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    Summary
    EudraCT Number:2019-000316-29
    Sponsor's Protocol Code Number:3571
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2020-07-09
    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 number2019-000316-29
    A.3Full title of the trial
    Hepatocytes co-encapsulated with mesenchymal stromal cells in alginate microbeads for the treatment of acute liver failure in paediatric patients: HELP study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Hepatocytes co-encapsulated with mesenchymal stromal cells in alginate micro-beads for the treatment of acute liver failure in paediatric patients: HELP study
    A.3.2Name or abbreviated title of the trial where available
    Help Trial
    A.4.1Sponsor's protocol code number3571
    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 SponsorKing's College Hospital NHS Foundation 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 supportNIHR
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKing's College Hospital NHS Foundation Trust
    B.5.2Functional name of contact pointPaediatric Liver Research
    B.5.3 Address:
    B.5.3.1Street AddressKing's College Hospital, Denmark Hill
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE5 9RS
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442032994408
    B.5.6E-mailanil.dhawan@kcl.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 name Alginate micro-beads containing hepatocytes and mesenchymal stromal cells
    D.3.2Product code HMB002
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraperitoneal use
    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 Yes
    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
    Acute liver failure in paediatric patients
    E.1.1.1Medical condition in easily understood language
    Infants and children with acute liver failure
    E.1.1.2Therapeutic area Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10049844
    E.1.2Term Acute liver failure
    E.1.2System Organ Class 100000004871
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety, biological activity and tolerability of transplantation of a single dose of microbeads made from the optimum combination of peptide-alginate, mesenchymal stromal cells (MSCs) and hepatocytes to paediatric patients with acute liver failure
    E.2.2Secondary objectives of the trial
    To establish proof of concept of the transplantation of microbeads made from the optimum combination of peptide-alginate, MSCs and hepatocytes.

    To inform sample size and confidence intervals to design a larger randomized clinical trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    I. Infant or child (male or female) under the age of 16 years at recruitment.
    II. Written informed consent obtained from a parent / legal guardian;
    III. Presence of ALF defined as a multisystemic disorder in which severe impairment of liver function with or without encephalopathy occurs in association with hepatocellular necrosis reflected as synthetic liver failure in a child with no recognised underlying chronic liver disease. Children must fit one of the ALF categories as described in Appendix 1b;
    IV. Willing and able to comply with the study visit schedule;
    E.4Principal exclusion criteria
    I. Severe ascites causing high intra-abdominal pressure and / or respiratory compromise;
    II. Intra-abdominal sepsis suspected or proven;
    III. Clinical condition too unstable to tolerate procedure without compromise;
    IV. Proven pre-existing allergy or intolerance to alginate on medical history;
    V. Proven pre-existing allergy to gentamicin on medical history;
    VI. Intraperitoneal or intra-abdominal malignancy;
    VII. Adhesions or fistulae to anterior abdominal wall;
    VIII. Children who weigh in excess of 33kg
    IX. Pregnant or lactating patients (positive pregnancy test for females of child bearing potential at screening).
    X. Female patients of childbearing potential who are not willing to use highly effective methods of contraception to prevent pregnancy or abstain from heterosexual activity for the duration of the study.
    *Females of child bearing potential are females who have experienced menarche and are not surgically sterilised (e.g. by tubal occlusion, hysterectomy, bilateral salpingectomy) or post-menopausal (defined as at least 1 year since last regular menstrual period).
    ** Highly effective methods of birth control are those with a failure rate of < 1% per year when employed consistently and correctly.
    Highly effective methods of contraception as per HMA / CTFG working group are combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, the preparation may be oral, intravaginal or transdermal; progesterone-only hormonal contraception associated with inhibition of ovulation which may be oral, injectable or implantable; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence through the trial study period;
    *** Sexual abstinence is considered to be highly effective method only if defined as refraining from heterosexual activity from the date of consent until the week 52 visit. The reliability of this method should be evaluated in relation to the duration of the study and the preferred and usual lifestyle of the participant.
    XI. Male patients who are not willing to use an effective method of contraception (condom, vasectomy, sexual abstinence) for the duration of the study, when engaging in sexual activity with a female of childbearing potential;
    XII. Participation in concurrent therapeutic trial for ALF;
    XIII. Imminent liver transplantation expected within 12 hours of infusion;
    XIV. Total hepatectomy;
    XV. Dependent on Extracorporeal Membrane Oxygenation (ECMO);
    XVI. Previous liver transplant
    E.5 End points
    E.5.1Primary end point(s)
    Safety: Moderate to severe adverse event occurrences due to product in 1st 52 weeks post procedure

    Tolerability: assessed by the proportion of initiated infusions where >80% of the infusion is received by the patient.

    Biological activity: Survival with native liver at 24 weeks post treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At IMP administration and then at 24 and 52 weeks post treatment
    E.5.2Secondary end point(s)
    Blood marker levels including haematological, biochemical and coagulation baseline to 52 weeks post treatment.
    Quality of life measures
    Survival with native liver at 52 weeks post treatment
    Survival with transplanted or native liver at 24- and 52-weeks post treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout 52 week trial period
    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 Yes
    E.6.13.1Other scope of the trial description
    Feasibility
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    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) No
    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 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.8.2.4Number 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 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
    Last patient last 10 year annual follow up post IMP infusion.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 17
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 8
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Infants and children less than 16 years of age are unlikely to give informed assent due to their age and/or clinical condition (ventilated or sedated)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state17
    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)
    IMP is a one-off administration however as the participants have received an ATIMP they will be followed up long-term for 10 years. Follow up will be annually and follow routine care clinic visit schedules. SAEs will be reported in this period as per regulatory requirements.
    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 Decision2020-09-14
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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