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    The EU Clinical Trials Register currently displays   38003   clinical trials with a EudraCT protocol, of which   6235   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2017-002762-44
    Sponsor's Protocol Code Number:RG-16-040
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2019-06-25
    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 ConcernedIreland - HPRA
    A.2EudraCT number2017-002762-44
    A.3Full title of the trial
    A phase I/II study evaluating the safety and activity of Pegylated recombinant human Arginase (BCT-100) in Relapsed/refractory cancers of Children and young adults
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study evaluating the safety and activity of a new drug - pegylated recombinant hyman arginase (BCT-100) in cancers in children and young people that have come back (relapsed) or have not responded to treatment (refractory).
    A.3.2Name or abbreviated title of the trial where available
    PARC
    A.4.1Sponsor's protocol code numberRG-16-040
    A.5.4Other Identifiers
    Name:EudraCT Number Number:2017-002762-44
    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 supportCancer Research UK
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportImagine for Margo
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportBioCancer Treatment International
    B.4.2CountryHong Kong
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity of Birmingham
    B.5.2Functional name of contact pointSean Jennings
    B.5.3 Address:
    B.5.3.1Street AddressResearch Support Group
    B.5.3.2Town/ cityBirmingham
    B.5.3.3Post codeB15 2TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01214158011
    B.5.6E-mailresearchgovernance@contacts.bham.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 namePEG-BCT-100
    D.3.2Product code PEG-BCT-100
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPEG-BCT-100
    D.3.9.3Other descriptive namerhArg1peg5000
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number3.06 to 3.74
    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) 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 Yes
    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
    Relpased/refractory paeditaric cancers:
    Leukaemias
    Sarcoma
    Neuroblastoma
    High grade glioma (brain cancers)
    E.1.1.1Medical condition in easily understood language
    Children's cancers that have come back or are resistant to treatment.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10000830
    E.1.2Term Acute leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10065443
    E.1.2Term Malignant glioma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10029260
    E.1.2Term Neuroblastoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10039494
    E.1.2Term Sarcoma NOS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives of this trial are to:
    - establish a safe and active dose of BCT-100 in children and young people
    - evaluate if BCT-100 is effective against acute leukaemias, neuroblastoma, sarcoma and high grade glioma which have come back (relapsed) or not responded to previous treatment (refractory)as measured by disease response at 8 weeks.

    The trial is made of two parts:
    In the first part, the doctors will be looking for the dose of BCT-100 which is both safe and active in children and young adults. This will involve giving increasing doses of the drug (BCT-100) to patients to find the dose which does not cause significant side effects (known as dose-limiting toxicities) and completely depletes arginine levels. All the doses of BCT-100 used in this trial have found to be safe in adults.

    In the second part the trial, the final dose chosen in part 1 will then be given to all patients who take part. Disease response at eight weeks will be measured to determine activity of
    E.2.2Secondary objectives of the trial
    The secondary objectives of the trial are:
    • To assess the safety of BCT-100.
    • To assess disease response at any time during treatment with BCT-100.
    • To assess progression free survival (PFS) and overall survival (OS).
    • To determine the pharmacokinetics of BCT-100 in the paediatric population.
    • To assess the duration and magnitude of arginine depletion.

    The exploratory objectives of the trial are:
    • To collect blood and tumour samples for (future) ethically approved projects.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Aged 1- <25 years old
    • Histologically confirmed disease in one of the following four cohorts. Patients with and without metastatic lesions are eligible.
    o Cohort 1 - Acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML)
    o Cohort 2 - Neuroblastoma
    o Cohort 3 - Sarcoma
    o Cohort 4 - High grade glioma (as defined by 2016 WHO CNS classification)
    • Radiological or laboratory evidence of disease progression (during or after completion of first line treatment) or any subsequent recurrence
    • Measurable bone marrow disease (cohort 1) or at least one evaluable radiological site of disease (cohort 2, 3 and 4)
    • Adequate liver function defined as a total bilirubin <1.5x the upper limit of normal for age and ALT <3x the upper limit of normal for age
    • Normal ECG
    • Documented negative pregnancy test for female patients of childbearing potential within 7 days of trial entry
    • Sexually active patients must agree to use adequate and appropriate contraception while on study drug and for 12 months following treatment discontinuation
    • Written informed consent given by patient and/or parents/legal representative

    E.4Principal exclusion criteria
    • Previous treatment with another therapeutic arginine depleting drug (bacterial or human) or arginase inhibitor
    • Presence of any ≥ CTCAE grade 3 clinically significant treatment-related toxicity from prior therapies
    • Pregnant or lactating female
    • Evidence of uncontrolled infection
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measures of this study are:

    1. The safe and optimal recommended dose of BCT-100 for children and young people. Safety profile as measured by the occurrence/non-occurrence of specified dose-limiting toxicities within 28 days of treatment with BCT-100.
    Optimal dose as measured by the complete depletion of arginine after 3 doses of BCT-100

    2. Disease response (Complete Response (CR) or Partial Response (PR))to BCT-100 after eight weeks of treatment with BCT-100. This is defined by the following criteria for each group:
    Group 1: Modified Cheson criteria
    Group 2: International Neuroblastoma Response Criteria
    Group 3: RECIST criteria
    Group 4: RANO criteria
    (see protocol for further details)




    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase I
    Dose-limiting toxicities within 28 days of treatment with BCT-100.
    Optimal dose as measured by the complete depletion of arginine after 3 doses of BCT-100

    Phase II
    Response at 8 weeks
    E.5.2Secondary end point(s)
    • To assess the safety of BCT-100.
    • To assess disease response at any time during treatment with BCT-100
    • To assess progression free survival (PFS) and overall survival (OS).
    • To determine the pharmacokinetics of BCT-100 in the paediatric population.
    • To assess the duration and magnitude of arginine depletion.

    E.5.2.1Timepoint(s) of evaluation of this end point
    •Incidence and severity of Adverse Events (AEs) defined by National Cancer Institute Common
    •Terminology Criteria for Adverse Events (CTCAE) v4 (see Appendix 3)
    • Disease response ( CR / PR) at any time during treatment with BCT-100
    • PFS - median survival at 6 and 12 months
    • OS - median survival at 6 and 12 months
    • PK profile of BCT-100 concentration in blood, bone marrow, and CSF - during and at end of treatment
    • Arginine concentrations in blood, bone marrow, and CSF - during and at end of treatment
    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 No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    First in paediatric population
    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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Australia
    Denmark
    France
    Germany
    Ireland
    Italy
    Netherlands
    New Zealand
    Spain
    United Kingdom
    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 years3
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days29
    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: 60
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 26
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 26
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) No
    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 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
    patients over 16 years with disease involving the brain may lack capacity to give consent. The presence of a legal representative will be required
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 52
    F.4.2.2In the whole clinical trial 64
    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)
    Patients will receive treatment with BCT-100 for up to 24 weeks as part of the study protocol and may continue treatment after this at the discretion of the chief investigator.
    BioCancer Treatment International will continue to provide BCT-100 for trial patients if having ongoing clinical benefit.

    If patients come off the trial due to disease progression or treatment toxicity, their treating physician will discuss further treatment options with them and their family as per standard of care.
    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 Decision2019-10-04
    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
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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