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    Summary
    EudraCT Number:2013-001870-38
    Sponsor's Protocol Code Number:GTi1201
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-09-23
    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 ConcernedSweden - MPA
    A.2EudraCT number2013-001870-38
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alphal-Proteinase Inhibitor (Human) in Subjects with Pulmonary Emphysema due to Alphal-Antitrypsin Deficiency
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency
    A.3.2Name or abbreviated title of the trial where available
    SPARTA
    A.4.1Sponsor's protocol code numberGTi1201
    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 SponsorGrifols Therapeutics Inc.
    B.1.3.4CountryUnited States
    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 supportGrifols Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGrifols Therapeutics Inc.
    B.5.2Functional name of contact pointRhonda Griffin-Ass.Director,ClinDev
    B.5.3 Address:
    B.5.3.1Street Address79 T.W. Alexander Drive, Research Triangle Park,
    B.5.3.2Town/ city4101 Research Commons,
    B.5.3.3Post codeNC 27709
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 919-316-6693
    B.5.5Fax number+1 919-287-2089
    B.5.6E-mailrhonda.griffin@grifols.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 Yes
    D.2.1.1.1Trade name Prolastin-C
    D.2.1.1.2Name of the Marketing Authorisation holderGrifols Therapeutics Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 (Human)
    D.3.2Product code Prolastin-C
    D.3.4Pharmaceutical form Powder for infusion
    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 INNAlpha1-Proteinase Inhibitor (Human)
    D.3.9.1CAS number 9041-92-3
    D.3.9.2Current sponsor codeTAL6004
    D.3.9.3Other descriptive nameALPHA-1-ANTITRYPSIN
    D.3.9.4EV Substance CodeSUB12796MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number47
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pulmonary Emphysema due to Alpha-1-Antitrypsin Deficiency
    E.1.1.1Medical condition in easily understood language
    Severe deficiency of alpha-1 antitrypsin, complicated by emphysema of the lungs
    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 LLT
    E.1.2Classification code 10014563
    E.1.2Term Emphysema pulmonary
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to demonstrate, using whole lung computed tomography (CT) densitometry (15th percentile point [PD15]), a slower progression of lung tissue loss in subjects with alpha1-antitrypsin deficiency (AATD) on Alpha-1 MP (alpha1-proteinase inhibitor [human], modified process) treatment (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to demonstrate the efficacy of each IV dose of Alpha-1 MP (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo in subjects with AATD by:

    - Reducing the incidence of severe COPD exacerbations as defined by an American Thoracic Society (ATS)/European Respiratory Society (ERS) Task Force criteria, and

    - Slowing the progression of lung tissue loss as measured by basal lung CT densitometry (PD15).

    The safety objective of this study is to evaluate the safety and tolerability of two separate IV dose regimens (60 mg/kg/week and 120 mg/kg/week) of Alpha-1 MP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Have a documented total alpha1-PI serum level < 11 μM.

    2. Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or “at-risk” alleles. If the genotype has yet to be documented, genotyping (i.e., allelic discrimination) and phenotyping will be performed at the Screening (Week -3) Visit.

    3. At the Screening (Week -3) Visit, have a post-bronchodilator FEV1 ≥ 30% and < 80% of
    predicted and FEV1/forced vital capacity (FVC) < 70% (Global Initiative for Chronic
    Obstructive Lung Disease [GOLD] stage II or III).

    4. Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator’s judgment.

    5. Have clinical evidence of pulmonary emphysema per the Investigator’s judgment.
    E.4Principal exclusion criteria
    1. Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening (Week -3) Visit.
    2. Has received alpha1-PI augmentation therapy within one month of the Screening (Week -3) Visit.
    3. Has had a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase. Investigator discretion should be used to determine if a subject is appropriate for study participation if the subject has had a COPD exacerbation which occurred more than 5 weeks prior to the Screening (Week -3) Visit.
    4. Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
    5. History of lung or liver transplant.
    6. Any lung surgery during the past 2 years (excluding lung biopsy).
    7. On the waiting list for lung surgery, including lung transplant.
    8. Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking.
    9. History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
    10. Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e.,
    10 mg every 2 days) within the 5 weeks prior to the Screening (Week -3) Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase. It is recommended to maintain the same dose throughout the study.
    11. Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase.
    12. Known selective or severe Immunoglobulin A (IgA) deficiency.
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective of this study is to demonstrate, using whole lung computed tomography (CT) densitometry (15th percentile point [PD15]), a slower progression of lung tissue loss in subjects with alpha1-antitrypsin deficiency (AATD) on Alpha-1 MP (alpha1-proteinase inhibitor [human], modified process) treatment (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    weeks 52, 104,130 & 156
    E.5.2Secondary end point(s)
    The secondary objectives of this study are to demonstrate the efficacy of each IV dose of Alpha-1 MP (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo in subjects with AATD by:

    - Reducing the incidence of severe COPD exacerbations as defined by an American Thoracic Society (ATS)/European Respiratory Society (ERS) Task Force criteria, and

    - Slowing the progression of lung tissue loss as measured by basal lung CT densitometry (PD15).

    The exploratory objectives for this study are to evaluate the effects of each IV dose of Alpha-1 MP (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo in subjects with AATD on:
    - The incidence and severity of COPD exacerbations as defined by ATS/ERS criteria,
    - Change from baseline in forced expiratory volume in 1 second (FEV1), and
    - Health-related quality of life as measured by the Saint George’s Respiratory Questionnaire (SGRQ).

    CT scan parameters other than basal and whole lung PD15 may also be evaluated.

    An additional exploratory objective is to compare the efficacy of 120 mg/kg/week Alpha-1 MP to 60 mg/kg/week Alpha-1 MP as evidenced by the change from baseline in whole lung PD15 using CT densitometry.

    The safety objective of this study is to evaluate the safety and tolerability of two separate IV dose regimens (60 mg/kg/week and 120 mg/kg/week) of Alpha-1 MP.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study (please ref. ATS/ERS COPD Exacerbation Assessment row in Appendix1: study flow chart - page 64)
    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 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 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 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 trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Argentina
    New Zealand
    Australia
    Brazil
    Canada
    United States
    Denmark
    Estonia
    Finland
    France
    Ireland
    Poland
    Romania
    Slovakia
    Spain
    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
    week 160
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years12
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years13
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days27
    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: 305
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 34
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 230
    F.4.2.2In the whole clinical trial 339
    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)
    Standard of care
    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 Decision2013-09-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-01-09
    P. End of Trial
    P.End of Trial StatusOngoing
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