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    Summary
    EudraCT Number:2009-018157-23
    Sponsor's Protocol Code Number:D0490C00014
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2010-07-07
    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 ConcernedAustria - BASG
    A.2EudraCT number2009-018157-23
    A.3Full title of the trial
    A Phase IIa, Multi-Centre, Double-Blind, Randomised, Placebo-Controlled, Parallel Group, 12-Month Treatment, Adaptive Design Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of AZD3241 in Patients with Multiple System Atrophy
    A.4.1Sponsor's protocol code numberD0490C00014
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAstraZeneca AB
    B.1.3.4CountrySweden
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameAZD3241
    D.3.2Product code AZD3241
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeAZD3241
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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.IMP: 2
    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 nameAZD3241
    D.3.2Product code AZD3241
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeAZD3241
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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 placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Multiple system atrophy
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.1
    E.1.2Level LLT
    E.1.2Classification code 10064060
    E.1.2Term Multiple system atrophy
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of AZD3241 on disease progression in patients with MSA using scales covering historical review, motor function, autonomic examination, and global disability.
    For Stage 1, the primary objective is to evaluate the safety and tolerability of AZD3241 in order to select a dose for treatment of MSA patients in Stage 2.
    E.2.2Secondary objectives of the trial
    1. To evaluate the effect of AZD3241 on brain neurodegeneration using magnetic resonance imaging (MRI)

    Safety objective:
    To evaluate the safety and tolerability of AZD3241.

    Exploratory objectives:
    1. To explore the pharmacokinetics (PK) of AZD3241
    2. To explore the effects of AZD3241 on α synuclein levels in plasma
    3. To evaluate the effect of AZD3241 on myeloperoxidase (MPO) activity in plasma
    4. To collect and store DNA samples that may be used for future, exploratory genetic research aimed at identifying/exploring genetic variations that may affect PK and pharmacodynamics (including biomarkers), safety, and tolerability related to AZD3241. In addition, susceptibility genes and genes related to underlying disease may be explored. The results of the pharmacogenetic analyses will be reported separately from the Clinical Study Report (CSR).

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of informed consent prior to any study-specific procedures
    2. Diagnosis of probable or possible MSA in accordance with consensus criteria (Gilman et al 2008) excluding patients with MSA-C without Parkinsonism

    Criteria for the diagnosis of probable MSA
    - Autonomic failure involving urinary incontinence (inability to control the release of urine from the bladder, with erectile dysfunction in males) or an orthostatic decrease of blood pressure within 3 minutes of standing by at least 30 mm Hg systolic or 15 mm Hg diastolic, and
    - Poorly levodopa-responsive Parkinsonism (bradykinesia with rigidity, tremor, or postural instability), or
    - A cerebellar syndrome (gait ataxia with cerebellar dysarthria, limb ataxia, or cerebellar oculomotor dysfunction)
    Criteria for the diagnosis of possible MSA
    - Parkinsonism (bradykinesia with rigidity, tremor, or postural instability), or
    - A cerebellar syndrome (gait ataxia with cerebellar dysarthria, limb ataxia, or cerebellar oculomotor dysfunction), and
    - At least 1 feature suggesting autonomic dysfunction (otherwise unexplained urinary urgency, frequency, or incomplete bladder emptying, erectile dysfunction in males, or significant orthostatic blood pressure decline that does not meet the level required in probable MSA), and
    - At least one of the additional features shown below
    Additional features of possible MSA
    Possible MSA-P or MSA-C
    - Babinski sign with hyperreflexia
    - Stridor
    Possible MSA-P
    - Rapidly progressive Parkinsonism
    - Poor response to levodopa
    - Postural instability within 3 years of motor onset
    - Gait ataxia, cerebellar dysarthria, limb ataxia, or cerebellar oculomotor dysfunction
    - Dysphagia within 5 years of motor onset
    - Atrophy on MRI of putamen, middle cerebellar peduncle, pons, or cerebellum
    - Hypometabolism on [18F]fluorodeoxyglucose positron emission tomography (FDG-PET) in putamen, brainstem, or cerebellum
    Possible MSA-C
    - Parkinsonism (bradykinesia and rigidity)
    - Atrophy on MRI of putamen, middle cerebellar peduncle, or pons
    - Hypometabolism on FDG-PET in putamen
    - Presynaptic nigrostriatal dopaminergic denervation on single photon emission computed tomography or positron emission tomography
    3. Females or males aged ≥40 years and ≤75 years. Females must be of non-childbearing potential, confirmed at enrolment by fulfilling any of the criteria:
    - Females >50 years of age who have been amenorrheic for 12 months or more and have not used exogenous hormonal treatment,
    - Females who are permanently or surgically sterilised or postmenopausal. Permanent sterilisation includes hysterectomy and/or bilateral oopherectomy and/or bilateral salpingectomy, but excludes bilateral tubal occlusion. Women will be considered postmenopausal if they are amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:
    (i) Women under 50 years old would be considered postmenopausal if they have been amenorrheic for 12 months or more following cessation of exogenous hormonal treatments and with luteinizing hormone (LH) and follicle stimulating hormone (FSH) levels in the postmenopausal range
    (ii) Women over 50 years of age would be considered postmenopausal if they have been amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments.
    4. Males should be willing to use barrier contraception (ie, condoms) from the first day of dosing until 3 months after the last dose of IP
    5. Treatment for symptoms of MSA (Parkinsonism, autonomic dysfunction, etc) should be stable for at least 1 month prior to enrolment (Visit 1)
    6. UMSARS Part IV ≤3
    7. Documented diagnosis less than 3 years
    8. Patients who are ambulatory
    9. Patient must be able to understand and be willing to comply with study procedures, restrictions and requirements as judged by the Investigator.
    In addition, for inclusion in the optional genetic research (DNA sampling), patients need to fulfil the following criterion:
    1. Provision of informed consent for optional exploratory genetic research.
    E.4Principal exclusion criteria
    1. Patients with: idiopathic Parkinson’s disease, other Parkinson plus syndromes (eg, progressive supranuclear palsy), or secondary Parkinsonism (eg, medication induced)
    2. Patients with speech problems as assessed by a score of ≥3 on UMSARS Part I, Question 1
    3. Patients with swallowing problems as assessed by a score of ≥3 on UMSARS Part I, Question 2
    4. Patients with walking problems as assessed by a score of ≥3 on UMSARS Part I, Question 7
    5. Patients with falling/postural instability problems as assessed by a score of ≥3 on UMSARS Part I, Question 8
    6. Patients with severe orthostatic symptoms as assessed by a score of ≥3 on UMSARS Part I, Question 9
    7. Patients with urinary function problems as assessed by a score of ≥3 on UMSARS Part I, Question 10
    8. Use of monoamine oxidase B (MAO-B) inhibitors, metoclopramide, CYP3A4 inhibitors, or CYP3A4 inducers within 1 month of randomisation; patients receiving such medications at enrolment should undergo a 1-month (4-week) washout period prior to randomisation
    9. Current significant major or unstable respiratory disease, heart disease, cerebrovascular disease, haematological disease, hepatic disease, renal disease, gastrointestinal disease, or other major disease as judged by the Investigator
    10. History of any clinically significant disease or disorder other than MSA which, in the opinion of the Investigator, may either put the patient at risk because of participation in the study, or influence the results or the patient’s ability to participate in the study
    11. Any clinically significant illness, medical/surgical procedure or trauma within 4 weeks prior to the first administration of IP as judged by the Investigator
    12. Significant abnormalities on the clinical examination (excluding findings of MSA), that may interfere with the study or present a safety risk to the patient, as judged by the Investigator
    13. Any clinically significant abnormalities in clinical chemistry, haematology, or urinalysis results as judged by the Investigator
    14. Having known or suspected systemic infection (eg, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, tuberculosis) as judged by the Investigator
    15. Any clinically important abnormalities in rhythm, conduction or morphology of resting ECG that may interfere with the interpretation of QTc interval changes. This includes patients with any of the following:
    - Clinically significant PR (PQ) interval prolongation
    - Intermittent second or third degree atrioventricular block
    - Incomplete, full or intermittent bundle branch block (QRS <110 msec with normal QRS and T wave morphology is acceptable if there is no evidence of left ventricular hypertrophy)
    - Abnormal T wave morphology, particularly in the protocol-defined primary lead
    16. Prolonged QTcF >450 msec or shortened QTcF <340 msec or a family history of long QT syndrome
    17. History of alcohol abuse or excessive intake of alcohol as judged by the Investigator
    18. Known or suspected history of drug abuse as judged by the Investigator
    19. History of hypersensitivity to drugs with a similar chemical structure or class to AZD3241
    20. History of intolerance or hypersensitivity to mannitol
    21. History of severe allergy/hypersensitivity or symptoms/signs of ongoing allergy/ hypersensitivity as judged by the Investigator
    22. History or presence of gastrointestinal, hepatic, or renal disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs
    23. Plasma donation within 1 month of enrolment or any blood donation/blood loss >500 mL during the 3 months prior to enrolment
    24. Participation in another clinical study with an IP during the past 12 weeks that is anticipated to interfere with the objectives of the study as judged by the Investigator
    25. Involvement in the planning and conduct of the study (applies to both AstraZeneca staff, contract research organisation [CRO], or staff at the study site)
    26. Previous randomisation or treatment in present study.
    In addition, any of the following is regarded as a criterion for exclusion from the optional genetic research:
    1. Previous allogenic bone marrow transplant
    2. Non-leukocyte depleted whole blood transfusion within 120 days of the date of the genetic sample collection.
    E.5 End points
    E.5.1Primary end point(s)
    The change from baseline to Month 12 in UMSARS I+II score (for evaluation of primary objective).
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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) No
    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 Yes
    E.8.1.7.1Other trial design description
    Adaptive design
    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.8.3 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA39
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    The end of the study is defined as “the last visit of the last patient undergoing the study.”
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 101
    F.4.2.2In the whole clinical trial 120
    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 Decision2010-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-06-10
    P. End of Trial
    P.End of Trial StatusOngoing
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