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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-006689-18
    Sponsor's Protocol Code Number:BraimTOR-ONKO
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-10-05
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2021-006689-18
    A.3Full title of the trial
    An open randomized phase II clinical trial evaluating the safety and efficacy of rapamycin in the treatment of gliomas high-grade malignant gliomas in children as part of the establishment management of rare and ultra rare diseases of the central nervous system associated with mTOR pathway activation: BraimTOR- ONKO
    Otwarte randomizowane badanie kliniczne II fazy oceniające bezpieczeństwo
    i skuteczność rapamycyny w leczeniu glejaków o wysokim stopniu złośliwości u dzieci w ramach ustalenia postępowania w rzadkich i ultrarzadkich chorobach ośrodkowego układu nerwowego związanych z aktywacją szlaku mTOR: BraimTOR- ONKO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study to assess safety and efficacy of rapamycin in paediatric high grade glioma
    Ocena bezpieczeństwa i skuteczności rapamycyny w leczeniu glejaków o wysokim stopniu złośliwości u dzieci
    A.3.2Name or abbreviated title of the trial where available
    BraimTOR-ONKO
    BraimTOR-ONKO
    A.4.1Sponsor's protocol code numberBraimTOR-ONKO
    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 SponsorThe Children's Memorial Health Institute
    B.1.3.4CountryPoland
    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 supportMedical Research Agency
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationThe Children's Memorial Health Institute
    B.5.2Functional name of contact pointMarta Perek-Polnik
    B.5.3 Address:
    B.5.3.1Street Address20 Dzieci Polskich Avenue
    B.5.3.2Town/ cityWarsaw
    B.5.3.3Post code04-730
    B.5.3.4CountryPoland
    B.5.4Telephone number48228151779
    B.5.6E-mailm.perek-polnik@ipczd.pl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Rapamune 1 mg/ml oral solution
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited Ramsgate Road Sandwich Kent, CT13 9NJ Great Britain
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameRapamune 1mg/ml oral solution
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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.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 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
    high grade glioma
    glejak o wysokim stopniu złośliwości
    E.1.1.1Medical condition in easily understood language
    high grade glioma
    glejak o wysokim stopniu złośliwości
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    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 of the study is to determine safety and tolerability of rapamycin in the treatment of high grade glioma in children
    Celem pierwszorzędowym badania klinicznego jest ocena bezpieczeństwa oraz tolerancji rapamycyny w leczeniu pacjentów pediatrycznych z HGG
    E.2.2Secondary objectives of the trial
    - The secondary aim of the study is to asses safety and efficacy of rapamycin in a treatment of high grade glioma in children.
    - The ecsplorative aim of the study is to collate genetic and epigenetic markers with the rapamycine efficacy and safety in pediatric high grade glioma patients.
    - Drugorzędowym celem jest ocena bezpieczeństwa i skuteczności klinicznej proponowanej interwencji terapeutycznej.
    - Celem eksploracyjnym jest ocena korelacji markerów genetycznych i epigenetycznych ze skutecznością terapii rapamycyną (identyfikacja molekularnych markerów prognostycznych i predykcyjnych).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 3 to 18 years, inclusive.
    2. Informed consent signed by parents or legal guardian of minor and by patient over 13 years of age) for the study diagnostic and therapeutic procedures, including molecular -based diagnostic and prognostic tests using NGS method.
    3. Histological diagnosis of glioma WHO grade III and IV confirmed by reference pathologist.
    4. Negative result of the pregnancy test performed within 2 weeks prior to the start of the study in patients of reproductive age.
    5. Written consent to the use of effective contraception in sexually active patients.
    1. Wiek pacjenta od ukończenia 3 roku życia do 18 roku życia.
    2. Pisemna świadoma zgoda opiekunów prawnych i pacjenta (jeśli ukończył 13 rok życia) na zaplanowane w projekcie działania diagnostyczne i terapeutyczne oraz na przeprowadzenie badań molekularnych w zakresie zmian somatycznych i germinalnych metodą NGS.
    3. Rozpoznanie histopatologiczne glejaka III lub IV st. WHO, potwierdzone przez referencyjnego patomorfologa.
    4. Negatywny wynik testu ciążowego, wykonanego w ciągu 2 tygodni poprzedzających rozpoczęcie badania u pacjentek w wieku rozrodczym.
    5. Pisemna zgoda na stosowanie skutecznej antykoncepcji u pacjentów aktywnych seksualnie.
    E.4Principal exclusion criteria
    1. Any severe comorbid disease (eg kidney insufficiency, immunological deficiencies, HIV infection).
    2. Clinically significant cardiovascular condition ( eg arrythmia)
    3. Previous (< 5 yrs ) diagnosis of malignant neoplasm.
    4. Active infection requiring systemic treatment.
    5. Known allergy to the studied products.
    6. Concomitant use of CYP3A or PgP inhibitors.
    7. Radiological evidence of active intracranial hemorrhage (excluding receding post biopsy or focal bleeding).
    8. Major surgery in the past 28 days
    9. Diagnosis of DIPG
    16. Live vaccine intake <6 weeks prior to study inclusion,
    17. Receipt of inactivated vaccine or mRNA vaccine <4 weeks prior to
    study inclusion.
    1. Ciężkie współistniejące choroby, np. niewydolność nerek, niedobory odporności, zakażenie HIV.
    2. Klinicznie istotna choroba układu sercowo-naczyniowego, np. arytmia.
    3. Wcześniejsze <5 lat rozpoznanie innego nowotworu.
    4. Aktywna infekcja wymagająca leczenia ogólnego.
    5. Przeciwwskazania do stosowania lub znana nadwrażliwość na lek stosowany w badaniu.
    6. Równoczesne przyjmowanie leków będących silnymi inhibitorami CYP3A lub PgP.
    7. Radiologiczne cechy aktywnego krwawienia w OUN poza bezobjawowymi ustępującymi zmianami po biopsji lub punktowym krwawieniem w guzie.
    8. Poważny zabieg chirurgiczny, otwarta biopsja < 28 dni przed rozpoczęciem leczenia.
    9. Pacjenci, u których stwierdzono DIPG
    10. Przyjęcie szczepionki żywej < 6 tygodni przed włączeniem do badania,
    11. Przyjęcie szczepionki inaktywowanej lub szczepionki mRNA <4 tygodni przed włączeniem do badania.
    E.5 End points
    E.5.1Primary end point(s)
    The primary safety endpoint of rapamycin will be the assessment of the incidence of 3 and 4 grade adverse reactions (according to the CTCAE classification) during the treatment phase and follow-up.
    Pierwszorzędowym punktem końcowym jest ocena liczby działań niepożądanych 3 i 4 stopnia (zgodnie z klasyfikacją CTCAE) w czasie fazy leczenia i obserwacji pacjentów z HGG.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - interim analysis
    -final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
    -analiza okresowa
    -ostateczna analiza po formalnym zamknięciu bazy danych, planowana w ciągu miesiąca od ostatniej wizycie ostatniego pacjenta w badaniu
    E.5.2Secondary end point(s)
    • OS (overall survival) after 15 months, 24 months, 3 years, 5 years,
    • EFS (event free survival) after 15 months, 24 months, 3 years, 5 years,
    • PFS (progression free survival) after 15 months, 24 months, 3 years, 5 years,
    • ORR ( overall response rate) according RAPNO (CR, PR, SD, PD) criteria,
    • Evaluation of adverse event seriousness according CTCAE,
    • Evaluation of the number of patients who experience adverse events that effects with premature termination of tratment during the therapeutic dose period.
    OS, EFS and PFS will be counted from the start of cancer treatment (the day of surgery).
    Drugorzędowe punkty końcowe obejmują analizę:
    • OS (overall survival) po 15 miesiącach, 24 miesiącach, 3 latach, 5 latach,
    • EFS (event free survival) po 15 miesiącach, 24 miesiącach, 3 latach, 5 latach,
    • czasu do progresji (PFS) po 15 miesiącach, 24 miesiącach, 3 latach, 5 latach,
    • całkowitego wskaźnika odpowiedzi (ORR, overall response rate) w kategoriach RAPNO (CR, PR, SD, PD),
    • ciężkości działań niepożądanych według klasyfikacji CTCAE,
    • liczby pacjentów doświadczających działań niepożądanych, wymagających wykluczenia z badania lub przedwczesnego zakończenia uczestnictwa w okresie podawania terapeutycznej dawki leku.
    OS, EFS i PFS będą liczone od początku leczenia nowotworu (dzień zabiegu operacyjnego).
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 15 months, 24 months, 3 years and 5 years from the start of cancer treatment (the day of surgery)
    Po 15 miesiącach, 24 miesiącach, 3 latach i 5 latach od początku leczenia nowotworu (dzień zabiegu operacyjnego)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Temozolomid, Lomustine
    Temozolomid, Lomustine
    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 concerned3
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    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: 50
    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) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    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 Yes
    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
    the study will include underage and incapacitated patients - their
    parents/caregivers will be asked for their consent
    do badania włączani będą pacjenci niepełnoletni oraz ubezwłasnowolnieni – o zgodę zostaną poproszeni opiekunowie prawni/rodzice
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    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 be offered standard treatment of that condition
    Pacjentom zostanie zaproponowane standardowe leczenie w tym
    schorzeniu
    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 Decision2022-04-30
    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 StatusTrial now transitioned
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