind treatment period. 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 during the 50‐week double‐blind treatment period. In addition, to evaluate the safety, tolerability of inhaled AAT during the 50 weeks open label extension period.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For the double-blind period:
    1. Diagnosis of emphysema confirmed by CT scan. If a report of past CT scan is not available at site documenting diagnosis then a CT scan is to be performed at screening
    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 with 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 (. 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.

    For open-label extension period:
    1. Patients who have completed the 50-week treatment period in the double-blind study as per the protocol.
    2. Patients who have already terminated their participation in the double‐blind period prior to the time of implementation of the OL extension period in the protocol, provided that the patient:
    a. received study treatment for at least 6 months in the double‐blind study
    b. did not prematurely discontinue from the study due to lost to follow-up or non‐compliance with study requirements.
    3. For women of childbearing potential, negative pregnancy test and use of contraceptive methods deemed reliable by the investigator. Women are not considered to be of childbearing potential if they are at least 2 years postmenopausal or surgically sterilized
    E.4Principal exclusion criteria
    For the double-blind period:
    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.

    For the open-label extension period:
    1. Clinically significant adverse event(s) in the double‐blind period or clinically significant intercurrent illness after termination of participation in the doubleblind period that, in the opinion of the investigator, might prevent the patient from safely participating in the OL extension period of the study.
    2. A clinically significant deterioration in lung function that, in the opinion in the investigator, might prevent the patient from safely participating in the OL extension period of the study.
    3. Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally prescribed drugs.
    4. Occurrence of a life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
    5. Received lung transplant or underwent lung surgery since termination of participation in the double blind study.
    6. Active smoking.
    7. Pregnancy or lactation.
    8. Women of childbearing potential not taking adequate contraception deemed reliable by the investigator.
    9. Participation in another clinical trial since termination of participation in the double‐blind period of the study.
    10. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol or would jeopardize the safety of the patient.
    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 during the double-blind treatment period. If patients do not have a moderate or severe exacerbation by their last scheduled dose of study drug during the double-blind period, 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 54.
    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 54.
    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. 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 years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days14
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial days14
    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 state80
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 200
    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)
    See protocol.
    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 Decision2009-08-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-02-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-12-11
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