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    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   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).

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    Summary
    EudraCT Number:2021-002713-34
    Sponsor's Protocol Code Number:ITCC-098
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-03-14
    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 number2021-002713-34
    A.3Full title of the trial
    A Phase I/II study of Brigatinib in pediatric and young adult patients with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors or other solid tumors
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Scientific research on the safety and efficacy of the drug brigatinib in children and young adults with a tumour with an ALK positive deviation
    A.3.2Name or abbreviated title of the trial where available
    BrigaPED
    A.4.1Sponsor's protocol code numberITCC-098
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04925609
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/430/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPrincess Maxima Center for Pediatric Oncology in The Netherlands
    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 supportTakeda
    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 for Pediatric Oncology in The Netherlands
    B.5.2Functional name of contact pointMaaike Boonstra-Schelfhorst
    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+3165000 66 72
    B.5.6E-mailm.boonstra@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 ALUNBRIG
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma A/S
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameBrigatinib
    D.3.2Product code AP26113
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNBrigatinib
    D.3.9.1CAS number 1197953-54-0
    D.3.9.2Current sponsor codeAP26113
    D.3.9.3Other descriptive nameAlunbrig
    D.3.9.4EV Substance CodeSUB184911
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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 Yes
    D.2.1.1.1Trade name ALUNBRIG
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma A/S
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameBrigatinib
    D.3.2Product code AP26113
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNBrigatinib
    D.3.9.1CAS number 1197953-54-0
    D.3.9.2Current sponsor codeAP26113
    D.3.9.3Other descriptive nameAlunbrig
    D.3.9.4EV Substance CodeSUB184911
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number90
    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: 3
    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 ALUNBRIG
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma A/S
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameBrigatinib
    D.3.2Product code AP26113
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNBrigatinib
    D.3.9.1CAS number 1197953-54-0
    D.3.9.2Current sponsor codeAP26113
    D.3.9.3Other descriptive nameAlunbrig
    D.3.9.4EV Substance CodeSUB184911
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number180
    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
    relapsed/refractory ALK rearranged** or ALK mutated tumors, including relapsed/refractory ALK+ALCL and ALK+IMT.
    E.1.1.1Medical condition in easily understood language
    a tumour with an ALK positive aberration
    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
    Phase 1:
    • To determine the MTD/RP2D regimen of brigatinib monotherapy when administered in pediatric and AYA patients with ALK+ ALCL or ALK+ solid tumors, including ALK+ IMT.
    • To characterize the PK of brigatinib administered as monotherapy in pediatric and AYA patients with ALK+ ALCL or ALK+ solid tumors, including ALK+ IMT.
    Note that:
    o If the MTD is not reached at the highest proposed test dose, no further dose-escalation will be performed.
    o Pediatric PK data, compared to exposure in adults, will be taken into consideration to determine the RP2D.
    Phase 2:
    • Cohort B1, ALK+ IMT:
    To establish the anti-tumor activity of single agent brigatinib when administered to children with ALK+ IMT.

    • Cohort B2, ALK+ ALCL:
    To establish the efficacy of single agent brigatinib when administered to children with ALK+ ALCL for a duration of 2 years, without planned HSCT in consolidation.

    E.2.2Secondary objectives of the trial
    Phase 1: To assess:
    • safety and tolerability
    • acceptability and palatability of brigatinib.
    • To describe long term toxicity
    • To describe the anti-tumor activity and survival estimates
    • To assess the cumulative incidence of non-response or relapse
    • To describe the outcome for ALCL patients with and without SCT,
    • To describe anti-tumor activity for ALCL patients
    • To describe histological response in resected specimens from IMT patients undergoing surgery
    • To describe on treatment survival,
    Phase 2:
    Safety (in both cohorts)
    • To assess the safety and tolerability
    • To assess the cumulative toxicities
    • To describe long term toxicity
    • To assess the acceptability and palatability of brigatinib.
    • To collect plasma concentration-time data for brigatinib and construct a population PK model, and to relate exposure to safety parameters.
    • To describe the cumulative incidence of non-relapse mortality.

    Efficacy/activity:
    see protocol.......
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patients must be ≥1 and < 26 years of age at the time of enrollment, and able to swallow brigatinib tablets at the time of enrollment, with a minimum weight of 10 kg. Note 1: for phase 1 only patients < 18 years old will be eligible. A liquid formulation for children with a weight lower than 10 kg or for those that cannot swallow tablets is in development. Note 2: for the Netherlands only a minimum age of ≥ 5 years is required.
    2.Patients must have a histologically confirmed diagnosis of cancer at baseline. In patients where a repeat biopsy at relapse (or moment of refractory disease) is considered not feasible by the treating physician, archived material from diagnosis needs to be available for central review.
    3.Patients are required to provide prior results showing an activating ALK aberration in the tumor per local laboratory results, and material needs to be available for central laboratory confirmation of ALK status. For ALK+ ALCL, detection of ALK with immunohistochemistry (IHC) is sufficient for inclusion, all others require molecular evidence of a ALK fusion gene or mutation by FISH, PCR or NGS. ALK detection will be confirmed centrally with FISH.
    4.For Phase 1:
    •Patients with ALCL must be relapsed/refractory or intolerant to standard therapies. Refractory disease for ALCL is defined as:
    ono response to at least one course of ALCL99/other standard of care chemotherapy (SD or PD of measurable lesions), and/or
    oMRD-positivity by qualitative PCR for NPM-ALK after at least one course of ALCL99/other standard of care chemotherapy (before the second course of chemotherapy).
    •Patients with relapsed/refractory (R/R) IMT must not be suitable for curative surgical resection without causing mutilation. Newly diagnosed patients with locally advanced IMT, for whom surgery may not be feasible for close proximity to vital structures, without prior tumor-shrinkage, may also be included, as well as metastatic disease.
    •Patients with other solid tumors (excluding IMT) must have relapsed or refractory disease.
    5.For Phase 2, patients must have measurable and/or evaluable disease:
    •Patients with ALCL must be relapsed/refractory. Refractory disease for ALCL is defined as:
    ono response to at least one course of ALCL99/other standard of care chemotherapy (SD or PD of measurable lesions), and/or
    oMRD-positivity by qualitative PCR for NPM-ALK after at least one course of ALCL99/other standard of care chemotherapy (before the second course of chemotherapy).
    •Patients with R/R IMT Relapsed/refractory IMT, or newly diagnosed, including locally advanced and metastatic IMT which cannot be surgically resected without causing mutilation.
    6.Performance Status:
    •Karnofsky performance status ≥40% for patients >16 years of age or Lansky Play Scale ≥40% for patients ≤16 years of age for ALCL patients in phase 2.
    •Karnofsky performance status ≥50% for patients >16 years of age or Lansky Play Scale ≥50% for patients ≤16 years of age, for IMT and other solid tumors and for ALCL patients in phase 1.
    7.Patients must not be receiving other investigational medications (defined as medicinal products not yet approved for any indications, including alternative/herbal therapies) within 30 days of first dose of study drug or while on study.
    8.For patients receiving prior therapy:
    •Patients who already received previous treatment with ALK inhibitors except for brigatinib can be included in this study.
    •Patients who relapsed while receiving cytotoxic therapy: at least 14 days must have passed since the completion of the last dose of chemotherapy before the first dose of brigatinib can be given.
    •Patients who have experienced relapse after a prior HSCT are eligible, provided they have no evidence of acute or chronic graft-versus-host disease (GVHD), are not receiving GVHD prophylaxis or treatment, and are at least 45 days posttransplant at the time of enrollment.
    •Hematopoietic growth factors: before the first dose of brigatinib, at least 7 days must have passed since completion of therapy with granulocyte colony-stimulating factor or other growth factors, and at least 14 days must have passed since completion of therapy with pegfilgrastim.
    •Biologics and Targeted Therapies:
    oImmunotherapy: before the first dose of brigatinib, at least 30 days must have passed after the completion of any type of immunotherapy, (i.e. monoclonal antibodies [anti-PD1/PDL1], tumor vaccines, chimeric antigen receptor [CAR] T cells, etc.).
    oOther: before the first dose of brigatinib, at least 7 days must have passed since the last dose of a biologic agent, including any other ALK inhibitor. For agents that have known adverse events (AEs) occurring beyond 7 days after administration, this period must be extended beyond the time during which AEs are known to occur. The duration of this interval must be discussed with the sponsor’s medical monitor/designee.
    rest see protocol

    E.4Principal exclusion criteria
    Patients meeting any of the following exclusion criteria are not to be enrolled in the study:

    1. Patients receiving systemic treatment with strong or moderate CYP3A inhibitors or inducers within 14 days or five half-lives, whichever the less, prior to the first dose of study drug (refer to Section 5.2 for a list of example medications).
    2. Diagnosis of another concurrent primary malignancy.
    3. Clinically significant cardiovascular disease, including any of the following:
    • Myocardial infarction or unstable angina within 6 months of study entry.
    • History of or presence of heart block, and/or clinically significant ventricular or atrial arrhythmias.
    • Uncontrolled hypertension defined as persistent elevation of systolic and/or diastolic blood pressures to ≥95th percentile based on age, sex, and height percentiles despite appropriate antihypertensive management.
    4. Planned non-protocol chemotherapy, radiation therapy, another investigational agent, or immunotherapy while patient is on study treatment.
    5. Any illness that affects gastrointestinal absorption.
    6. Ongoing or active systemic infection, active seropositive HIV, or known active hepatitis B or C infection.
    7. Any pre-existing condition or illness that, in the opinion of the investigator or sponsor, would compromise patient safety or interfere with the evaluation of the safety or efficacy of brigatinib.
    8. Patients with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
    9. Patients with a history of cerebrovascular ischemia/hemorrhage with residual deficits are not eligible (patients with a history of cerebrovascular ischemia/hemorrhage remain eligible provided all neurologic deficits and causative have resolved).
    10. Uncontrolled seizure disorder (patients with seizure disorders that do not require antiepileptic drugs, or are well controlled with stable doses of antiepileptic drugs are eligible).
    11. Patients with electrolytes imbalances ≥ grade 2 NCI CTCAE v5.0 are not eligible (supplementation or medical intervention is allowed to correct electrolyte imbalance before inclusion).
    12. Patients with uncontrolled diabetes, i.e. patients with persistent
    hyperglycemia ≥ grade 2 NCI CTCAE v5.0 despite well conducted treatment with either oral anti glycemic agent and/or insulin are not eligible (patients with well controlled diabetes with either insulin or oral anti glycemic agents are eligible).
    E.5 End points
    E.5.1Primary end point(s)
    Phase 1:
    • Dose-limiting toxicities (DLTs) during the first course of therapy.
    • Brigatinib plasma PK parameters to be determined:
    o maximum observed concentration (Cmax),
    o time of first occurrence of maximum observed concentration (Tmax),
    o area under the concentration-time curve from time 0 to the time of the last quantifiable concentration (AUClast).
    • The RP2D will be selected by the DSMB and will be based on the dose that results in equivalent (approximately ±20% of the adult values) PK exposure to the adult comparator and with <2 out of 6 patients at this dose level present with a DLT and taking into account responses observed in phase 1.
    Phase 2:
    In Cohort B1, ALK+ IMT :
    • Overall response rate (ORR), defined as the percentage of patients with CR or PR according to RECIST 1.1 after 1 course and best ORR during brigatinib treatment.
    In Cohort B2, ALK+ ALCL:
    • EFS (using the IPNHL response criteria), defined as the time between start of study treatment and first event being progressive disease, relapse, death of any cause and second malignancies, whatever happens first. Patients consolidated with HSCT will be censored.
    E.5.1.1Timepoint(s) of evaluation of this end point
    see E.5.1.
    E.5.2Secondary end point(s)
    Phase 1:
    Safety
    • Adverse events (AEs), as characterized by type, frequency, severity (graded using CTCAE v5.0), including ocular, pulmonary, endocrine AEs, and height, weight or growth abnormalities, timing and relation to the study therapy, during the first and subsequent courses of therapy.
    •Occurrence of toxic death, i.e. death attributable to brigatinib therapy, as well as other causes of death.
    • Laboratory abnormalities as characterized by type, frequency, severity and timing.
    • The cumulative incidence of non-relapse mortality, with time calculated between start of study treatment and death.
    • Palatability questionnaire during two years of treatment (for frequency see SOE table).
    • Acceptability: diary reporting number of times a dose was not effectively administered.
    • Occurrence of any long-term toxicity during the off-therapy period up to 5 years after study inclusion with special attention to ocular, pulmonary, endocrine AEs, and height, weight or growth abnormalities .
    Activity/efficacy
    • ORR, defined as CR or PR, by RECIST 1.1 for solid tumors (other than neuroblastoma or brain tumors), by IPNHL (International Pediatric revised Response Criteria for Malignant Lymphoma) for ALCL, by NANT (New Approaches to Neuroblastoma Therapy) response criteria for neuroblastoma, by RANO (Responses Assessment in Neuro-Oncology) criteria for brain tumors, measured after 1 course and as best response during brigatinib treatment,
    • Time to best response, defined as the time between achieving the best response and the start of treatment with brigatinib For patients with ALCL; qualitative minimal residual disease (MRD) measured at multiple timepoints during treatment, including the percentage of patients who become MRD-negative, and time to MRD negativation.
    • Cumulative incidence of non-response or relapse and/or non-relapse mortality or patient withdrawal due to side effects in a competing risk model.
    • Duration of response (DOR), defined as the time between achieving response (CR or PR) after starting study treatment and documented disease progression, relapse or death.
    • EFS, defined as the time between start of study treatment and first event: relapse, progressive disease, death of any cause and second malignancies, whichever happens first.
    • OS, defined as time to death following start of study treatment.
    • Number of patients with IMT that undergo a complete (microscopic radical R0) resection after treatment with brigatinib.
    • Comparison of survival estimates for ALCL patients consolidated with SCT with patients that did not receive consolidation for SCT.
    • ORR, defined as CR or PR, (by IPNHL), measured after 1 course and as best response during brigatinib treatment (re-induction) in patients with ALCL that relapse after brigatinib discontinuation and that are subsequently re-challenged with brigatinib.
    • Number and percentage of IMT patients with completely necrotic tumors by pathology evaluation.
    • Duration of on treatment survival, defined as time from first treatment date to disease progression, death, or discontinuation of treatment for any reason (e.g., toxicity, patient/physician preference, or initiation of a new treatment without documented progression, using RECIST 1.1 or IPNHL response criteria).
    Phase 2 :
    Safety (in both cohorts)
    • Adverse events (AEs), as characterized by type, frequency, severity (graded using CTCAE v5.0), including pulmonary, ocular, endocrine AEs and height, weight or growth abnormalities, timing and relation to the study therapy, during brigatinib treatment.
    • Occurrence of toxic death, i.e. death attributable to brigatinib therapy as well as other causes of death.
    • Laboratory abnormalities as characterized by type, frequency, severity and timing.
    • The cumulative incidence of non-relapse mortality, defined as the cumulative probability of non-relapse mortality, with time calculated between start of study treatment and death.
    • Palatability questionnaire during two years of treatment (for frequency see SOE table).
    • Acceptability: diary reporting number of times a dose was not effectively administered.
    • Brigatinib plasma PK parameters
    o maximum observed concentration (Cmax),
    o time of first occurrence of maximum observed concentration (Tmax),
    o area under the concentration-time curve from time 0 to the time of the last quantifiable concentration (AUClast).
    • Occurrence of any long-term toxicity during the off-therapy period up to 5 years after study inclusion) with special attention pulmonary, ocular, endocrine AEs and height, weight or growth abnormalities.
    Activity/efficacy: see protocol
    E.5.2.1Timepoint(s) of evaluation of this end point
    See 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 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 Yes
    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
    therapeutic exploratory
    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.8.2.4Number of treatment arms in the trial2
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    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
    Switzerland
    Israel
    United Kingdom
    Austria
    Belgium
    Czechia
    Denmark
    Finland
    France
    Germany
    Ireland
    Italy
    Netherlands
    Norway
    Poland
    Spain
    Sweden
    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
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years9
    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: 50
    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: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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.3.3.6.1Details of subjects incapable of giving consent
    This study will be conducted in patients ≥1 year of age and < 26 years.
    For minors the informed consent of the parents or legal guardians will
    be mandatory.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 65
    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)
    The patient can continue to take medication after the study if parent, child, the treating doctor and the study team feel that the medication has a beneficial effect on your child's treatment. There are also possibilities to resume the medication if the disease returns in the short term if parent, child, the treating doctor and the study team feel that the medication has a beneficial effect on your child's treatment.
    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 European pediatric Soft Tissue Sarcoma Study Group (EpSSG)
    G.4.3.4Network Country Italy
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-08-12
    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
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