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    Summary
    EudraCT Number:2021-003832-96
    Sponsor's Protocol Code Number:ChIm-NB-PL
    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:2021-09-14
    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 number2021-003832-96
    A.3Full title of the trial
    Immunotherapy with dinutuximab beta in combination with chemotherapy for the treatment of patients with primary neuroblastoma refractory to standard therapy and with relapsed or progressive disease
    Immunoterapia z zastosowaniem dinutuksymabu beta skojarzona z chemioterapią w leczeniu pacjentów z neuroblastoma pierwotnie opornym na leczenie standardowe oraz ze wznową lub progresją choroby
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Combination therapy with anti-GD2 antibody immunotherapy (dinutuximab beta) in combination with chemotherapy for refractory neuroblastoma
    Leczenie skojarzone z zastosowaniem immunoterapii przeciwciałami anty-GD2 (dinutuksymab beta) w skojarzeniu z chemioterapią w opornej postaci neuroblastoma
    A.3.2Name or abbreviated title of the trial where available
    ChIm-NB-PL
    A.4.1Sponsor's protocol code numberChIm-NB-PL
    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 SponsorJagiellonian University Medical College
    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 supportMedicinal Research Agency
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJagiellonian University Medical College
    B.5.2Functional name of contact pointClinical Trial Office
    B.5.3 Address:
    B.5.3.1Street AddressPodwale 3
    B.5.3.2Town/ cityKraków
    B.5.3.3Post code31-118
    B.5.3.4CountryPoland
    B.5.4Telephone number+4812370 43 30
    B.5.5Fax number+4812422 49 18
    B.5.6E-mailbadania.kliniczne@uj.edu.pl
    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 Yes
    D.2.1.1.1Trade name Qarziba 4,5 mg/ml concentrate for solution for infusion
    D.2.1.1.2Name of the Marketing Authorisation holderEUSA Pharma (Netherlands), B.V.,Beechavenue 54, 1119PW, Schiphol-Rijk, The Netherlands
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation number EU/3/12/1062
    D.3 Description of the IMP
    D.3.1Product nameDINUTUXIMAB BETA
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInfusion (Noncurrent)
    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) Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeMonoclonal antibody
    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
    Recurrent or progression of neuroblastoma or neuroblastoma refractory to first-line treatment
    Wznowa lub progresja neuroblastoma lub neuroblastoma oporna na leczenie pierwszej linii
    E.1.1.1Medical condition in easily understood language
    Neuroblastoma refractory to treatment
    Nerwiak zarodkowy oporny na leczenie
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    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 21.0
    E.1.2Level PT
    E.1.2Classification code 10066595
    E.1.2Term Neuroblastoma recurrent
    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 10029261
    E.1.2Term Neuroblastoma 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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To test the safety and efficacy of dinutuximab beta therapy in combination with chemotherapy for the treatment of patients with relapsed or progressive neuroblastoma and for first-line treatment of neuroblastoma refractory to standard therapy.
    The primary objective of the study is to evaluate the safety profile of 20 patients receiving dinutuximab beta in combination with chemotherapy compared to historical data from two reference groups:
    - group 1 for patients treated with chemotherapy without immunotherapy
    - group 2 for patients treated with immunotherapy without chemotherapy in patients with relapsed or progressive or primary resistance to treatment.
    The reference groups will consist of 2 groups of 10 patients who meet the inclusion criteria for the target group, but are treated only with chemotherapy (reference group 1) or only with immunotherapy (reference group 2) at the Department of Pediatric Oncology and Hematology in Krakow.
    Sprawdzenie bezpieczeństwa i skuteczności terapii dinutuksymabem beta w skojarzeniu z chemoterapią w leczeniu pacjentów ze wznową lub progresją neuroblastoma oraz w leczeniu pierwszej linii neuroblastoma opornego na leczenie standardowe.
    Celem pierwszorzędowym badania jest ocena profilu bezpieczeństwa 20 pacjentów przyjmujących dinutuksymab beta w skojarzeniu z chemioterapią w porównaniu do danych historycznych dwóch grup referencyjnych:
    - grupy 1 dotyczącej pacjentów leczonych z zastosowaniem chemioterapii bez immunoterapii
    - grupy 2 dotyczącej pacjentów leczonych z zastosowaniem immunoterapii bez chemioterapii u pacjentów ze wznową lub progresją lub pierwotną opornością na leczenie.
    Grupy referencyjne stanowić będą 2 grupy obejmujące po 10 pacjentów, spełniających kryteria włączenia do badania dla grupy docelowej, ale leczonych tylko chemioterapią (grupa referencyjna 1) lub tylko immunoterapią (grupa referencyjna 2) w Klinice Onkologii i Hematologii Dziecięcej w Krakowie.
    E.2.2Secondary objectives of the trial
    The secondary objective of the study is an initial efficacy assessment as a percentage of patients:

    1.complete remission assessed during the study and at the last visit,
    2.with partial remission assessed during the study and at the last visit,
    3.with disease stabilization assessed during the examination and at the last visit,
    4. PFS assessed during the study,
    5. ESF assessed during the study.
    In addition, the tertiary and exploratory objective of the study is to evaluate the usefulness of laboratory parameters in predicting response to dinutuximab beta therapy. Tertiary goals will not be subject to formal analysis.
    Celem drugorzędowym badania jest wstępna ocena skuteczności wyrażona jako odsetek pacjentów:

    1. z całkowitą remisją ocenianą w trakcie badania i na ostatniej wizycie,
    2. z częściową remisją ocenianą w trakcie badania i na ostatniej wizycie,
    3. z stabilizacją choroby ocenianą w trakcie badania i na ostatniej wizycie,
    4. PFS oceniany w trakcie trwania badania,
    5. EFS oceniany w trakcie trwania badania.
    Ponadto, trzeciorzędowym i eksploracyjnym celem badania jest ocena przydatności parametrów laboratoryjnych w predykcji odpowiedzi na leczenie dinutuksymabem beta. Cele trzeciorzędowe nie będą poddawane analizie formalnej.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of NBL according to international criteria (International Neuroblastoma Risk Group, INRG).
    2. Patients 1-18 years of age with HR-NBL with primary refractory disease, disease progression or recurrence.
    3. Adequate function of vital organs (if abnormal, dysfunction below grade 4 according to the CTC AE WHO classification, except for disorders defined in the exclusion criteria).
    4. Life expectancy ≥6 months.
    5. Obtaining the informed written consent of the patient and / or statutory representative for the treatment.
    6. Female patients of childbearing potential must consent to the use of effective contraception; Breastfeeding patients must consent to the termination of breastfeeding.
    7. Patients who have previously received immunotherapy with DB or other anti-GD2 specific antibodies may be eligible for this study.
    1. Rozpoznanie NBL zgodnie z międzynarodowymi kryteriami (International Neuroblastoma Risk Group, INRG).
    2. Pacjenci w wieku 1-18 lat z HR-NBL z chorobą pierwotnie oporną na leczenie, progresją lub wznową choroby.
    3. Adekwatna funkcja życiowo ważnych narządów (jeżeli funkcja nieprawidłowa, dysfunkcja poniżej 4 stopnia według klasyfikacji CTC AE WHO, z wyjątkiem zaburzeń zdefiniowanych w kryteriach wykluczenia).
    4. Przewidywany czas przeżycia ≥6 miesięcy.
    5. Uzyskanie świadomej pisemnej zgody pacjenta i/lub przedstawiciela ustawowego na zastosowanie leczenia.
    6. Pacjentki w wieku rozrodczym muszą wyrazić zgodę na stosowanie skutecznej metody antykoncepcji; pacjentki karmiące piersią muszą wyrazić zgodę na zakończenie karmienia.
    7. Pacjenci, którzy wcześniej otrzymywali immunoterapię z zastosowaniem DB lub innych przeciwciał o swoistości anty-GD2 mogą być zakwalifikowani do tego badania.
    E.4Principal exclusion criteria
    1. Patients with toxicities of ≥3 CTCAE WHO grade, except hearing impairment, hematological disorders, liver and kidney disorders.
    2. Patients with neurological toxicities of ≥2 CTCAE WHO grade.
    3. Active life-threatening infection until stabilization of the patient's condition.
    4. Pregnancy and / or lactation.
    5. Sexually active patients who refuse to use an effective method of contraception.
    6. Current treatment with experimental drugs or use of such treatment within 2 weeks before signing the informed consent to participate in the study.
    7. Radiotherapy within 3 weeks prior to the start of the study.
    8. Participation in another clinical trial within 6 months before signing the informed consent to participate in the trial (not applicable to clinical trials in 1st line of treatment in HR-NBL).
    9. Lack of informed written consent to treatment.
    1. Pacjenci z toksycznościami ≥3 stopnia CTCAE WHO, z wyjątkiem uszkodzenia słuchu, zaburzeń hematologicznych, zaburzeń funkcji wątroby i nerek.
    2. Pacjenci z toksycznościami neurologicznymi ≥2 stopnia CTCAE WHO.
    3. Aktywna infekcja zagrażająca życiu do czasu stabilizacji stanu pacjenta.
    4. Ciąża i/lub laktacja.
    5. Pacjenci aktywni seksualnie, którzy odmawiają stosowania skutecznej metody antykoncepcji.
    6. Aktualne leczenie z zastosowaniem leków eksperymentalnych lub stosowanie takiego leczenia w ciągu 2 tygodni przed podpisaniem świadomej zgody na udział w badaniu.
    7. Radioterapia w ciągu 3 tygodni przed rozpoczęciem badania.
    8. Udział w innym badaniu klinicznym w ciągu 6 miesięcy przed podpisaniem świadomej zgody na udział w badaniu (nie dotyczy badań klinicznych 1. linii leczenia HR-NBL).
    9. Brak świadomej pisemnej zgody na leczenie.
    E.5 End points
    E.5.1Primary end point(s)
    The following safety endpoints will be assessed:
    1.number of cycles aborted due to toxicity,
    2.number of cycles in which treatment interruptions due to the occurrence of side effects will be longer than provided for in the treatment protocol,
    3.number of episodes of Capillary Leak Syndrome, regardless of severity
    4.number of episodes of cytokine release syndrome, regardless of severity
    5.number of episodes of allergic reactions in CTCAE grade 3 and 4 (version in force at that time),
    6.number of hematological toxicities in grade 3 and 4 CTCAE (version in force at that time),
    7.Number of neurological toxicity episodes, regardless of severity
    8.the percentage of patients with pupil disorders and / or visual disturbances,
    9.proportion of patients with renal or hepatic impairment in CTCAE grade 3 and 4 (version in force at that time),
    10. Other side effects in grade 3 and 4 CTCAE (version in force at the time).
    Ocenione zostaną następujące punkty końcowe dotyczące oceny bezpieczeństwa:
    1. liczba cykli przerwanych z powodu toksyczności,
    2. liczba cykli, w których przerwy w leczeniu spowodowane wystąpieniem działań niepożądanych będą dłuższe niż przewidziane w protokole leczenia,
    3. liczba epizodów zespołu przesiąkania włośniczek, niezależnie od ciężkości,
    4. liczba epizodów zespołu uwalniania cytokin, niezależnie od ciężkości,
    5. liczba epizodów reakcji alergicznych w 3 i 4 stopniu CTCAE (wersja obowiązująca ówcześnie),
    6. liczba toksyczności hematologicznych w 3 i 4 stopniu CTCAE (wersja obowiązująca ówcześnie),
    7. liczba epizodów toksyczności neurologicznych, niezależnie od ciężkości,
    8. odsetek pacjentów z wystąpieniem zaburzenia źrenic i/lub zaburzeń widzenia,
    9. odsetek pacjentów z zaburzeniami funkcji nerek lub wątroby w 3 i 4 stopniu CTCAE (wersja obowiązująca ówcześnie),
    10. inne działania niepożądane w 3 i 4 stopniu CTCAE (wersja obowiązująca ówcześnie).
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the study and at the last visit - 12 months after end of treatment,
    Okres leczenia i okres 12 miesięcy po zakończeniu leczenia
    E.5.2Secondary end point(s)
    Percentage of patients:
    1.with complete remission assessed during the study and at the last visit,
    2.with partial remission assessed during the study and at the last visit,
    3.with disease stabilization assessed during the examination and at the last visit,
    4. PFS assessed during the study,
    5. ESF assessed during the study.
    Odsetek pacjentów:
    1. z całkowitą remisją ocenianą w trakcie badania i na ostatniej wizycie,
    2. z częściową remisją ocenianą w trakcie badania i na ostatniej wizycie,
    3. z stabilizacją choroby ocenianą w trakcie badania i na ostatniej wizycie,
    4. PFS oceniany w trakcie trwania badania,
    5. EFS oceniany w trakcie trwania badania.
    E.5.2.1Timepoint(s) of evaluation of this end point
    During the study and at the last visit - 12 months after end of treatment,
    Okres leczenia i okres 12 miesięcy po zakończeniu leczenia
    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 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 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 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 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 - Last Visit Last Subject
    Ostatnia wizyta wizyta ostatniego uczestnika badania klinicznego
    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 months0
    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: 28
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 20
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    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
    małoletniość
    minority
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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)
    Possible therapeutic strategy will depend on both the stage of chim used and the results of treatment. Possible variants: treatment for high or very high risk NBL, removal of the primary tumor, radiotherapy of the primary tumor area, high-dose MIBG therapy followed by autoHSC transplantation, immunotherapy with DB only and 13-cis retinoic acid, haploidentical transplant followed by DB immunotherapy, 3rd generation ALK inhibitors, termination of ChIm treatment or additional cycles of chim
    Możliwe postępowanie terapeutyczne będzie zależało zarówno od etapu zastosowanej ChIm, jak i uzyskanego wyniku . Możliwe warianty: leczenie dla protokołu NBL wysokiego lub b. wysokiego ryzyka, usunięcie guza pierwotnego, radioterapia okolicy guza pierwotnego, terapia MIBG z następowym autoHSC, immunoterapia z zastosowaniem tylko DB oraz 13-cisRA, przeszczepienie haploidentyczne, z immunoterapią DB, włączenie inhibitorów ALK 3 generacji, zakończenie leczenia ChIm lub dodatkowe cykle chim
    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 Decision2021-10-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-29
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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