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    Summary
    EudraCT Number:2015-000087-34
    Sponsor's Protocol Code Number:XM22-08
    National Competent Authority:Czech Republic - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-05-06
    Trial results View 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 ConcernedCzech Republic - SUKL
    A.2EudraCT number2015-000087-34
    A.3Full title of the trial
    An Open-Label, Randomized, Active-Controlled, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Immunogenicity of Lipegfilgrastim 100 μg/kg Body Weight in Comparison to Filgrastim 5 μg/kg Body Weight in Pediatric Patients Diagnosed with Ewing Family of Tumors or Rhabdomyosarcoma Receiving Chemotherapy
    Otevřená randomizovaná aktivně kontrolovaná multicentrická klinická studie hodnotící účinnost,
    farmakokinetiku, farmakodynamiku, bezpečnost, snášenlivost a imunogenicitu lipegfilgrastimu v dávce 100 μg/kg
    tělesné hmotnosti ve srovnání s filgrastimem v dávce 5 μg/kg tělesné hmotnosti u pediatrických pacientů
    s Ewingovým sarkomem nebo rabdomyosarkomem, kteří podstupují chemoterapii
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial comparing Lipegfilgrastim to Filgrastim in Pediatric Patients Diagnosed with Ewing Family of Tumors or Rhabdomyosarcoma Receiving Chemotherapy.
    A.4.1Sponsor's protocol code numberXM22-08
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/303/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMerckle GmbH
    B.1.3.4CountryGermany
    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.4.2CountryGermany
    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.5.3.4CountryGermany
    B.5.4Telephone number497314023891
    B.5.5Fax number497314027656
    B.5.6E-mailandreas.lammerich@ratiopharm.de
    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 LONQUEX
    D.2.1.1.2Name of the Marketing Authorisation holderSicor Biotech UAB
    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 nameLipegfilgrastim
    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 INNLipefilgrastim
    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.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.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 Neupogen 30 MU (0.3 mg/ml) solution for injection
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe B.V.
    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 nameNeupogen
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    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 INNFILGRASTIM
    D.3.9.1CAS number 121181-53-1
    D.3.9.4EV Substance CodeSUB07627MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.3
    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
    Neutropenia in pediatric patients diagnosed with Ewing family of Tumors or rhabdomyosarcoma receiving chemotherapy
    E.1.1.1Medical condition in easily understood language
    treatment of chemotherapy induced neutropenia (white blood cell decrease)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10029354
    E.1.2Term Neutropenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10016288
    E.1.2Term Febrile neutropenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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 objective of this study is to assess the efficacy of a single subcutaneous (sc) dose of 100 μg/kg body weight (BW) of lipegfilgrastim per cycle compared to daily 5 μg/kg BW of filgrastim doses in children receiving CTX
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to assess pharmacodynamics, pharmacokinetics, safety, tolerability, and immunogenicity of a single sc dose of 100 μg/kg BW of lipegfilgrastim per cycle compared to daily doses of 5 μg/kg BW of filgrastim.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a. Male or female children and adolescents aged 2 to <18 years at screening.
    b. 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.
    c. Able to understand and/or follow study instructions alone or with parental assistance.
    d. Diagnosed with the Ewing family of tumors, or rhabdomyosarcoma.
    e. Scheduled to receive 1 of the following CTX regimens (inpatient or outpatient):
    For the Ewing Family of Tumors:
    - Vincristine/Ifosfamide/Doxorubicin/Etoposide (VIDE); with concomitant sodium 2-mercaptoethane sulfonate (MESNA) according to local standards.
    - Vincristine/Doxorubicin/Cyclophosphamide alternating with Ifosfamide/Etoposide (VDC/IE); with concomitant MESNA treatment according to local standards.
    - Vincristine/Actinomycin/Cyclophosphamide (VAC); with concomitant MESNA treatment according to local standards.
    For Rhabdomyosarcoma:
    - VAC; Vincristine/Actinomycin/Cyclophosphamide (VAC); with concomitant MESNA treatment according to local standards.
    - VDC/IE; with concomitant MESNA treatment according to local standards.
    - Ifosfamide, Vincristine, and Actinomycin D (IVA); with concomitant MESNA treatment according to local standards.
    - Ifosfamide/Vincristine/Actinomycin/Doxorubicin (IVADo); with concomitant MESNA treatment according to local standards.
    f. Minimum BW 12.5 kg.
    g. Life expectancy of at least 3 months with appropriate therapy.
    h. White blood cell (WBC) count >2.5 × 10^9/L, absolute neutrophil count (ANC) ≥1.5 × 10^9/L, and platelet count ≥100 × 10^9/L (at screening and prior to CTX).
    i. For patients aged ≥12 years, Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
    j. Fertile patients (male or female) must use highly reliable contraceptive measures for the entire duration of the study.
    k. Female patients who have attained menarche must have a negative urine pregnancy test at the screening visit.
    E.4Principal exclusion criteria
    a. Previous exposure to filgrastim, pegfilgrastim, lenograstim or other granulocyte colony stimulating factors (G-CSFs) / granulocyte macrophage colony-stimulating factors (GM-CSFs) in clinical development within 6 weeks prior to the first lipegfilgrastim/filgrastim administration in this study.
    b. Known hypersensitivity to filgrastim, pegfilgrastim, lenograstim, any other G-CSF in clinical development or any LONQUEX (lipegfilgrastim) excipients.
    c. History of congenital neutropenia or cyclic neutropenia.
    d. Patients with a clinically significant or unstable medical or surgical condition that would preclude safe and complete study participation, as determined by medical history, physical examinations, electrocardiogram (ECG), laboratory tests, or imaging.
    e. Pregnancy or breast feeding (if a patient becomes pregnant during the study she will be withdrawn from the study).
    f. 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 first dose of study drug (lipegfilgrastim/filgrastim).
    g. Major surgery or serious infection within 3weeks before first administration of study drug (lipegfilgrastim/filgrastim), serious trauma or compound medical procedure within the 4 weeks prior to the first study drug dose.
    h. Treatment with lithium at screening or planned during the study.
    i. Participation in a clinical study with an investigational product within 30 days or 5 half-lives before randomization, whichever is longer.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the duration of severe neutropenia (DSN) in cycle 1, defined as the number of days with severe neutropenia in cycle 1 (from start of CTX until day 15). Severe neutropenia is defined as grade 4 neutropenia, with ANC <0.5 × 10^9/L.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Samples for ANC assessments in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit.
    E.5.2Secondary end point(s)
    Secondary Efficacy Variables and Endpoints:
    The secondary efficacy endpoints for this study are as follows:
    - Incidence of severe neutropenia (ANC <0.5 × 10^9/L) in each cycle (1-4).
    - Incidence of very severe neutropenia (ANC <0.1 × 10^9/L) in each cycle (1-4).
    - Incidence of febrile neutropenia (FN) (defined as body temperature >38.3°C or 2 consecutive readings higher than 37.8°C measured at axilla or external ear for 2 hours and ANC <0.5 × 10^9/L or expected to fall below 0.5 × 10^9/L) per cycle and across all cycles.
    - DSN in cycles 2-4 per cycle.
    - Duration of very severe neutropenia in cycles 1-4 per cycle.
    Efficacy evaluations will be performed on blood samples taken for the pharmacodynamic assessments in combination with temperature measurements.

    Secondary Pharmacodynamic Variables and Endpoints:
    - Area under the curve of ANC (AUCANC) until day 15 in cycle 1.
    - ANC nadir, ie, the lowest ANC value recorded, per cycle.
    - Time to ANC nadir per cycle, defined as the time from start of CTX until occurrence of the ANC nadir in the cycle.
    - Time to ANC nadir per cycle, defined as the time from first study drug administration in a cycle until occurrence of the ANC nadir in the cycle.
    - Time to ANC recovery (ANC >1.0 × 10^9/L and ANC >2.0 × 10^9/L) from first day of CTX.
    - Time to ANC recovery (ANC >1.0 × 10^9/L and ANC >2.0 × 10^9/L) from nadir per cycle.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Samples for PD assessments (ANC) in cycles 1 to 4 will be obtained on day 1 prior to study drug administration, on days 2, 4, 5, 6, 7, 8, 9, 10, 12, and 15 and at EOS/(or ET) visit.

    Continous tempareture measurements during the days 1-21 of each cycle.
    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) 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 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) Yes
    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 Yes
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Bulgaria
    Czech Republic
    Germany
    Hungary
    Lithuania
    Poland
    Romania
    Russian Federation
    Slovakia
    Spain
    Ukraine
    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
    LVLS
    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 months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months11
    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: 42
    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: 28
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 14
    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 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 27
    F.4.2.2In the whole clinical trial 42
    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
    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 Decision2015-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-27
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-04-18
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