Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42869   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2016-000227-71
    Sponsor's Protocol Code Number:ITCC-059
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-01-03
    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 - PEI
    A.2EudraCT number2016-000227-71
    A.3Full title of the trial
    A phase I/II study of Inotuzumab Ozogamicin as a single agent and in combination with chemotherapy for pediatric CD22-positive relapsed/refractory Acute Lymphoblastic Leukemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The safety and efficacy of the medicine Inotuzumab Ozogamicin in children with relapsed/refractory acute lymphatic leukemia (ALL)
    A.3.2Name or abbreviated title of the trial where available
    ITCC-059
    A.4.1Sponsor's protocol code numberITCC-059
    A.5.4Other Identifiers
    Name:NTRNumber:NTR5736
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/304/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorErasmus Medical Center
    B.1.3.4CountryNetherlands
    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 supportPfizer
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPrincess Maxima Center
    B.5.2Functional name of contact pointTrial and datacenter
    B.5.3 Address:
    B.5.3.1Street AddressHeidelberglaan 25
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584 CS
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31650006679
    B.5.5Fax number+31(0)889729009
    B.5.6E-mailtrialmanagement@prinsesmaximacentrum.nl
    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 BESPONSA
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    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/3/13/1127
    D.3 Description of the IMP
    D.3.1Product nameInotuzumab Ozogamicin
    D.3.2Product code PF-05208773
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNINOTUZUMAB OZOGAMICIN
    D.3.9.1CAS number 635715-01-4
    D.3.9.4EV Substance CodeSUB33081
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.25
    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) 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
    pediatric CD22-positive relapsed/refractory Acute Lymphoblastic Leukemia
    E.1.1.1Medical condition in easily understood language
    Pediatric relapsed Acute Lymphoblastic Leukemia
    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 21.0
    E.1.2Level LLT
    E.1.2Classification code 10063625
    E.1.2Term Acute lymphoblastic leukemia recurrent
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary objective Stratum 1A:
    To establish the maximum tolerated dose of single agent InO when administered in children with CD22-positive relapsed/refractory BCP-ALL.

    Primary objective Phase 2 Cohort:
    To establish the activity (ORR) of single agent InO when administered in children with CD22-positive relapsed/refractory BCP-ALL.

    Primary objective Stratum 1B (and 1B-ASP): determine the RP2D of InO in children with CD22-positive relapsed/refractory BCP-ALL in combination with a modified UKALL-R3 based re-induction regimen.

    Primary objective Stratum 2: To explore the safety and tolerability of InO as a single agent in children with relapsed/refractory other CD22 positive B-cell malignancies.

    Primary objective Stratum 3: establish the preliminary efficacy (ORR)
    with respect to ORR of single agent InO in children with CD22-positive Very High Risk 1st relapse BCP-ALl (excluding patients transplanted in 1st CR).
    E.2.2Secondary objectives of the trial
    All strata
    •(hematological) response rate
    •MRD levels, % of patients with complete MRD (not Stratum 2)
    •durability of response / long-term FUP

    Str1A&Ph2:
    •safety & tolerability of InO
    •relation of CD22 receptor density, WBC-Count, CD22 saturation
    kinetics, cytogenetics, in-vitro calicheamicin resistance to response
    •persistence of B-Cell aplasia, hypogammaglobulinemia
    •nr of patients developing ADAs
    •serum PK parameters

    Str1B and Stratum 1B-ASP:
    •safety & tolerability of InO in combination with backbone regimen
    •relationship of CD22 expr to response
    •nr of patients developing CD22-neg relapse
    •serum PK parameters

    Str2:
    •persistence of B-Cell aplasia, hypogammaglobulinemia
    •nr of patients developing ADA
    •serum PK parameters

    Str3:
    •safety & tolerability of InO
    •relation of CD22 receptor density, WBC-Count, cytogenetics, in-vitro
    calicheamicin resistance to response.
    •persistence of B-Cell aplasia, MRD negativity
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age for all strata (Str1A, ph2, Str1B, Str2 and Str3): Patients must be ≥
    1 and < 18 years of age
    Patients with Down syndrome are excluded in Stratum 1A and 1B/1BASP
    but not in the phase 2 cohort and Stratum 3
    Additional criteria for Stratum 1A and 1B:
    •First 3 patients on dose level 1 must be 6-18 yrs.
    •Then at least 2 additional patients must be enrolled from age 1-6 yrs at
    the same dose level.
    •After this: subsequent dose levels may enroll patients aged 1-18 yrs.
    •In case 2 younger patients are not yet recruited, patients aged 6-18yrs
    may continue to be enrolled at dose level 1 until a maximum of 6
    patients are enrolled.
    Stratum 1A, phase 2 and stratum 1B/1B-ASP: Diagnosis
    Patients must have either 1st relapsed BCP-ALL after allo-HSCT or 2nd or
    greater relapsed or refractory BCP-ALL, or refractory disease (after at
    least 2 prior regimens):
    •M2 or M3 marrow status (≥ 5% blasts by morphology)
    •CD22 surface antigen positive (either BM or PB)
    •Stratum 1 only: The first 6 patients must have M3 marrow status (≥
    25% blasts by morphology).
    Stratum 2: Diagnosis
    Patients must have 2nd or greater relapsed or refractory CD22-positive
    B-cell malignancy including but not limited to diffuse large B-cell
    lymphoma (DLBCL), primary mediastinal large B-cell lymphoma
    (PMBCL), Burkitt lymphoma, Burkitt leukemia or B-cell precursor
    lymphoblastic lymphoma:
    •Histologic verification of disease at original diagnosis or subsequent
    relapse
    •Evaluable or measurable disease (by radiographic criteria or BM
    disease present)
    • CD22 surface antigen positive (either biopsy material, BM or PB)

    Str3: diagnosis:
    • 1st BM or combined relapse of CD22+ VHR BCP-ALL defined as:
    - any relapse <18 months from initial diagnosis and/or
    - cytogenetic-high risk characteristics: KTM2A/AF4, E2A/TCF3-PBX1
    t(1;19) or E2A/TCF3-HLF t(17;19), hypodiploidy (less than 40
    chromosomes), TP53 mutation and/or deletion
    • excluding patients transplanted in 1st CR.
    • M2 or M3 marrow status (≥ 5% blasts by morphology)
    • CD22 surface antigen positive (in either the BM or PB)
    • Evidence of prior fusion gene abnormalities is acceptable
    • cytogenetic-high risk characteristics determined by chromosome
    banding analysis (CBA), FISH, PCR and/or Next Generation Sequencing
    All strata:
    Performance Level and Life Expectancy:
    •Karnofsky > 60% for patients > 16 years of age and Lansky > 60% for
    patients ≤ 16 years of age.
    •life expectancy of at least 6 weeks.

    Prior Therapy:
    Patients must have fully recovered from the acute toxic effects of all
    prior chemotherapy, immunotherapy, or radiotherapy defined as
    resolution of all such non-hematologic toxicities to ≤ Grade 2 per the
    CTCAE 4.03.
    •Chemotherapy: At least 7 days wash-out; except for hydroxyurea, 6-mp
    and steroids (wash-out 48 hrs) and intrathecal therapy (no wash-out).
    Patients who relapse while receiving maintenance chemotherapy will not
    be required to have a waiting period.
    •Radiotherapy: At least 28 days must have elapsed since any prior
    radiation therapy.
    •Hematopoietic Stem Cell Transplant: At least 90 days must have
    elapsed since previous allo-HSCT. No evidence of active GVHD; not
    receiving GVHD prophylaxis or treatment.
    •Hematopoietic growth factors: At least 7 days wash-out of therapy with
    GCSF or other growth factors. At least 14 days wash-out of pegfilgrastim
    (Neulasta®).
    •Immunotherapy: At least 42 days wash-out of any type of
    immunotherapy, e.g. CART therapy. No prior CD22-targeted therapy or
    tumor vaccines permitted.
    •Monoclonal antibodies: wash-out of at least 3 half-lives of the antibody
    (ie: Rituximab = 66 days, Epratuzumab = 69 days), with the exclusion of
    blinatumomab. Patients must have been off blinatumomab infusion for at
    least 14 days and all drug-related toxicity must have resolved to grade 2
    or lower.
    •Investigational drugs: At least 7 days or 5 drug half-lives (whichever is
    longer) must have elapsed since prior treatment with any experimental
    drug (with the exception of monoclonal antibodies).
    •no prior treatment with a calicheamicin-conjugated antibody (e.g.
    gemtuzumab ozogamicin).
    Renal and Hepatic Function:
    •serum creatinine ≤ 1.5 x ULN according to age. If the serum creatinine
    is > than 1.5 xULN, the patient must have a GFR ≥ 70mL/min/1.73m2.
    •AST and ALT ≤ 2.5 x ULN.
    •total bilirubin ≤ 1.5 x ULN (unless patient has documented Gilbert
    syndrome &AST and ALT are <=2.5 x ULN).
    Cardiac Function:
    •shortening fraction ≥ 30% by ECG or an ejection fraction > 50% by
    MUGA.
    Reproductive Function:
    •If applicable, negative urine or serum pregnancy test confirmed prior to
    enrollment.
    •If applicable, agree not to breastfeed while on this study.
    •If applicable, agree using effective method of contraception during the
    study for 5 months (for male patients) or 8 months (for female
    patients) after the last dose of InO.
    E.4Principal exclusion criteria
    Isolated extramedullary relapse:
    •Patients with isolated extramedullary disease are excluded (not applicable to lymphoma patients except for isolated CNS-relapse)

    VOD/SOS:
    •Patients with any history of prior or ongoing VOD/SOS per the modified Seattle criteria are excluded, as specified in appendix 3, or prior liver-failure [defined as severe acute liver injury with encephalopathy and impaired synthetic function (INR of ≥1.5)].

    Infection:
    Patients will be excluded if they have a systemic fungal, bacterial, viral or other infection that is exhibiting ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment. The patient may not have:
    •A requirement for vasopressors;
    •Positive blood culture within 48 hours of study enrollment;
    •Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability.
    •A positive fungal culture within 30 days of study enrollment.
    •Active fungal, viral, bacterial, or protozoal infection requiring IV or oral treatment. Chronic prophylaxis therapy to prevent infections is allowed.

    Other anti-cancer therapy:
    •Patients will be excluded if there is a plan to administer non-protocol anti-cancer therapy including but not limited to chemotherapy, radiation therapy, or immunotherapy during the study period.

    Allergic reaction:
    •Patients with prior Grade 3/4 allergic reaction to a monoclonal antibody are excluded.

    Concurrent disease:
    •Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with protocol therapy, interfere with consent, study participation, follow up, or interpretation of study results.
    •Patients with Down syndrome are excluded in the dose finding
    parts (stratum 1A and 1B), but not in the phase 2 cohort or VHR cohort.


    Additional exclusion criteria for Stratum 1B
    • Patients with grade 3-4 peripheral neuropathy (as defined in the Delphi consensus of acute toxic effects for childhood ALL by Schmiegelow et al.1 ). Patients with prior history of thrombosis during steroid and/or asparaginase are eligible provided they use adequate anti-coagulant prophylaxis, according to institutional guidelines.
    • Patients in whom prior experience suggests that a timely delivery of therapy is unlikely or associated with an undue risk because of intolerance. 

    Additional exclusion criteria for Stratum 1B-ASP cohort only
    • Patients with any history of PEG-asparaginase intolerance due to allergic reactions or silent inactivation during prior treatment.
    • Patients with any history of prior asparaginase-associated acute pancreatitis (any grade as defined in the Delphi consensus.1).

    Patients who are excluded from Stratum 1B-ASP may potentially be enrolled in Stratum 1B expansion cohort.

    Additional exclusion criteria for Stratum 3 (VHR cohort) only:
    • Patients who are transplanted in CR1 (such patients are eligible for
    the phase 1B cohort).
    E.5 End points
    E.5.1Primary end point(s)
    Stratum 1A:
    Dose-limiting toxicities (DLTs) during the first cycle of therapy.

    Phase 2 cohort:
    Overall Response Rate (ORR), defined as CR, CRi, CRp, measured as best response during InO treatment.

    Stratum 1B and 1B-ASP:
    Dose-limiting toxicities (DLTs) during the first cycle of InO when added to a modified UKALL-R3 re-induction chemotherapy regimen without or with ASP.

    Stratum 2: Safety and tolerability:
    • AEs, as characterized by type, frequency, severity (as graded using CTCAE version, v4.03), timing, seriousness, and relation to study therapy, during the first and subsequent cycles of therapy.
    • Occurrence of toxic death; i.e., death attributable to InO therapy.
    • Occurrence of hepatic VOD/SOS during or after therapy with InO.
    • Laboratory abnormalities as characterized by type, frequency, severity and timing.
    • Cumulative incidence of non-relapse mortality

    Str3 cohort:
    • ORR: % of pats with CR, CRi, CRp, measured as best response to InO
    Treatment as a single agent in CD22+ VHR 1st relapse BCP ALL patients.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After cycle 1
    E.5.2Secondary end point(s)
    Stratum 1A:
    1. Safety and tolerability:
    • AEs
    • Occurrence of toxic death
    • Occurrence of hepatic VOD/ SOS during or after therapy with InO
    • Laboratory abnormalities
    • Cumulative incidence of non-relapse mortality
    2. Measures of anti-leukemic activity:
    • ORR defined as CR, CRi, CRp , best response after C1 and over multiple cycles
    • MRD levels, incl. % of pats who become MRD- (defined as an MRD-level < 1x10-4), after C1, as well as the best response (MRDnegativity) over multiple cycles.
    • Duration of response
    • Nr and % of pats being transplanted or having received CAR-T
    • Event-free survival (EFS)
    • Overall survival
    • Cumulative incidence of non-response or relapse
    3. Serum PK parameters of InO and unconjugated calicheamicin.
    4. Relationship between response (ORR) and CD22 expression levels, WBC, CD22 saturation kinetic, calicheamicin sensitivity
    • Clonal evolution (CD22-negativity) and relation to loss of response
    5. Other
    • % of pats responding to InO (ORR) without adequate recovery of CD19-positive B-cells or immunoglobulins following 4 wks, 10 wks, 3, 6 and 12 mnths after treatment (excl. pats having received HSCT or CAR-T).
    • % of pats with anti-drug antibodies (ADA).

    Phase 2 cohort:
    1. Safety:
    • AEs
    • Occurrence of toxic death
    • Occurrence of VOD/SOS during or after therapy with InO.
    • Laboratory abnormalities
    • Cumulative incidence of non-relapse mortality
    2. Other measures of anti-leukemic activity:
    • ORR after C1
    • MRD levels, including % of pats who become MRD- , after C1, as well as the best response (MRD-negativity) over multiple cycles.
    • Duration of response
    • Nr and % of pats being transplanted or having received CAR-T cells after treatment with Ino.
    • EFS
    • Survival
    • Cumulative incidence of non-response or relapse
    3. Serum PK parameters of InO and unconjugated calicheamicin
    4. PD parameters
    • Relationship between response (ORR) and CD22 expression levels, WBC, CD22 saturation kinetics and calicheamicin sensitivity.
    • Clonal evolution (CD22-negativity) and relation to loss of response.
    5. Other
    • The % of pats responding to InO (ORR) without adequate recovery of CD19-positive B-cells or immunoglobulins following 4 wks, 10 wks, 3, 6 and 12 months after treatment with InO, (excl. pats from the date of HSCT or CAR-T cell therapy)
    • % of pats who exhibit ADA

    Stratum 1B and 1B-ASP:
    1. Safety:
    • AEs
    • Occurrence of toxic death (death attributable to InO)
    • Occurrence of hepatic VOD/SOS during or after InO.
    • Laboratory abnormalities
    • Cumulative incidence of non-relapse mortality
    2. Other measures of anti-leukemic activity:
    • ORR
    • MRD levels
    • Duration of response
    • Nr and % of pats who undergo HSCT and those receiving CAR-T cell therapy
    • EFS
    • Overall survival
    • Cumulative incidence of non-response or relapse
    3. Serum PK parameters of InO and unconjugated calicheamicin during treatment combined with modified UKALL-R3 re-induction regimen both with and without pegylated asparaginase
    4. PD parameters
    • Relationship between response (ORR) and CD22 expression levels
    • Clonal evolution (CD22-negativity) and relation to loss of response

    Stratum 2:
    1. Measures of anti-tumor activity:
    • ORR (CR and PR) both after C1 as well as overall best response in patients receiving multiple cycles of InO therapy
    • Duration of response
    • Nr and % of pats being transplanted or having received CAR-T cell
    therapy after treatment with Ino.
    • EFS
    • Overall survival
    • Cumulative incidence of non-response or relapse.
    2. Serum pharmacokinetic parameters of InO and unconjugated calicheamicin.
    3. Other
    • The % of pats responding to InO (ORR) without adequate recovery of CD19-positive B-cells or immunoglobulins following 4 wks, 10 wks, 3, 6 and 12 mths after treatment, (excl pats from the date having received HSCT or CAR-T)
    • % of pats who exhibit ADA.


    Stratum3:
    1. Safety:
    • AEs
    • induction death and/or toxic death.
    • VOD/SOS during or after InO.
    • Laboratory abnormalities
    • The cumulative incidence of non-relapse mortality.
    2. Other measures of anti-leukemic activity:
    • ORR after cycle 1
    • MRD levels, incl % of pats who become MRD- after C1, best
    response (MRD-negativity) over multiple cycles.
    • Duration of response
    • Nr and % of pats being transplanted and/or receiving CAR T-cells.
    • EFS, Survival
    • cumulative incidence of non-response or relapse.
    • Interval between InO re-induction and CAR-T cells therapy based on
    MRD negativity and B cell aplasia after InO re-induction.
    3. PD parameters
    • Relationship between response (ORR) and CD22 expr, WBC, and
    calicheamicin sensitivity.
    • Clonal evolution (CD22-negativity) and relation to loss of response.
    4. Other
    • % of pats responding to InO without adequate recovery of CD19-
    positive B-cells and CD4+/CD8+ T-cells or immunoglobulins at 4,6,8 and
    10 weeks, 3, 6 and 12 months after treatment with InO, excl pats from
    the date of HSCT or CAR T-cell infusion.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Response: After cycle 1, after each subsequent cycle of therapy,
    Immunology: at 4wks, 10wks, 3months, 6 months and 9 months of FUP

    For str3 pats also at 6 and 8 wks of FUP.
    Survival/remission status: monthly in the 1st yr, once every 3 months in
    the 2nd and 3rd yr of FUP.
    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 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
    First administration in pediatric population
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 EEA28
    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
    Austria
    Belgium
    Czechia
    Denmark
    Finland
    France
    Germany
    Ireland
    Israel
    Italy
    Netherlands
    Norway
    Poland
    Portugal
    Spain
    Sweden
    Switzerland
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years10
    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 years11
    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: 156
    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: 8
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 90
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 58
    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 state29
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 142
    F.4.2.2In the whole clinical trial 156
    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
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation ITCC
    G.4.3.4Network Country Netherlands
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-07-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-11
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA