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    Summary
    EudraCT Number:2015-002468-18
    Sponsor's Protocol Code Number:EZH-102
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2015-12-15
    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 ConcernedGermany - BfArM
    A.2EudraCT number2015-002468-18
    A.3Full title of the trial
    A Phase I Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study on the Effect of Tazemetostat (study drug) in Pediatric Subjects with relapsed or refractory Tumors or Synovial Sarcoma
    A.4.1Sponsor's protocol code numberEZH-102
    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 SponsorEpizyme, Inc.
    B.1.3.4CountryUnited States
    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 supportEpizyme, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEpizyme, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address400 Technology Square, 4th Floor
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number001855500-1011
    B.5.6E-mailclinicaltrials@epizyme.com
    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.3.1Product nametazemetostat
    D.3.2Product code EPZ-6438 (E7438)
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTAZEMETOSTAT
    D.3.9.2Current sponsor codeEPZ-6438
    D.3.9.3Other descriptive nameESQR
    D.3.9.4EV Substance CodeSUB178719
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 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.3.1Product nametazemetostat
    D.3.2Product code EPZ-6438 (E7438)
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTAZEMETOSTAT
    D.3.9.2Current sponsor codeEPZ-6438
    D.3.9.3Other descriptive nameESQR
    D.3.9.4EV Substance CodeSUB178719
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number7
    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.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
    Atypical teratoid rhabdoid tumor (ATRT), Malignant rhabdoid tumor (MRT), Rhabdoid tumor of kidney (RTK).

    Outside of Germany :
    - selected tumors with rhabdoid features;
    - INI-negative tumors or synovial sarcoma: Epithelioid sarcoma, Epithelioid malignant peripheral nerve sheath tumor, Extraskeletal myxoid chondrosarcoma, Myoepithelial carcinoma, Renal medullary carcinoma, other INI1-negative malignant tumors (like dedifferentiated chordoma), Synovial Sarcoma with a SS18-SSX rearrangement.
    E.1.1.1Medical condition in easily understood language
    Relapsed/refractory rhabdoid tumors and central nervous system (CNS) tumors.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10064886
    E.1.2Term Renal medullary carcinoma
    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 LLT
    E.1.2Classification code 10065870
    E.1.2Term Atypical teratoid/rhabdoid tumor of CNS
    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 10073134
    E.1.2Term Extraskeletal myxoid chondrosarcoma
    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 21.1
    E.1.2Level LLT
    E.1.2Classification code 10073335
    E.1.2Term Rhabdoid tumor
    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 10074121
    E.1.2Term Rhabdoid tumor of the kidney
    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 PT
    E.1.2Classification code 10042863
    E.1.2Term Synovial sarcoma
    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 HLT
    E.1.2Classification code 10015100
    E.1.2Term Epithelioid sarcomas
    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
    Dose Escalation:
    To determine the maximum tolerated dose (MTD) or the recommended Phase 2 dose (RP2D) of tazemetostat when administered as an oral suspension twice daily (BID) in pediatric subjects with relapsed/refractory rhabdoid tumors, (outside of Germany to include integrase interactor 1 (INI1)-negative tumors or synovial sarcoma).

    Dose Expansion:
    To evaluate the antitumor activity of tazemetostat as assessed by overall response rate (ORR) in pediatric subjects with relapsed/refractory atypical teratoid rhabdoid tumor (ATRT) (Cohort 1), non-ATRT rhabdoid tumors (Cohort 2), INI1-negative tumors (Cohort 3)(outside of Germany ) and tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (Cohort 4 [outside of Germany]) using disease appropriate standardized response criteria.

    E.2.2Secondary objectives of the trial
    Dose Escalation: To evaluate the preliminary antitumor activity of tazemetostat as assessed by ORR using disease appropriate standardized response criteria.
    Dose Expansion: To determine the progression-free survival (PFS) & overall survival (OS) at 24 and 56 weeks & overall pediatric subjects with relapsed/refractory atypical teratoid rhabdoid tumor (ATRT) (Cohort 1), non-ATRT rhabdoid tumors (Cohort 2), INI1-negative tumors or synovial sarcoma ((Cohort 3) outside of Germany),& tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (Cohort 4 [outside of Germany]) using disease-appropriate standardized response criteria.
    ALL subjects:
    - To assess safety&tolerability and PK parameters of tazemetostat administered as an oral suspension BID in pediatric subjects;
    -To evaluate the duration of response in subjects achieving a PR or CR according to disease appropriate standardized response criteria.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Age (at the time of consent/assent): ≥6 months to <18 years of age Cohort 4 only: ≥10 y to <18 y
    2.Performance Status:If <12 y: Lansky Performance Status >50% ,If ≥ 12 y of age: Karnofsky Performance Status >50%
    3.Has provided signed written ICF 4. Has a life expectancy of >3 months. 5.Has relapsed or refractory disease and no standard treatment
    options. 6.Is ineligible for other treatment regimens. 7.Has a documented local diagnostic pathology of original biopsy. 8.Has all prior treatment related
    clinically significant toxicities resolve to ≤ Grade 1 per CTCAE, version 4.03 or are clinically stable& not clinically significant. 9.Prior therapies
    must be completed according to the protocol. 10.Has adequate hemat. BM & coagulation factors, renal&hepatic function as defined by criteria in
    the protocol 11.Specific requirements for subjects with CNS involvement eg: stable deficits within certain timeframe, stable seizure, treated brain
    metastases without evidence of progression. 12.Has a LV fractional shortening of >27% or an LV ejection fraction of ≥50% by ECHO or
    MUGA scan & NYHAC ≤2. 13. Has a QT interval corrected by Fridericia's formula (QTcF) ≤450 msec. 14.Is able to swallow and retain orally administered medication and does not have any uncontrolled GI condition that may alter absorption. 15.Has sufficient tumor tissue for central confirmatory
    testing of IHC and/or cytogenetics/FISH and/or DNA mutation analysis. 16.Is willing and able to comply with all aspects of the protocol. 17.18.
    For female subjects of childbearing potential and for male subjects with a female partner of childbearing potential Subject must adhere to
    contraception methods described in the protocol. For Dose Escalation Only:ALL criteria above and the following:1.Has evaluable disease as defined as lesions that can be accurately measured at least in one dimension by radiographic examination or physical examination or and other lesions such as bone lesions, leptomeningeal disease, ascites, pleural/pericardial effusions, lymphangitis cutis/pulmonitis or hepatosplenomegaly from disease2.Has one of the
    following histologically confirmed tumors: Rhabdoid tumor: ATRT (closed to enrollment), MRT, RTK, selected tumors with rhabdoid features, INI1-
    negative tumor: (Epithelioid sarcoma, Epithelioid malignant peripheral nerve sheath tumor, EMC, Myoepithelial carcinoma, Renal medullary
    carcinoma), other INI1-negative malignant tumors, Synovial sarcoma with SS18-SSX rearrangement (closed to enrollment).
    3. For subjects with ATRT, MRT, and RTK or tumors with rhabdoid features only: the following test results must be available:
    •Morphology and immunophenotypic panel consistent with rhabdoid tumor, and •Loss of INI1 or SMARCA 4 confirmed by IHC, or
    •Molecular confirmation of tumor bi-allelic INI1 or SMARCA 4 loss or mutation when INI1 or SMARCA4 IHC is equivocal or unavailable
    4. For subjects with INI1 negative tumor only the following test results must be available:
    •Morphology and immunophenotypic panel consistent with INI1 negative tumors, and
    •Loss of INI1 confirmed by IHC, or
    •Molecular confirmation of tumor bi-allelic INI1 loss or mutation when INI1 IHC is equivocal or unavailable 5.For subjects with synovial sarcoma with SS18-SSX rearangement only the following test results must be available:
    •Morphology consistent with synovial sarcoma, and
    •Cytogenetics or FISH and/or molecular confirmation (e.g.DNA sequencing) of SS18 rearrangement t(X;18)(p11;q11) For Dose Expansion Only: ALL listed above for All subjects and in addition:
    1. Has measurable disease 2.Has one of the following tumors: Cohort 1: ATRT-(enrollment closed), Cohort 2: MRT, RTK, Selected tumors with
    rhabdoid features, Cohort 3:INI1-negative tumors or synovial sarcoma: ES, Epithelioid malignant peripheral nerve sheath tumor, EMC,
    Myoepithelial carcinoma, Renal medullary carcinoma, Chordoma (poorly differentiated or de-differentiated), other INI1-negative malignant
    tumors, Cohort 4 (enrollment closed):one of the tumor types defined in Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement.
    3.For subjects with ATRT/MRT/RTK only -have test results available:
    •Morphology and immunophenotypic panel consistent with rhabdoid tumor, and •Loss of INI1 or SMARCA4 confirmed by IHC, or •Molecular
    confirmation of tumor bi-allelic INI1 or SMARCA4 loss or mutation. 4. For subjects with INI1-negative tumors only – test results available: •
    Morphology and immunophenotypic panel consistent with INI1-negative tumors, and •Loss of INI1 confirmed by IHC, or •Molecular confirmation
    of tumor bi-allelic INI1 loss/mutation. 5.For subjects with synovial sarcoma with SS18-SSX rearrangement (in Cohort 4 only- closed to enrollment): Morphology consistent with synovial sarcoma & Cytogenetics or FISH &/or molecular confirmation of SS18 rearrangement t(X;18)(p11;q11) 6.For subjects Cohort 4-(closed to enrollment): Able to swallow&retain orally taken tablets
    E.4Principal exclusion criteria
    1.Has had prior exposure to tazemetostat or other inhibitor(s) of enhancer of zeste homolog2 (EZH2)
    2.Is being actively treated for another concurrent malignancy or is less than five years from completion of treatment for another malignancy
    3.Has participated in another interventional clinical study and received investigational drug within 30 days or five half-lives, whichever is longer, prior to the planned first dose of tazemetostat
    4.Has had major surgery within 2 weeks prior to enrollment
    5.Has thrombocytopenia, neutropenia, or anemia of Grade ≥3 (per CTCAE 4.03 criteria) or any prior history of myeloid malignancies,
    including myelodysplastic syndrome (MDS). Has abnormalities known to be associated with MDS (e.g del 5q, chr 7 abn) and MPN (e.g. JAK2 V617F) observed in cytogenetic testing and DNA sequencing.
    Note: Bone marrow aspirate/biopsy will be conducted following abnormal peripheral blood smear morphology assessment at Screening.
    Cytogenetic testing and DNA sequencing will be conducted by a central laboratory following an abnormal result of bone marrow aspirate/biopsy.
    6.Has a prior history of T-cell lymphoblastic lymphoma/T-cell acute
    lymphoblastic lymphoma (T-LBL/T-ALL).
    7.Has clinically active heart disease including prolonged QTcF (>450 msec)
    8.Is currently taking any prohibited medication(s) as described in section 7.3
    9.Is unwilling to exclude grapefruit juice, Seville oranges and grapefruit from the diet and all foods that contain those fruits from time of enrollment to while on study
    10.Has an active infection requiring systemic treatment
    11.Is immunocompromised (ie congenital immunodeficiency), including subjects with a known history of infection with human immunodeficiency virus (HIV)
    12.Has known history of chronic infection with hepatitis B virus (hepatitis B surface antigen positive) or hepatitis C virus (detectable HCV RNA)
    13.Has had a symptomatic venous thrombosis within 14 days prior to study enrollment
    14.For subjects with CNS involvement (primary tumor or metastatic disease): Have any active bleeding, or new intratumoral hemorrhage of more than punctate size on Screening MRI obtained within 14 days of starting study drug,or known bleeding diathesis or treatment with anti-platelet or anti-thrombotic agents
    15.Has known hypersensitivity to any of the components of tazemetostat or other inhibitor(s) of EZH2, or hypersensitivity to Ora-sweet or methylparaben
    16.Has an uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, or psychiatric illness/social situations that would limit compliance with study requirements
    17.For female subjects of childbearing potential: Is pregnant or nursing
    For male subjects: Is unwilling to adhere to contraception criteria from time of enrollment in study to at least 3 months after last dose of tazemetostat.
    E.5 End points
    E.5.1Primary end point(s)
    Dose Escalation:
    •Incidence and severity of treatment -emergent AEs (TEAEs) qualifying as protocol-defined dose-limiting toxicities (DLTs), and establishment of the protocol-defined RP2D and/or MTD.
    Dose Expansion:
    •Overall response rate CR + PR to tazemetostat in pediatric subjects with relapsed or refractory atypical teratoid rhabdoid tumor (ATRT) (Cohort 1), non-ATRT rhabdoid tumors (Cohort 2), (outside of Germany) INI1-negative tumors (Cohort 3), and tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (Cohort 4 [outside Germany only]) using disease- appropriate standardized response criteria .
    E.5.1.1Timepoint(s) of evaluation of this end point
    Dose Escalation: All DLTs occurring during the first 28 days of exposure summarized.
    Dose Expansion: Data sets for each cohort will be reviewed as completed.
    timepoint for every 8 weeks until documented disease progression or new anti-cancer therapy started.
    E.5.2Secondary end point(s)
    Dose Escalation:
    •Overall response rate (CR+PR) to tazemetostat in pediatric subjects with relapsed or refractory CNS and solid tumors, using disease-appropriate standardized response criteria;
    Dose Expansion:
    •Progression-free survival (PFS) and overall survival (OS) at 24 and 56 weeks and overall following receipt of tazemetostat for subjects with relapsed/refractory atypical teratoid rhabdoid tumor (ATRT) (Cohort 1), non-ATRT rhabdoid tumors (Cohort 2), (outside of Germany) INI1-negative tumors (Cohort 3), and tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (Cohort 4 [outside of Germany]) using disease-appropriate standardized response;
    All Parts and Cohorts:
    •Safety and tolerability parameters including treatment-emergent adverse events (TEAEs), clinical laboratory evaluations, and other safety measures
    •PK parameters including Cmax, Tmax, t1/2, AUC(0-t), AUC(0-12hr), CL/F, Vd/F, Ka (if data permit)
    •Response duration, for the subset of subjects with a confirmed CR or PR, defined as the time from the first documented evidence of CR or PR to time of first documented disease progression or death due to any cause, using disease appropriate standardized response criteria.
    Exploratory Endpoints:
    •To assess the PK and pharmacodynamic (PD) relationship for tazemetostat in pediatric subjects
    •Tumor target gene expression and phenotypic markers including those for differentiation, apoptosis, inflammation, and cell proliferation and
    their correlation with activity
    •H3K27 methylation in PBMC population
    •Somatic mutation analysis of tumor tissue and blood derived circulating DNA
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Dose Escalation
    ORR: every 8 weeks for tumor response until disease progression
    2.Dose Expansion
    PFS: every 8 weeks until disease progression or death
    OS: throughout the treatment period and then every 16 weeks until death, withdrawal of consent or they are lost to follow up
    3.All Groups
    •Safety: At each visit or required timepoint during treatment and until for 30 days after last dose of tazemetostat or until new anti-cancer therapy
    •PK parameters: Cycle 1 (Days 1 and 15) and Cycles 2, 3 and 4 (Day 1).

    •Response duration: every 8 weeks from the time of confirmed response (CR or PR) until disease progression or death.
    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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    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.7.1.3.1Other trial type description
    Dose Escalation and Dose Expansion
    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 Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Australia
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    United Kingdom
    United States
    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
    The end of study will occur when the last subject discontinues the study treatment and has had to opportunity to complete the safety follow-up visit or the long-term survival follow-up period, whichever is later. The study may end prior to this if 80% of enrolled subjects are deceased prior to the collection of the last survival follow-up.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    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 years8
    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: 111
    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: 37
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 37
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 37
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 120
    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)
    Treatment may continue, assuming subject and/or parent/guardian and Investigator consent, until disease progression or unacceptable toxicity. Long-term safety and overall survival will be collected for subjects who continue.

    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 Decision2016-04-26
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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