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    Summary
    EudraCT Number:2018-001063-23
    Sponsor's Protocol Code Number:012357
    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:2018-03-21
    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 ConcernedUK - MHRA
    A.2EudraCT number2018-001063-23
    A.3Full title of the trial
    PHASE II STUDY ASSESSING THE COMBINED USE OF AUTOLOGOUS BONE MARROW DERIVED MONONUCLEAR CELLS AND G-CSF WITH PERCUTANEOUS CIRCULATORY ASSISTANCE IN THE TREATMENT OF DILATED CARDIOMYOPATHY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    THE COMBINED USE OF BONE MARROW DERIVED STEM CELLS AND A BONE MARROW BOOSTING DRUG (G-CSF) IN THE PRESENCE OF A PUMP THAT SUPPORTS THE HEART IN THE TREATMENT OF DILATED CARDIOMYOPATHY.
    A.3.2Name or abbreviated title of the trial where available
    DCM SUPPORT Study
    A.4.1Sponsor's protocol code number012357
    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 SponsorQueen Mary Innovation Centre, Queen Mary University of London
    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 supportThe Bart's Charity
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCardiac Research Department (Bart's Health NHS Trust)
    B.5.2Functional name of contact pointProfessor Anthony Mathur
    B.5.3 Address:
    B.5.3.1Street AddressSt Bartholomew's Hospital, West Smithfield
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeEC1A 7BE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number02037657807
    B.5.6E-maila.mathur@qmul.ac.uk
    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 nameAutologous Bone Marrow Derived Mononuclear Cells
    D.3.2Product code aBMC
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntracoronary use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAutologous Bone Marrow Derived Mononuclear Cells
    D.3.9.3Other descriptive nameaBMC
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number75,000,000 cells
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    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
    Heart failure is caused by a cardiomyopathy, which is defined as a disorder of the heart muscle. Dilated cardiomyopathy (DCM) is characterised by enlargement of the ventricles and impaired systolic function of one or both ventricles in the absence of significant coronary artery disease.
    E.1.1.1Medical condition in easily understood language
    DCM is a disorder of heart muscle characterised by enlargement of the heart and impaired systolic function (pumping) of one or both vemtricles in the absence of coronary artery disease.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10056419
    E.1.2Term Dilated cardiomyopathy
    E.1.2System Organ Class 100000004849
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The principle research question of this trial is to determine if the injection of autologous bone marrow derived mononuclear cells (cells obtained from the patient’s own bone marrow) into the coronary arteries with co - current G-CSF and a percutaneous mechanical support device is feasible and improves left ventricular ejection fraction (pumping of the main heart chamber). The percutaneous mechanical support device is essentially a pump that is inserted into the femoral artery (the main artery in the groin) and ultimately positioned in the heart. It helps the heart pump blood around the body and takes some of the strain off the heart. Importantly, this device is removed at the end of the procedure and therefore does not remain in long-term.
    E.2.2Secondary objectives of the trial
    Assessment of major adverse cardiac events (MACE; death, Q wave myocardial infarction, need for repeat revascularisation) and changes in New York Heart Association (NYHA) class status. NYHA classification is a simple way of assessing patient’s heart failure symptoms. It places patients into one of four categories (I-IV).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Patients with a confirmed diagnosis of dilated cardiomyopathy under the supervision of a physician or a heart failure nurse specialist.
    • NYHA class III or IV symptoms despite having received optimal medical therapy and appropriate device therapy, as per clinical guidelines for an interval of at least 3 months.
    • No other treatment options available as part of current best standard care.
    • LVEF ≤30% on the cardiac CT scan performed as part of the screening phase.
    • Patient between 18-85 years old.
    E.4Principal exclusion criteria
    • NYHA I-II.
    • Documented latest ejection fraction >30% (any imaging modality)
    • Congenital heart disease.
    • Clinically significant valvular heart disease.
    • Patients who are not suitable for a Percutaneous Mechanical Support Device (E.g. unsuitable femoral artery anatomy, unable able to lie flat for prolonged time to accommodate the stem cell infusion & presence of LV thrombus)
    • Weight of patient that exceeds the maximum limit of the cardiac catheter laboratory table / CT scanner.
    • Cardiomyopathy 2o to a reversible cause that has not been treated e.g. thyroid disease, alcohol abuse, hypophosphataemia, hypocalcaemia, cocaine abuse, selenium toxicity & chronic uncontrolled tachycardia.
    • Cardiomyopathy in association with a neuromuscular disorder e.g. Duchenne’s progressive muscular dystrophy.
    • Previous cardiac surgery.
    • Contra-indication for bone marrow aspiration.
    • Known active infection at time of randomisation.
    • Positive virology tests.
    • Chronic inflammatory disease requiring on-going medication.
    • Concomitant disease with a life expectancy of less than one year
    • Follow-up impossible (no fixed abode, etc.)
    • Neoplastic disease without documented remission within the past 5 years.
    • Patients on renal replacement therapy.
    • Subjects of childbearing potential unless βHCG negative and are on adequate contraception during the trial.
    E.5 End points
    E.5.1Primary end point(s)
    Change in Left Ventricular Ejection Fraction as measured with advanced cardiac imaging (Cardiac CT) at 3 months. Although this is a feasibility study with no control group, a comparator group is available from our recently published REGENERATE DCM Support Trial.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary end point will be assessed at 3 months.
    E.5.2Secondary end point(s)

    • Change in LVEF from baseline measured by advanced cardiac imaging (cardiac CT) at 12 months.
    • Procedural safety as assessed by in hospital procedural related morbidity/mortality.
    • Change in NT-proBNP, Troponin T, renal function and inflammatory profile at 3 and 12 months.
    • Assessment of MACE endpoints at 3 months and 12 months.
    • Assessment of arrhythmia burden through follow up.
    • Change in exercise capacity and NYHA class at 3 months and 12 months as assessed by 6 minute walk test.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secondary end points will be assessed at 12 months.
    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 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) 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.8.5The 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 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
    The study will end when all patients recruited have completed the 12 month follow up, all data has been analysed and the results accepted for journal publication.
    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 days1
    E.8.9.2In all countries concerned by the trial years0
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number 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: 5
    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 state20
    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)
    Participants of this trial will receive a one-off treatment during their hospital admission at the start of the trial. At the end of the trial, no treatment is being withdrawn, and therefore, no arrangements for continued provision of the intervention are required.
    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 Decision2018-05-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-14
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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