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    EudraCT Number:2011-004742-18
    Sponsor's Protocol Code Number:XM22-07
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-02-07
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2011-004742-18
    A.3Full title of the trial
    Multicenter, Open-label Study to Assess the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, Tolerability, and Immunogenicity of a Single, Subcutaneous Dose of 100μg/kg XM22 in 21 Children with Ewing Family of Tumors or Rhabdomyosarcoma
    Többközpontú, nyílt vizsgálat, egyetlen 100 µg/kg-os adag, bőr alá adott XM22 injekció farmakokinetikájának, farmakodinámiájának, hatásosságának, biztonságosságának, tolerálhatóságának, valamint immunogenitásának vizsgálatára 21 gyermeknél, akik Ewing típusú szarkómában, vagy rhabdomyo-szarkómában szenvednek
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Assess the Pharmacokinetics (absorption, distribution, metabolisation, excretion), Pharmacodynamics (biochemical and physiological effects of the drug), Efficacy, Safety, Tolerability, and Immunogenicity (Immune response) of a Single, Subcutaneous Dose of 100μg/kg XM22 in 21 Children with Ewing Family of Tumors or Rhabdomyosarcoma
    Egyetlen 100 µg/kg-os adag, bőr alá adott XM22 injekció farmakokinetikájának, farmakodinámiájának, hatásosságának, biztonságosságának, tolerálhatóságának, valamint immunogenitásának vizsgálata 21 gyermeknél, akik Ewing típusú szarkómában, vagy rhabdomyo-szarkómában szenvednek
    A.4.1Sponsor's protocol code numberXM22-07
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/028/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMerckle GmbH, a member of the ratiopharm group, a subsidiary of Teva Pharmaceutical Industries Ltd. Germany
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMerckle GmbH
    B.4.1Name of organisation providing supportTeva ratiopharm
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMerckle GmbH
    B.5.2Functional name of contact pointDr med Andreas Lammerich
    B.5.3 Address:
    B.5.3.1Street AddressGraf-Arco-Strasse 3
    B.5.3.2Town/ cityUlm
    B.5.3.3Post code89079
    B.5.4Telephone number+497314023891
    B.5.5Fax number+497314027656
    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.3.2Product code XM22
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLipegfilgrastim
    D.3.9.1CAS number 1117844-87-7
    D.3.9.2Current sponsor codeXM22
    D.3.9.3Other descriptive nameglyco-PEGylated r-metHuG-CSF
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Children with Ewing Family of Tumors or Rhabdomyosarcoma receiving cytotoxic Chemotherapy for malignancy inducing neutropenia
    Kemoterápia okozta neuropénia Ewing tumoros vagy rhabdoszarkómás gyerekeknél
    E.1.1.1Medical condition in easily understood language
    Children with special cancer of the bone or soft tissue or cancer of muscle or connective tissue receiving drug cancer treatment inducing white blood cell decrease
    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 assess the pharmacokinetics (PK) of a single subcutaneous (SC) injection of XM22, 100 μg/kg body weight (BW), in children with Ewing family of tumors or rhabdomyosarcoma.
    A vizsgálat elsődleges célja az egyetlen adagban, bőr alá adott (SC), 100 µg/testtömegkilogramm (TT), XM22 injekció farmakokinetikájának (PK) mérése olyan gyermekeknél, akik Ewing típusú szarkómában, vagy rhabdomyo-szarkómában szenvednek.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to assess the pharmacodynamics (PD), efficacy, safety, tolerability, and immunogenicity of this single dose in the same patient population.
    A vizsgálat másodlagos céljai, hogy mérjük ugyanebben a populációban ennek az egyetlen adagnak a farmakodinámiáját, (PD), hatékonyságát, biztonságosságát, tolerálhatóságát és immunogenitását
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female children and adolescents aged 2 to <18 years
    2. Written informed consent provided by parent(s)/legal
    representative(s) of the pediatric patient and patient's assent if appropriate, before screening procedures are initiated.
    3. Able to understand and/or follow study instructions alone or with
    parental assistance
    4. Diagnosed with the Ewing family of tumors or rhabdomyosarcoma
    5. Scheduled to receive 1 of the following chemotherapy regimens
    (inpatient or outpatient)
    • For the Ewing family of tumors:
    - vincristine/ifosfamide/doxorubicin/etoposide (VIDE); concomitant
    treatment with sodium 2-mercaptoethane sulfonate (MESNA) according
    to local standards
    - vincristine/doxorubicin/cyclophosphamide alternating with
    ifosfamide/etoposide (VDC/IE); concomitant treatment with MESNA
    according to local standards
    • For rhabdomyosarcoma:
    - vincristine/actinomycin/cyclophosphamide (VAC), concomitant treatment with MESNA according to local standards
    - vincristine/doxorubicin/cyclophosphamide alternating with ifosfamide/etoposide (VDC/IE); concomitant treatment with MESNA according to local standards
    - ifosfamide, vincristine and actinomycin D (IVA),concomitant treatment with MESNA according to local standards

    7. Body weight ≥15 kg for the 2 highest age strata and ≥12.5 kg for patients in the lowest age stratum
    8. White blood cell (WBC) count >2.5 x 10(9)/L, absolute neutrophil
    count (ANC) ≥1.5 x 10(9)/L, and platelet count ≥100 x 10(9)/L (at
    screening and prior to CTX).
    9. For patients aged ≥12 years, Eastern Cooperative Oncology Group
    (ECOG) performance status ≤2
    10. Fertile patients (male or female) must use highly reliable
    contraceptive measures (i.e. two of the following: oral contraception,
    implants, injections, barrier contraception, and intrauterine device, or
    vasectomized/sterilized partners, or sexual abstinence). For purposes
    of this study, a fertile female patient is any female patient who has
    experienced menarche and who has not undergone tubal ligation.
    11. Female patients who have attained menarche must have a negative
    urine pregnancy test at the screening visit.

    1.Férfi- és nőbetegek, kisgyermek korúak és serdülők 2 és <18 év között.
    2.Írásos beleegyező nyilatkozat a szülő(k)től, vagy törvényes képviselő(k)től, és, ha lehetséges, a gyermek egyetértő nyilatkozata még a szűrővizsgálati eljárások megkezdése előtt.
    3.Önállóan, vagy szülői segítséggel képes megérteni és/vagy követni a vizsgálattal kapcsolatos utasításokat.
    4.Ewing típusú szarkómával, vagy rhabdomyo-szarkómával diagnosztizált beteg.
    5.A tervek szerint a következő kemoterápiás protokollok egyikét fogja kapni (akár kórházi, akár járóbeteg ellátásban):
    •A Ewing típusú szarkóma esetén:
    -vincristin/ifoszfamid/doxorubicin/etoposid (VIDE); egyidejűleg alkalmazott kezelés nátrium 2-merkaptoetán szulfonáttal (MESNA a helyi előírások szerint
    -vincristin/doxorubicin/ciklofoszfamid váltakozva ifoszfamid/etoposiddal (VDC/IE); egyidejűleg a helyi előírások szerint alkalmazott MESNA kezeléssel
    •A rhabdomyo-szarkómára:
    -vincristin/actinomycin/ ciklofoszfamid (VAC), egyidejűleg a helyi előírások szerint alkalmazott MESNA kezeléssel
    -vincristin/doxorubicin/ ciklofoszfamid, váltakozva ifosfamide/etoposiddal (VDC/IE); egyidejűleg a helyi előírások szerint alkalmazott MESNA kezeléssel
    -ifoszfamid, vincristin és actinomycin D (IVA), egyidejűleg a helyi előírások szerint alkalmazott MESNA kezeléssel
    7.Testsúly ≥15 kg a két legidősebb korcsoportban és ≥12.5 kg a legfiatalabb korosztálynál8.Fehérvérsejt (fvs) szám >2.5 x 109/L, abszolút neutrofil szám (ANC) ≥1.5 x 109/L, és thrombocyta szám ≥100 x 109/L (szűréskor és a CTX-et megelőzően).
    9.A ≥12 éves betegeknél, Eastern Cooperative Oncology Group (ECOG) státusz ≤2
    10.Fogamzóképes betegek (mind a férfi, mind a nőbetegek) kötelesek nagyon megbízható fogamzásgátló módszert alkalmazni (azaz egyszerre kettőt a felsoroltakból: szájon át szedett fogamzásgátló tabletta, implantátum, injekció, barrier módszer, valamint méhen belüli eszköz, vagy vazektómián/sterilizáláson átesett partner, vagy tartózkodás a nemi élettől). A jelen vizsgálat céljaira a fogamzóképes nőbeteg alatt bármely olyan nő értendő, aki már túl van az első menstruáción és nem kötötték el a petevezetékét.
    11.Annak a nőbetegnek, aki már túl van az első menstruáción, vizeletből vizsgált negatív terhességi teszttel kell rendelkeznie a szűrővizsgálat idején.
    E.4Principal exclusion criteria
    1. Previous exposure to filgrastim, pegfilgrastim or lenograstim or
    other G-CSFs in clinical development within 6 months prior to the XM22
    2. Known hypersensitivity to filgrastim, pegfilgrastim or lenograstim or
    any other G-CSF in clinical development
    3. History of congenital neutropenia or cyclic neutropenia
    4. Any illness or condition that in the opinion of the Investigator may
    affect the safety of the patient or the evaluation of any study endpoint
    5. Pregnant or nursing women
    6. Fertile patients who do not agree to use highly reliable contraceptive
    measures during the entire duration of the study
    7. Prior bone marrow or stem cell transplant, or prior radiation to ≥
    25% of bone marrow (eg, whole pelvic radiation) for any reason, or
    any therapeutic radiation within the 3 weeks prior to the XM22 dose
    8. Ongoing active infection or history of infectious disease within 2
    weeks prior to the screening visit
    9. Treatment with lithium at screening or planned during the study.
    1.Az XM22 beadását megelőző 6 hónapon belül filgrastimot, pegfilgrastimot, vagy lenograstimot, illetve más, klinikai vizsgálatban alkalmazott G-CSF-eket kapott
    2.Ismert hiperszenzitivitás a filgrastimra, pegfilgrastimra, vagy lenograstimra, illetve bármely, klinikai vizsgálatban alkalmazott G-CSF-re
    3.Veleszületett neutropénia, vagy ciklikus neutropénia
    4.Bármely betegség, vagy állapot, amely a Vizsgáló megítélése szerint befolyásolhatja a beteg biztonságát, vagy a vizsgálat valamely végpontjának értékelését.
    5.Terhes, vagy szoptató nők
    6.Olyan fogamzóképes betegek, akik nem egyeznek bele, hogy nagyon megbízható fogamzásgátló módszert használjanak a vizsgálat teljes időtartama alatt.
    7.Az XM22 injekciót megelőző 3 hétben csontvelő transzplantáción, vagy őssejt transzplantáción estek át, illetve a csontvelő ≥25% -ára kiterjedő (pl. teljes medence) besugárzást kaptak bármilyen ok miatt, vagy bármilyen terápiás célú besugárzásban részesültek
    8.Aktív fertőzésük van jelenleg, vagy a szűrővizitet megelőző 2 hétben volt fertőző betegségük.
    9.Lítiumos kezelést kapnak a szűrés idején, illetve ilyet terveznek végezni a vizsgálat során.
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of pharmacokinetics of single dose XM22
    Farmakokinetikai értekelés az XM22 egyszeri adagolása után
    E.5.1.1Timepoint(s) of evaluation of this end point
    Pharmacokinetic profile up to 240 hours postdose
    a PK értékeléshez a mintavétel rendje a következő: az injekció előtt, majd 3, 8, 24, 30, 48, 72, 96, 144, 240 órával az XM22 beadása után (mindösszesen10 minta)
    E.5.2Secondary end point(s)
    Pharmacodynamic (ANC, CD34+), Efficacy, Safety, Tolerability,
    Parmakodinámia(ANC, CD34+), hatásosság, biztonság,tolerálhatóság,immunogenitás
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacodynamic (ANC, CD34+) until day 15
    Efficacy, Safety, Tolerability until day 21
    Immunogenicity until day 21 and follow-up on day 180 and day 360
    Parmakodinámia(ANC, CD34+) 15 napig
    hatásosság, biztonság,tolerálhatóság -21 napig,
    immunogenitás 21 napig és utánkövetés 180 napon és 360 napon.
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability, Immunogenicity
    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. trial type description
    Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, Tolerability, and Immunogenicity
    farmakokinetika, farmakodinámika, hatásosság, biztonságosság, tolerálhatóság
    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 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.8.2.4Number of treatment arms in the trial1
    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 EEA4
    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
    Russian Federation
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial 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: 21
    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. of subjects for this age range: 14
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 7
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 66
    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)
    In case of study drug related adverse events, these will be followed up until solution.
    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 Decision2012-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-04-21
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