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    Summary
    EudraCT Number:2007-002809-48
    Sponsor's Protocol Code Number:CRO782
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-03-20
    Trial results View 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 number2007-002809-48
    A.3Full title of the trial
    Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure and a left ventricular assist device
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure and a left ventricular assist device
    A.3.2Name or abbreviated title of the trial where available
    The SERCA2a-LVAD Trial
    A.4.1Sponsor's protocol code numberCRO782
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00534703
    A.5.4Other Identifiers
    Name:Funder referenceNumber:SP/09/007/27920
    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 SponsorImperial College London and Imperial College Healthcare NHS Trust
    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 supportBritish Heart Foundation
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportCelladon Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Trials and Evaluation Unit, Royal Brompton Hospital
    B.5.2Functional name of contact pointDaphne Babalis
    B.5.3 Address:
    B.5.3.1Street AddressSydney street
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSW3 6NP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number02073518889
    B.5.5Fax number02073518829
    B.5.6E-maild.babalis@rbht.nhs.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 nameMYDICAR
    D.3.2Product code AAV1/SERCA2a
    D.3.4Pharmaceutical form Solution for injection/infusion
    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 INNAAV1/SERCA2a
    D.3.9.2Current sponsor codeCRO782
    D.3.9.3Other descriptive nameMYDICAR
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1 x 10^13
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboIntracoronary use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Advanced heart failure
    E.1.1.1Medical condition in easily understood language
    Advanced heart failure is a medical condition that occurs when the heart cannot pump blood sufficiently.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10010684
    E.1.2Term Congestive heart failure
    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
    To determine the safety and feasibility of increasing the level of SERCA2a protein by viral gene transfer into patients with advanced heart failure who have had a mechanical pump (left ventricular assist device) that assists heart function implanted.
    E.2.2Secondary objectives of the trial
    1. Presence of viral DNA and RNA molecules will be measured in patient heart samples to evaluate whether the virus has been delivered to the heart cells
    2. Function of the left ventricle will be assessed by echocardiography and physical activity tests (MVO2 treadmill testing and six-minute walk tests)

    Depending on availability of heart samples the following may also be investigated:
    3. The levels of SERCA protein in the heart will be measured
    4. Changes in other proteins relevant for heart contraction such as phospholamban, sarcoplasmic reticulum release channel and Na2+/Ca2+-exchanger will be determined
    5. The function of isolated heart muscle cells
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients that have had a left ventricular assist device (LVAD) implanted for chronic heart failure
    2. Patients are clinically stable in the opinion of the clinical team looking after the patient
    3. Written informed consent
    E.4Principal exclusion criteria
    1. <18 or >70 years of age at the time of consent
    2. Pregnancy or within 6 months of giving birth
    3. Women of child-bearing potential not using an effective method of contraception
    4. Men not using an effective method of contraception
    5. Suspected or active viral, fungal or parasitic infection within 48 hours prior to administration of IMP, in the opinion of the investigator*.
    6. Patients participating in another clinical trial
    7. Patients unable to comply with the protocol mandated procedures for social or other reasons, in the opinion of the investigator and primary care physician

    * Eligible, enrolled and randomised patients who develop an infection will have study treatment delayed until 7 or more days after the time point when infection is no longer clinically evident.
    E.5 End points
    E.5.1Primary end point(s)
    Overall safety and feasibility of administering AAV1/SERCA2a to patients with left ventricular assist devices (LVAD) implanted.

    Safety is defined as incidence of death and major adverse cardiovascular events, and out of range laboratory values. Both AAV1/SERCA2a treated cohorts (with and without presence of neutralising antibodies to AAV) will be compared to the placebo group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months post-randomisation
    E.5.2Secondary end point(s)
    Key comparisons will be performed between NAb+ and NAb- cohorts; additional comparisons will be performed between each of NAb+ and NAb- treated cohorts and placebo groups, including comparison of longitudinal changes from baseline (paired analyses).
    1. Presence of exogenous viral vector genome in the myocardium measured by qPCR for the viral DNA
    2. Left ventricular function assessed by echocardiography and exercise capacity (6MWT, MVO2) during minimal LVAD support (low/no flow settings depending upon device)

    Depending on availability of cardiac tissue also:
    3. Levels of SERCA2a protein
    4. Other relevant proteins e.g. phospholamban, the sarcoplasmic reticulum calcium release channel, the Na+/Ca2+-exchanger.
    5. Function of isolated myocytes
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months post-randomisation
    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) 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 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 trial2
    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 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
    The end of this trial is defined as completion of the last follow-up visit for the last patient recruited. The end of trial will be taken as completion of the 6-month follow-up visit for the last patient recruited.
    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.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: 24
    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 state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 24
    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)
    After participation in the trial, ongoing care will continue according to routine clinical care for this patient population.
    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 Decision2013-04-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-12-20
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2015-09-19
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