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    Summary
    EudraCT Number:2021-001375-16
    Sponsor's Protocol Code Number:DIPGen
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-07
    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 number2021-001375-16
    A.3Full title of the trial
    DIPGEN – Multicenter, open label study using molecularly determined targeted therapies in children 3-18 years of age with DIPG (diffuse intrinsic pontine glioma- DIPG)
    Zastosowanie terapii celowanej u dzieci od 3 do 18 roku życia z rozpoznaniem rozlanego naciekającego glejaka mostu (diffuse intrinsic pontine glioma - DIPG) w oparciu o wyniki badań genetycznych - DIPGen
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the effectiveness of drugs selected depending on the molecular profile of diffuse intrinsic pontine glioma (DIPG)
    Badanie skuteczności leków dobranych w zależności od profilu molekularnego rozlanego glejaka pnia mózgu (diffuse intrinsic pontine glioma, DIPG)
    A.3.2Name or abbreviated title of the trial where available
    DIPGen
    DIPGen
    A.4.1Sponsor's protocol code numberDIPGen
    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 pointBożenna Dembowska-Bagińska
    B.5.3 Address:
    B.5.3.1Street AddressAv. Dzieci Polskich 20
    B.5.3.2Town/ cityWarsaw
    B.5.3.3Post code04-730
    B.5.3.4CountryPoland
    B.5.4Telephone number+48228151787
    B.5.5Fax number+48228157575
    B.5.6E-mailb.dembowska@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 Mekinist 0,5 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited Vista Building Elm Park, Merrion Road Dublin 4 Ireland
    D.2.1.2Country which granted the Marketing AuthorisationPoland
    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.3.4Pharmaceutical form Film-coated tablet
    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.IMP: 2
    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 coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited Ramsgate Road Sandwich Kent, CT13 9NJ Wielka Brytania
    D.2.1.2Country which granted the Marketing AuthorisationPoland
    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.3.4Pharmaceutical form Coated tablet
    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.IMP: 3
    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 No
    D.2.1.1.1Trade name Rapamune 1mg/ml oral solution
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    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.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    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) 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
    diffuse intrinsic pontine glioma
    rozlany naciekający glejak mostu
    E.1.1.1Medical condition in easily understood language
    brain stem tumor
    guz pnia mózgu
    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 aim of the study is to develop the optimal treatment for patients with DIPG by identifying molecular markers important for therapy, adjusting the type of medicinal product to the indicated markers and assessing the safety and efficacy of selected drugs (sirolimus, trametinib) in the treatment of DIPG in children
    Celem badania jest opracowanie optymalnego leczenia dla pacjentów z DIPG poprzez identyfikację istotnych dla terapii markerów molekularnych, dostosowanie rodzaju produktu leczniczego do wskazanych markerów oraz ocena bezpieczeństwa i skuteczności wybranych leków (syrolimus, trametynib) w leczeniu DIPG u dzieci.
    E.2.2Secondary objectives of the trial
    1. assessment of the efficacy and safety of selected drugs (sirolimus, trametinib) in the treatment of diffuse infiltrating ponsal gliomas in children;
    2. to evaluate the safety and efficacy of the simultaneous irradiation and use of sirolimus;
    3. adjusting the type of medicinal product to the indicated markers;
    4. identification of molecular markers important for therapy;
    5. creating a repository of genetic and clinical data of patients with DIPG;
    6. development of the molecular profile of DIPG tumors.
    1. ocena skuteczności i bezpieczeństwa i wybranych leków (syrolimus, trametynib) w leczeniu rozlanych naciekających glejaków mostu u dzieci;
    2. ocena bezpieczeństwa i skuteczności równoczesnego napromieniania i stosowania syrolimusu;
    3. dostosowanie rodzaju produktu leczniczego do wskazanych markerów;
    4. identyfikacja istotnych dla terapii markerów molekularnych;
    5. utworzenie repozytorium danych genetycznych i klinicznych pacjentów z DIPG;
    6. opracowanie profilu molekularnego guzów typu DIPG.
    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. Diagnosis of diffuse intrinsic pontine glioma (DIPG) based on clinical and radiological criteria
    4. Histological diagnosis of glioma WHO grade III and IV confirmed by reference pathologist.
    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 DIPG - kliniczno-radiologiczne.
    4. Rozpoznanie histopatologiczne glejaka III lub IV st. WHO, potwierdzone przez referencyjnego patomorfologa.
    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

    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.

    E.5 End points
    E.5.1Primary end point(s)
    1. Overall survival (OS) from the time of diagnosis until last visit or death.
    2. Safety assessment of sirolimus administered during radiotherapy and in combination with trametinib as adjunctive treatment after irradiation.
    1. Całkowite przeżycie (OS) mierzone od daty rozpoznania do ostatniej wizyty lub zgonu niezależnie od jego przyczyny.
    2. Ocena bezpieczeństwa stosowania syrolimusu w trakcie radioterapii oraz bezpieczeństwa stosowania syrolimusu i trametynibu po zakończonym napromienianiu.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary endpoints will be evaluated at 1,2,3 years from diagnosis.
    Momenty ewaluacji Pierwszorzędowego Punktu Końcowego: 1 rok, 2 lata, 3 lata od rozpoczęcia leczenia czyli od dnia biopsji
    E.5.2Secondary end point(s)
    1. Progression free survival from diagnosis to progression
    2. Response rate assessed at time points specified in the protocol (MRI every 2 months from commnecement of systemic treatment)
    3. Quality of life assessment (using PedQoL- pediatric quality of life inventory) at time points specified in the protocol.
    4. Comparison of safety in 2 groups of patients; treated with sirolimus alone or in combination with trametinib.
    1.Czas przeżycia do progresji (PFS) mierzony od daty rozpoznania do progresji.
    2.Odpowiedź nowotworu na leczenie oceniana w ustalonych protokołem punktach czasowych (w badaniu MR wykonywanym co 2 miesiące od momentu włączenia leczenia systemowego).
    3.Ocena jakości życia - w ustalonych punktach czasowych przeprowadzona będzie ocena jakości życia, z zastosowaniem walidowanego kwestionariusza (PedQoL- pediatric quality of life).
    4.Porównanie bezpieczeństwa w grupie pacjentów leczonych syrolimusem z grupą pacjentów leczonych syrolimusem i trametynibem
    E.5.2.1Timepoint(s) of evaluation of this end point
    .
    .
    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 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 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 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 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
    .
    .
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 100
    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: 75
    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 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
    .
    Do badania DIPGen włączani będą pacjenci w wieku 3-18 lat, zgodę na udział w badaniu będą musieli wyrazić rodzice/opiekunowie prawni.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state100
    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)
    none
    brak
    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 Decision2021-08-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-28
    P. End of Trial
    P.End of Trial StatusOngoing
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