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    EudraCT Number:2010-019340-40
    Sponsor's Protocol Code Number:MOZ15609-DFI12860
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2016-05-10
    Trial results View results
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    A. Protocol Information
    A.2EudraCT number2010-019340-40
    A.3Full title of the trial
    A Phase 1/2 Combined Dose Ranging and Randomised, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilisation of Haematopoietic Stem Cells into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilisation Regimens Alone in Paediatric Patients, Aged 1 to <18 Years, with Solid Tumours Eligible for Autologous Transplants
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
    A.4.1Sponsor's protocol code numberMOZ15609-DFI12860
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01288573
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/253/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Europe B.V.
    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 supportGenzyme Europe B.V.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenzyme Europe B.V.
    B.5.2Functional name of contact point-
    B.5.3 Address:
    B.5.3.1Street Address-
    B.5.3.2Town/ city-
    B.5.3.3Post code-
    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. name Mozobil
    D. of the Marketing Authorisation holderGenzyme Europe BV
    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 Yes
    D.2.5.1Orphan drug designation numberEu/3/04/227
    D.3 Description of the IMP
    D.3.1Product namePlerixafor
    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 INNplerixafor
    D.3.9.1CAS number 110078-46-1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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. 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 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
    Paediatric cancer patients (aged 1 to <18 years) with Ewing’s sarcoma/soft tissue sarcoma, lymphoma, neuroblastoma and all other malignancies (excluding leukaemia) who are planned to undergo high dose chemotherapy followed by autologous haematopoietic stem cell transplantation (HSCT) rescue
    E.1.1.1Medical condition in easily understood language
    Solid tumours (excluding leukaemia) which will be treated with high dose chemotherapy followed by transplantation of the child's own stem cells.
    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 this study is to confirm the appropriate dose and efficacy, and to characterise the safety, pharmacokinetics and pharmacodynamics of plerixafor across age and size in paediatric cancer patients when given in addition to standard mobilisation of HSCs into peripheral blood.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to confirm the efficacy and safety of plerixafor in addition to standard mobilisation of HSCs into peripheral blood, and subsequent collection by apheresis, in paediatric cancer patients.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 1 to <18 years
    2. Ewing’s sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, or other malignancy including brain tumours
    (excluding any form of leukaemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
    3. Eligible for autologous transplantation
    4. Recovered from all acute significant toxic effects of prior chemotherapy
    5. Adequate performance status
    – for patients ≥16 years of age, defined as Karnofsky score >60
    – for patients <16 years of age, defined as Lansky score >60
    6. Absolute neutrophil count >0.75 × 10P9/L
    7. Platelet count >50 × 10P9/L
    8. Calculated creatinine clearance (using the Schwartz method):
    – during study Stage 2, >60 mL/min/1.73mP2
    9. Liver functions <3 × upper limit of normal :
    Aspartate aminotransferase/serum glutamic oxaloacetic transaminase
    (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase
    (ALT/SGPT) and total bilirubin
    10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
    11. Patients who are sexually active must be willing to abstain from sexual intercourse or agree to use an approved form of contraception while receiving plerixafor and/or standard mobilisation treatment and for at least 3 months following any plerixafor treatment.
    E.4Principal exclusion criteria
    1. Any form of leukaemia
    2. A co-morbid condition, such as ventricular arrhythmias, which, in the view of the Investigator, renders the patient
    at high-risk from treatment complications
    3. Previous stem cell transplantation
    4. Patients with persistent high percentage marrow involvement prior to mobilisation will be prohibited.
    5. On-going toxicities (excluding alopecia) Grade ≥2 resulting from prior chemotherapy
    6. Acute infection
    7. Fever (temperature >38.5°C) - if fever is between 37°C and 38.5°C, infection must be excluded as a cause
    8. Known HIV seropositivity, AIDS, hepatitis C or active hepatitis B infections.
    9. Positive pregnancy test in post pubertal girls
    10. History of clinically significant cardiac abnormality or arrhythmia
    11. Use of an investigational drug which is not approved in any indication either in
    adults or paediatrics within 2 weeks prior to the first dose of G-CSF to be administered as part of the patient’s planned standard mobilisation regimen, and/or during the study up until engraftment of the transplant. If patients are on
    investigational drugs as part of their anti-cancer regimen, this should be discussed with the Sponsor before screening. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed
    12. The patient (and/or their parent/legal guardian), in the opinion of the Investigator,
    is unable to adhere to the requirements of the study
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the difference between the 2 treatment arms in Stage 2 (comparative part of the study) in the proportion of patients achieving at least a doubling of peripheral blood CD34+ count from the morning of the day preceding the first apheresis day to the morning prior to apheresis.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 5 days
    E.5.2Secondary end point(s)
    •Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    •Yield of CD34+ cells for each apheresis [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2
    •Total CD34+ cell yield [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    •Percentage of patients proceeding to transplant [ Time Frame: Within 6 months of last apheresis ] During Stage 1 and Stage 2
    •Percentage of patients successfully engrafting [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    •Percentage of patients with durable engraftment [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    •Summary of adverse events (AEs) [ Time Frame: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis) ] During Stage 1 and Stage
    •Duration of hospitalizations (planned or unplanned) [ Time Frame: Throughout the duration of the study ] During Stage 1 and Stage 2
    •Mobilization of tumor cells into peripheral blood [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    •Relapse rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2
    •Occurrence of secondary malignancies [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    •Incidence of primary and secondary graft failure [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    •Time to secondary graft failure [ Time Frame: Up to 24 months posttransplant] During Stage 1 and Stage 2
    •Survival rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2
    E.5.2.1Timepoint(s) of evaluation of this end point
    see section E.5.2.
    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 No
    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
    Stage 1 only: dose ranging
    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
    Stage 1 only: dose ranging - Stage 1 completed
    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 Yes
    E. trial design description
    Stage 2 only: a comparative randomised study
    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. description
    Stage 2 only: standard mobilisation treatment
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Czech Republic
    United Kingdom
    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.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    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: 70
    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. of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 50
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 19
    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. of subjects incapable of giving consent
    Children age 1-18 Parents sign ICF
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 70
    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)
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Israel
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