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    EudraCT Number:2018-000668-28
    Sponsor's Protocol Code Number:RG_17-250
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-06-25
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-000668-28
    A.3Full title of the trial
    A Phase II pilot safety and tolerability study of ILB in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess safety and acceptability of a treatment in patients with ALS
    A.3.2Name or abbreviated title of the trial where available
    The ALS Study
    A.4.1Sponsor's protocol code numberRG_17-250
    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 SponsorUniversity of Birmingham
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportTikoMed AB
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity of Birmingham, D3B Team, Cancer Research UK Clinical Trials Unit
    B.5.2Functional name of contact pointMiss Claire Potter
    B.5.3 Address:
    B.5.3.1Street AddressInstitute of Translational Medicine (ITM), 5th Floor, Heritage Building, Mindelsohn Way
    B.5.3.2Town/ cityBirmingham
    B.5.3.3Post codeB15 2TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01213718492
    B.5.5Fax number01213717246
    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 nameILB: 100mg/ml solution for injection and infusion
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDextran Sodium Sulfur Salt 5, Mw 5 kDa. a type of Low Molecular Weight Dextran Sulfate (LMW-DS)
    D.3.9.3Other descriptive nameILB
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    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. 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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS)
    E.1.1.1Medical condition in easily understood language
    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 20.0
    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
    The Primary Objective is:

    To determine the safety and acceptability of ILB in ALS patients
    E.2.2Secondary objectives of the trial
    To describe the effect of ILB on:
    Symptoms and quality of life
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients ≥18 years and who have provided written informed consent to participate in the study
    2. Prior to trial entry patients will have a definite diagnosis of ALS according to El Escorial Criteria. All patients will demonstrate either:
     presence of UMN (increased tone, brisk reflexes) as well as LMN (weakness, wasting and fasciculation) signs in the bulbar region and at least two of the other spinal regions (cervical, thoracic or lumbosacral)


     presence of UMN and LMN signs in all three spinal regions (cervical, thoracic or lumbosacral)

    3. Electrophysiological tests (Electromyography (EMG) / Nerve Conduction Study (NCS)) that supports the diagnosis of Motor Neurone Disease (MND)
    4. Forced Vital Capacity (FVC) ≥50% of predicted value for gender, height and age at screening and a mean Sniff Nasal Inspiratory Pressure (SNIP) ≥50% of predicted value for age
    5. Adequate haematological function (Hb≥10g/dl), absolute neutrophil count ≥1.5x109/L and a platelet count ≥60 x109/L
    6. International Normalised Ratio (INR) ≤ 1.5, aPTT 30 – 40 seconds, PT 11-13.5 seconds
    7. Patient willing and able to comply with schedule visits, treatment plan and other study procedures.
    8. Patients taking Riluzole must have discontinued treatment ≥28 days prior to study entry (and following consent to take part in the study)
    9. Women Of Child Bearing Potential (WOCBP) who agree to use highly effective means of contraception (as defined in the Heads of Medicines Agencies_Clinical Trials Facilitation Group (HMA_CTFG) guideline for the entirety of the study
    E.4Principal exclusion criteria
    1. Patients classified as either probable or possible ALS according to El Escorial Criteria
    2. Subjects in whom other causes of neuromuscular weakness have not been excluded.
    3. Assisted ventilation of any type within 3 months before the screening visit or at screening
    4. Patients requiring Radiologically Inserted Gastrostomy (RIG) or Percutaneous Endoscopic Gastroscopy (PEG) feeding
    5. Involvement in any other interventional study involving use of another IMP or biological product within 3 months of screening
    6. Any use of antioxidants, edaravone, tirasemtiv or CK-2127107 within 1 month before the screening visit.
    7. Any botulinum toxin use within 3 months before the screening visit
    8. Any form of stem cell or gene therapy for the treatment of amyotrophic lateral sclerosis (ALS)
    9. Neuroimaging of brain and cervical spine with Magnetic Resonance Imaging (MRI) indicating compressive myelopathy as an alternate diagnosis
    10. Laboratory examinations including Acetylcholine receptor (AChR) antibodies and Muscle Specific Kinase (MuSK) antibodies to exclude Bulbar onset Myasthenia gravis from Bulbar onset Motor Neurone disease as an alternate diagnosis and Antinuclear Antibodies (ANA), Anti-neutrophil cytoplasmic antibodies (ANCA), Extractable Nuclear Antigen (ENA) antibodies, Creatine Kinase (CK), electrophoresis and immunoglobulin indicating an alternate diagnosis for muscle disease like Myositis
    11. Abnormal liver function defined as AST and/or ALT >3 times upper limit of normal
    12. Any head trauma, intracranial or spinal surgery within 3 months of trial entry
    13. Patients who have had recurrent falls will be excluded to reduce the risk of intracerebral haemorrhage with this Investigational Medicinal Product (IMP)
    14. Current use of an anticoagulant e.g Warfarin, Aspirin, Clopidogrel, any novel anticoagulants (NOAC)s or low molecular weight heparin
    15. Uncontrolled hypertension
    16.Current or previous history of heparin-induced thrombocytopenia
    17. Active peptic ulcer disease
    18. Known hypersensitivity to sulphur
    19. Severe liver insufficiency
    20. Patients with evidence of major psychiatric illness, significant cognitive impairment or clinically evident dementia that may interfere with the patient`s ability to comply with study procedures.
    21. Pulmonary illness (e.g asthma or Chronic Obstructive Pulmonary Disease (COPD) requiring regular treatment
    22. Patient judged to be actively suicidal by the Investigator during 3 months before the screening visit
    23. Subjects with a diagnosis of another neurodegenerative disease (e.g. Parkinson`s disease, Altzheimers disease and Frontotemporal dementia)
    E.5 End points
    E.5.1Primary end point(s)
    Our primary outcome measures will inform whether the therapy is feasible in this patient group.

    The number of SAEs and AEs will be calculated and summarised. Furhthermore, the number of patients experiencing each will be calculated. Each of these analyses will be broken down by grade, relatedness, event type (admitting event for SAEs); expectedness (SAEs only) and sequelae (SAEs only).

    Measured by the incidence of intolerable adverse events. An intolerable adverse event will satisfy all of the following criteria:
    1. Associated with a serious adverse event or a drug discontinuation of greater than three weeks
    2. Grade 3, 4 or 5 in severity according to CTCAE version 4;
    3. In the opinion of the Investigator is i) definitely related or ii) probably related or iii) possibly related to the study drug treatment.
    Adverse events which are considered unrelated or probably not related will not be classed as intolerable events

    Quantity of study drug administered
    Total drug administered, number of administrations, number and length of interruptions, and number of discontinuations will be calculated for each patient and summarised.

    E.5.1.1Timepoint(s) of evaluation of this end point
    Interim analyses of safety outcomes and available efficacy outcomes will be presented to the relevant independent oversight committee after the sentinel patients; and periodically thereafter.

    Planned final analysis within 6 months of the final protocol assessment of the final patient.
    E.5.2Secondary end point(s)
    Revised ALS Functional Rating Scale (ALSFRS-R)
    QOL (ALSAQ-40)
    Urinary p75ECD levels
    NfL levels

    HPLC analyses of purine-pyrimidine metabolites (serum and urine)
    Water-soluble and Lipid-soluble Antioxidants and Vitamins (serum)
    Analysis of lactate (serum)
    HPLC analyses of primary amino acids (AA) and amino-group containing compounds (ACCG) (serum and urine)

    E.5.2.1Timepoint(s) of evaluation of this end point
    Interim analyses of safety outcomes and available efficacy outcomes will be presented to the relevant independent oversight committee (the DMC) after the sentinel patients; and periodically thereafter.

    Planned final analysis within 6 months of the final protocol assessment of the final patient.
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    and Tolerability study
    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) 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 No
    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 No
    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.8.3 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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    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 No
    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
    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 months6
    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 months6
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F. of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F. of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F. of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F. of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F. of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F. of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 0
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 15
    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)
    Dosing beyond 10 weeks is dependent upon TikoMed AB providing satisfactory long-term safety toxicology data to the Medicines and Healthcare Products Regulatory Agency (MHRA). This work is currently ongoing. Once this report is available the sponsor will submit a Substantial Amendment for regulatory approval to the MHRA and Research Ethics Committee (REC).
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-08-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-09
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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