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    Summary
    EudraCT Number:2019-000826-22
    Sponsor's Protocol Code Number:CBLZ945C12201
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Restarted
    Date on which this record was first entered in the EudraCT database:2019-08-02
    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 number2019-000826-22
    A.3Full title of the trial
    An open-label, adaptive design study in patients with amyotrophic lateral sclerosis (ALS) to characterize safety, tolerability and brain microglia response, as measured by TSPO binding, following multiple doses of BLZ945 using positron emission tomography (PET) with the radioligand [11C]-PBR28
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of safety and of the mechanism of BLZ945 in amyotrophic lateral sclerosis (ALS) patients.
    A.4.1Sponsor's protocol code numberCBLZ945C12201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04066244
    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 SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Sverige AB
    B.5.2Functional name of contact pointMedical information
    B.5.3 Address:
    B.5.3.1Street AddressBox 1218
    B.5.3.2Town/ cityKista
    B.5.3.3Post code164 28
    B.5.3.4CountrySweden
    B.5.4Telephone number+46 8 7323200
    B.5.6E-mailmedinfo.se@novartis.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code BLZ945
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet defined
    D.3.9.2Current sponsor codeBLZ945
    D.3.9.3Other descriptive nameBLZ945
    D.3.9.4EV Substance CodeSUB182305
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) 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 No
    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.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.3.2Product code BLZ945
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot yet defined
    D.3.9.2Current sponsor codeBLZ945
    D.3.9.3Other descriptive nameBLZ945
    D.3.9.4EV Substance CodeSUB182305
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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) 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 No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic lateral sclerosis (ALS)
    E.1.1.1Medical condition in easily understood language
    Nervous system disease that attacks nerve cells called neurons in the brain and spinal cord, causing muscles problems.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system 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 evaluate brain microglial response, as measured by TSPO binding
    following dosing with BLZ945 in ALS subjects
    E.2.2Secondary objectives of the trial
    - To characterize the pharmacokinetics (PK) of BLZ945 in ALS subjects
    - To evaluate safety and tolerability of BLZ945 in ALS subjects
    - To assess the CYP2C8 pharmacogenomic-pharmacokinetic relationship
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able to communicate well with the investigator, to understand and
    comply with the study visits and procedures of the study.
    2. Written informed consent must be obtained before any assessment is
    performed.
    3. Male and female subjects at least 18 year old , diagnosed with
    familial or sporadic ALS according to the World Federation of Neurology
    Revised El Escorial criteria of either bulbar or limb onset.
    4. Able to swallow medication capsules, in the opinion of the
    investigator.
    5. Disease duration from symptoms onset no longer than 48 months at
    the screening visit.
    6. Having a SVC (slow vital capacity) equal to or more than 60%
    predicted normal value per local standards for gender, height, and age at
    the screening visit.
    7. Females of childbearing potential must have a negative pregnancy
    test at screening and baseline.
    8. High-affinity binders (HAB) or mixed-affinity binders (MAB) to TSPO
    as evaluated by genotyping for the rs6971 polymorphism in the TSPO
    gene at the screening visit.
    9. Baseline PET scan of sufficient image quality, as determined locally by
    the PET experts, to enable the measurement of [11C]-PBR28 volume of distribution (Vt) in the relevant CNS regions.
    10. Treatment with riluzole and/or edaravone are allowed but subjects
    must be on a stable dose and regimen for at least 3 months prior to
    baseline. For subjects taking edaravone, BLZ945 dosing must be
    scheduled during the 20 days off-drug period of the edavarone treatment
    regimen.
    11. An Upper Motor Neuron Burden (UMNB) scale >25 at the screening
    visit
    12. BMI between 18 – 35 kg/m2 at the screening visit.
    E.4Principal exclusion criteria
    1. A history of clinically significant ECG abnormalities
    2. Active hematologic, hepatic, respiratory disorders that are clinically
    significant and may jeopardize the patient's safety if participating in the
    study or limit his/her participation in the study, including ability to
    tolerate the imaging studies.
    3. Active dementia, neurologic diseases other than ALS, or psychiatric
    illness that in the opinion of the investigator would limit their
    participation in the current study .
    4. Use of other investigational drugs within 5 half-lives of screening, or
    until the expected PD effect has returned to baseline , whichever is
    longer; or longer if required by local regulations.
    5. History of hypersensitivity to any of the study treatments or
    excipients or to drugs of similar chemical classes.
    6. Presence of human immunodeficiency virus (HIV) infection based on
    screening lab results .
    7. Evidence of active or latent tuberculosis as assessed by Quantiferon
    testing at the screening visit.
    8. Positive serology for hepatitis B surface antigen, or hepatitis C
    antibodies confirmed by an appropriate licensed test at screening.
    9. Signs or symptoms, in the judgement of the investigator, of a clinically
    significant systemic viral, bacterial or fungal infection within 30 days
    prior to the screening visit.COVID-19 specifically: PCR testing for COVID-19 will be completed Prior
    to first dosing.
    10. Cardiac disorders, such as recent cardiac history (within 6 months of
    screening) of acute coronary syndrome, acute heart failure, or
    significant ventricular arrhythmia at the screening visit. Patient with
    cardiac failure class 3 or 4 of the NYHA classification. Patients with
    implanted cardiac pacemaker, or defibrillator.
    11. Significant haematological laboratory abnormalities.
    12. Clinical evidence of liver disease or liver injury or any of the
    following hepatic conditions at the screening visit
    13. Women of child-bearing potential, defined as all women
    physiologically capable of becoming pregnant, unless they are using
    highly effective methods of contraception during dosing and for 14 days
    after last dose of BLZ945.
    14. Pregnant or nursing female subjects
    15. Sexually active males unless they use a condom during intercourse
    while taking the drug during treatment, for 14 days after stopping
    BLZ945 and should not father a child in this period.
    16. Intentionally left blank : removed in amendment 02
    17. Any contraindications to MRI
    18. Taking medications prohibited by the protocol
    19. Any contraindications to the arterial line sampling
    20. History or presence of impaired renal function at the screening visit.
    21. Active suicidal ideation
    22. History of drug abuse or harmful alcohol use within the 12 months
    prior to dosing within the judgement of the investigator, or evidence of
    such abuse as indicated by the laboratory assays conducted during
    screening.
    23. Inability or unwillingness to undergo repeated venipuncture or arterial cannulation or in the opinion of the investigator, patient would
    be at increased risk for adverse events related to these procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline - Volume of distribution (Vt) in different brain regions for each [11C]-PBR28 PET scan
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day -28 up to Day 22
    E.5.2Secondary end point(s)
    - Plasma Pharmacokinetics (PK) of BLZ945 (Cmax, Tmax, AUC, T1/2)
    - Renal Clearance (CLR) of BLZ945
    - CYP2C8 genotyping and BLZ945 plasma PK parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Day 1; up to Day 17
    - Day 1; up to Day 7
    - Day 1; up to Day 17
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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
    Adaptive design (multiple ascending dose study)
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA2
    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
    Finland
    Sweden
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days5
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    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: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 20
    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 Decision2019-09-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-16
    P. End of Trial
    P.End of Trial StatusRestarted
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