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    Summary
    EudraCT Number:2021-005339-22
    Sponsor's Protocol Code Number:REGBONE
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-12-03
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2021-005339-22
    A.3Full title of the trial
    To evaluate the efficacy and safety of regorafenib in patients with refractory primary bone tumors.
    Ocena skuteczności i bezpieczeństwa zastosowania regorafenibu u pacjentów z opornymi na leczenie pierwotnymi nowotworami kości.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To evaluate the efficacy and safety of regorafenib in patients with refractory primary bone tumors.
    Ocena skuteczności i bezpieczeństwa zastosowania regorafenibu u pacjentów z opornymi na leczenie pierwotnymi nowotworami kości.
    A.3.2Name or abbreviated title of the trial where available
    REGBONE
    A.4.1Sponsor's protocol code numberREGBONE
    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 SponsorInstytut Matki i Dziecka
    B.1.3.4CountryPoland
    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 supportMedical Research Agency
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstytut Matki i Dziecka
    B.5.2Functional name of contact pointSponsor Study Manager
    B.5.3 Address:
    B.5.3.1Street AddressMarcina Kasprzaka 17a
    B.5.3.2Town/ cityWarszawa
    B.5.3.3Post code01-211
    B.5.3.4CountryPoland
    B.5.4Telephone number00482232 77 205
    B.5.5Fax number004822632 98 51
    B.5.6E-mailkatarzyna.maleszewska@imid.med.pl
    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 Stivarga 40 mg
    D.2.1.1.2Name of the Marketing Authorisation holderBayer AG
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form 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 INNRegorafenib
    D.3.9.1CAS number 755037-03-7
    D.3.9.4EV Substance CodeSUB73090
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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
    Refractory primary bone tumors
    E.1.1.1Medical condition in easily understood language
    Refractory primary bone tumors
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10015562
    E.1.2Term Ewing's sarcoma metastatic
    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 10015564
    E.1.2Term Ewing's sarcoma recurrent
    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 PT
    E.1.2Classification code 10031294
    E.1.2Term Osteosarcoma metastatic
    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 PT
    E.1.2Classification code 10031296
    E.1.2Term Osteosarcoma recurrent
    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
    To evaluate the efficacy and safety of regorafenib in patients with refractory primary bone tumors
    E.2.2Secondary objectives of the trial
    1. Determination of the optimal dosing regimen of the test substance in patients from 2 years to 21 years through monitored pharmacokinetic parameters and pharmacodynamic effects.
    2. To determine the safety of regorafenib treatment in patients with primary bone tumors refractory to conventional therapy, aged 2 to 21 years.
    3. Determination of pharmacokinetic parameters Cmaxs, Cmins, Css, time to steady-state concentration.
    4. Evaluation of clinical response to regorafenib treatment in terms of pharmacokinetics / serum drug concentration.
    5. Assessment of adverse effects of regorafenib in terms of pharmacokinetics / serum drug concentration.
    6. Assessment of the molecular profile in patients with primary bone tumors refractory to conventional therapy, aged 2 to 21 years.
    7. Assessment of the molecular profile as a prognostic factor in comparison with other recognized factors.
    8. Derivation of an immortalized cell line.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age> 2 years and ≤ 21 years at the time of inclusion for the study.
    2. Ewing's sarcoma or osteosarcoma confirmed by histopathological examination and the tests performed so far.
    3. Treatment failure identified no earlier than 30 days prior to study treatment initiation (at least one subsection must be met for the patient to meet this criterion):
    a. progression on treatment of I or another line or
    b. relapse.
    4. Giving written, informed consent to participate in the study prior to the commencement of the procedures included in the study protocol, including treatment with regorafenib in accordance with the current legal regulations.
    5. Life expectancy of at least 12 weeks from signing the informed consent.
    6. Consent to use effective contraception throughout the period of regorafenib treatment and at least 2 years after its discontinuation in patients in puberty.
    E.4Principal exclusion criteria
    1. Failure to meet any of the inclusion criteria.
    2. Prior treatment with regorafenib.
    3. Pregnancy and breastfeeding.
    4. Known hypersensitivity to the drug or any of its ingredients.
    5. Taking medications that cannot be used while under regorafenib treatment.
    6. Persistent toxicity related to previous therapy, preventing drug incorporation.
    7. Diagnosis of other neoplastic disease prior to inclusion in the study.
    8. Patients with uncontrolled hypertension.
    9. Patients with diseases related to the coagulation disorders.
    10. Patients with heart defects and / or cardiac arrhythmias requiring permanent treatment with antiarrhythmic drugs.
    11. Other acute or chronic medical conditions, behaviors, or abnormal laboratory values ​​that may increase the risk of participating in this clinical trial or taking the study medication, or may affect the interpretation of the study results, or, in the investigator's opinion, may cause that the patient should not be enrolled in the study.
    E.5 End points
    E.5.1Primary end point(s)
    • EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm.
    • Determination of the dose of the test substance in patients between 2 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved.
    • To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance.
    • Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study. Interim analyzes were scheduled at least every 12 months from the opening of the study.
    E.5.2Secondary end point(s)
    • PFS (Progression-Free Survival) - progression-free survival - will be measured from randomization to finding disease progression in imaging studies.
    • OS (Overall Survival) - will be measured from randomization to death due to neoplastic disease.
    • ORR (Overall Response Rate) - Percentage of patients who achieved a protocol-defined response to treatment.
    • Time to reach the target serum concentration of the test substance.
    • Maximum serum concentration at steady state Cmaxs.
    • Steady-state trough serum concentration Cminss.
    • Casual steady-state serum concentration Css.
    • Exposure to Ctau.
    • Time to steady-state concentration of the test substance in the serum.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study. Interim analyzes were scheduled at least every 12 months from the opening of the study.
    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 No
    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) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned 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 concerned2
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days17
    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: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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
    underage patients
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    After the end of the examination, the patient remains under standard medical care at the Children's and Adolescent Colony and Surgery Clinic until the age of 25. After the age of 25, the patient is transferred to an adult care facility.
    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 Stowarzyszenie Pomocy Chorym na Mięsaki i Czerniaki Sarcoma
    G.4.3.4Network Country Poland
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-04-11
    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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