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    Summary
    EudraCT Number:2019-000602-30
    Sponsor's Protocol Code Number:Kamada-AATInhaled008
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2020-06-12
    Trial 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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-000602-30
    A.3Full title of the trial
    A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of “Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%), Followed by a 2-Year Open-Label Extension.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of “Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%), Followed by a 2-Year Open-Label Extension.
    A.3.2Name or abbreviated title of the trial where available
    Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
    A.4.1Sponsor's protocol code numberKamada-AATInhaled008
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04204252
    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 Ltd.
    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 Ltd.
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKamada Ltd.
    B.5.2Functional name of contact pointSharon Castro
    B.5.3 Address:
    B.5.3.1Street Address2 Holtzman St, Science Park
    B.5.3.2Town/ cityRehovot
    B.5.3.3Post code7670402
    B.5.3.4CountryIsrael
    B.5.4Telephone number+ 972722211600
    B.5.6E-mailnaveht@kamada.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/04/244
    D.3 Description of the IMP
    D.3.1Product nameKamada -AAT for Inhalation or inhaled AAT (AAT)
    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.3.8INN - Proposed INNAlpha-1 Antitrypsin (AAT)
    D.3.9.1CAS number 9041-92-3
    D.3.9.2Current sponsor codeKamada-AAT
    D.3.9.4EV Substance CodeSUB197026
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 Yes
    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
    Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%), and with no history of two or more moderate or one or more severe exacerbations of COPD during the past year.
    E.1.1.1Medical condition in easily understood language
    Treatment and management of adult patients with congenital Alpha Antitrypsin Deficiency.
    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 20.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    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
    Primary Objective (Double Blind)
    To assess the efficacy of Kamada-AAT for Inhalation administered at a dose of 80 mg daily vs. placebo, with efficacy measured by FEV1 post bronchodilator change from baseline at 104 weeks

    OLE Objectives
    1. To assess the long-term safety of Kamada-AAT for Inhalation for up to
    208 weeks of treatment.
    2. To assess the long-term efficacy of Kamada-AAT for Inhalation, as
    measured by FEV1 post bronchodilator for up to 208 weeks of treatment.
    3. To assess the long-term efficacy of Kamada-AAT for Inhalation, as
    measured by CT densitometry change for up to 208 weeks of treatment.
    E.2.2Secondary objectives of the trial
    Secondary Objectives
    To assess the efficacy of Kamada-AAT for Inhalation administered at a dose of 80 mg daily vs placebo as measured by computed tomography (CT) densitometry change from baseline at 104 weeks.
    Safety Objective
    1. To assess the safety of Kamada-AAT for Inhalation administered at a dose of 80 mg daily vs placebo
    2. To assess the immunogenicity of Kamada-AAT for Inhalation and characterize the effect of Anti-drug antibodies (ADA) on drug levels in plasma.

    First (Safety) Cohort Objective
    To assess the safety of Kamada-AAT for Inhalation administered at a dose of 80 mg vs placebo once daily during the first 24 weeks of treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Double-Blind Period
    1. Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes confirmed by genotype blood test documented prior to screening.
    2. Serum AAT levels ≤ 11 µM at screening.
    3. Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC≤70%) at screening.
    4. 40% ≤ FEV1 ≤ 80% of predicted post-bronchodilator at screening.
    5. Patients who are either naïve or washed out of any AAT treatment (by IV) for at least 8 weeks prior to randomization.
    6. Age between 18 to 65 years inclusive at screening.
    7. Able to read and sign informed consent and willing to participate in the study.
    8. Males or non-pregnant, non-lactating females whose screening pregnancy test is negative, who are using contraceptive methods deemed reliable by the investigator, who are post-menopausal, or are surgically sterilized.
    9. Study medication use for at least 20 out of the 28 days of run-in, as
    recorded in the study nebulization PARI Track data.
    10. Demonstrated ability to complete eDiary for at least 20 out of the
    first 28 days of run-in.

    Open-Label Period
    1. Patients who completed 104 weeks of DB study treatment and
    attended the end of treatment visit.
    2. Patients who completed the DB period and attended follow-up visits
    are eligible for the OLE provided that they comply with all other OLE
    eligibility criteria.
    3. Consenting to continue study participation in the OLE phase.
    4. Agree to continue using contraceptive methods deemed reliable by the
    investigator for an additional 2 years, unless post-menopausal or
    surgically sterilized.
    E.4Principal exclusion criteria
    Double-Blind Period
    1. Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels<0.05 g/L at screening.
    2. History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.
    3. Two or more moderate or any severe exacerbation(s) within the year prior to the baseline visit.
    4. A moderate exacerbation within 6 weeks prior to the baseline.
    5. Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).
    6. Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal, or other. Patients might be included after consultation with the treating physician and the sponsor if, in the opinion of the Investigator, their condition will not interfere with the safety, compliance or other aspects of this study.
    7. Hospitalization for any cause 6 weeks prior to screening.
    8. History of lung or liver transplant.
    9. On any thoracic or hepatic surgery waiting list.
    10. Any lung surgery within the past two years (including bronchoscopic lung volume reduction).
    11. Any smoking within the year prior to screening.
    12. Evidence of alcohol abuse or history of alcohol abuse, or use of illegal drugs and/or abuse of legally prescribed drugs in the last 5 years prior to screening.
    13. Acute or chronic hepatitis (hepatitis A, hepatitis B, hepatitis C,) or positive human immunodeficiency virus (HIV) serology.
    14. Signs of significant abnormalities in serum hematology, serum chemistry, serum inflammatory / immunogenic markers and urinalysis per investigator judgment, taking into considerations the potential effects of the AAT deficiency.
    15. Signs of significant abnormalities in ECG per investigator judgment at screening.
    16. Presence of psychiatric/ mental disorder or any other medical disorder that might impair the patient’s ability to give informed consent or to comply with the requirements of the study protocol. If, in the opinion of the Investigator, the condition will not interfere with the compliance or other aspects of this study, the patient might be included after consultation with the treating physician and the sponsor.
    17. Previous exposure to inhaled AAT.
    18. Participation in another clinical trial involving investigational medication or interventional treatment within 30 days and/or last dose 5 half-lives prior to screening visit.
    19. Inability to attend scheduled clinic visits and/or comply with study protocol.
    20. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.

    Open-Label Period
    1. Any adverse event(s) in the DB period and/or medical condition that,
    in the opinion of the investigator, might prevent the patient from safely
    participating in the OLE period of the study, including but not limited to:
    a. Occurrence of a life-threatening allergy, anaphylactic reaction, or
    systemic response to human plasma derived products.
    b. Received lung transplant, entered a waiting list for lung
    transplantation, or underwent lung surgery.
    The investigator should consult the sponsor before inclusion of any
    patient with a significant condition if the investigator believes that it will
    not pose an unacceptable risk for the patient.
    2. Evidence of alcohol abuse or history of alcohol abuse or illegal and/or
    legally prescribed drugs, within the DB study or since the DB study.
    3. Any smoking within the DB study or since the DB study.
    4. Pregnancy or lactation.
    5. Participation in another clinical trial since termination of participation
    in the DB period.
    6. Inability to attend scheduled clinic visits and/or comply with study
    protocol.
    7. 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)
    DB period
    FEV1 (L) post bronchodilator change from baseline at 104 weeks.

    OLE period
    1. FEV1 (L) post bronchodilator change from DB baseline at OLE 104
    weeks (total 208 weeks), and from OLE baseline at OLE 104 weeks (total
    104 weeks of open label treatment), stratified by treatment during the
    DB part.
    2. Change from DB at OLE 104 weeks (total 208 weeks of treatment),
    and from OLE baseline at OLE 104 weeks (total 104 weeks), in CT
    densitometry whole-lung 15th percentile lung density (PD15) at total
    lung capacity (TLC), stratified by treatment during the DB part.
    3. Change from OLE baseline over 104 weeks of open-label treatment in
    Post bronchodilator FEV1 % of predicted, FEV1/FVC %, as well as in post
    bronchodilator volumes (body plethysmography) and diffusion (DLCO),
    stratified by treatment during the DB part.
    4. BODE index score and 6MWT and MMRC dyspnea score from OLE
    baseline over 104 weeks of open-label treatment stratified by treatment
    during the DB part.
    5. Quality of life score as measured by the COPD assessment tool (CAT)
    and EQ-5D-5L from OLE baseline over 104 weeks of open-label
    treatment, stratified by treatment during the DB part.
    6. Desmosine level in plasma from OLE baseline over 104 weeks of open
    label
    treatment, stratified by treatment during the DB part.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at 104 weeks
    E.5.2Secondary end point(s)
    DB Period
    1. Change from baseline over 104 weeks of treatment in CT densitometry whole-lung 15th percentile lung density (PD15) at total lung capacity (TLC).
    2. Change from baseline over 104 weeks of treatment in Post bronchodilator spirometry measures
    a. FEV1 % of predicted
    b. FEV1/FVC %
    3. Exacerbations; annual rate by severity and duration.
    4. Change from Baseline over 104 weeks of treatment in 6 minute walk test (6MWT).
    E.5.2.1Timepoint(s) of evaluation of this end point
    at 104 weeks
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Long Term safety
    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 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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Prospective study, Followed by a 2-Year Open-Label Extension
    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 EEA5
    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
    New Zealand
    Australia
    United Kingdom
    Belgium
    Finland
    Ireland
    Netherlands
    Sweden
    E.8.7Trial has a data monitoring committee Yes
    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 years11
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years11
    E.8.9.2In all countries concerned by the trial months0
    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: 220
    F.1.3Elderly (>=65 years) No
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 125
    F.4.2.2In the whole clinical trial 220
    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)
    None
    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 Decision2020-06-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-23
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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