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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   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:2021-006702-78
    Sponsor's Protocol Code Number:BraimTOR-NEURO
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-04-19
    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 number2021-006702-78
    A.3Full title of the trial
    Open, randomized, IInd phase clinical study evaluating the safety and efficacy of rapamycin in the treatment of drug-resistant epilepsy in children with rare and ultra-rare diseases of the central nervous system associated with the activation of the mTOR pathway : BraimTOR-NEURO
    Otwarte randomizowane badanie kliniczne II fazy oceniające bezpieczeństwo i skuteczność rapamycyny w leczeniu padaczki lekoopornej u dzieci w ramach ustalenia postępowania w rzadkich i ultrarzadkich chorobach ośrodkowego układu nerwowego związanych aktywacją szlaku mTOR: BraimTOR – NEURO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open-label study to assess safety and efficacy of rapamycin in drug-resistant epilepsy associated with mTOR pathway pathologies
    Ocena bezpieczeństwa i skuteczności rapamycyny w leczeniu rzadkich padaczek wieku dziecięcego
    A.3.2Name or abbreviated title of the trial where available
    BraimTOR-NEURO
    BraimTOR-NEURO
    A.4.1Sponsor's protocol code numberBraimTOR-NEURO
    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 SponsorThe Children's Memorial Health Institute
    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 organisationThe Children's Memorial Health Institute
    B.5.2Functional name of contact pointKrzysztof Sadowski
    B.5.3 Address:
    B.5.3.1Street Address20 Dzieci Polskich Avenue
    B.5.3.2Town/ cityWarsaw
    B.5.3.3Post code04-730
    B.5.3.4CountryPoland
    B.5.4Telephone number48228157460
    B.5.6E-mailk.sadowski@ipczd.pl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    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 Rapamune 1 mg/ml oral solution
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited Ramsgate Road Sandwich Kent, CT13 9NJ Great Britain
    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.3.1Product nameRapamune 1mg/ml oral solution
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    epilepsy, mTORopathies, focal cortical dysplasia, LEATS
    padaczka, „mTORopatie”, ogniskowa dysplazja korowa, LEATS
    E.1.1.1Medical condition in easily understood language
    epilepsy, mTORopathies, focal cortical dysplasia, LEATS
    padaczka, „mTORopatie”, ogniskowa dysplazja korowa, LEATS
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to determine safety and tolerability of rapamycin in a drug resistant epilepsy associated with rare and ultra-rare diseases of the central nervous system associated with the activation of the mTOR pathway
    Podstawowym celem badania jest określenie bezpieczeństwa i tolerancji rapamycyny w padaczce lekoopornej związanej z rzadkimi i ultrarzadkimi chorobami ośrodkowego układu nerwowego związanymi z aktywacją szlaku mTOR
    E.2.2Secondary objectives of the trial
    The secondary aim of the study is to ascertain the antiseizure efficacy of rapamycin in a drug resistant epilepsy associated with rare and ultra-rare diseases of the central nervous system associated with the activation of the mTOR pathway
    Drugorzędowym celem badania jest ustalenie skuteczności przeciwnapadowej rapamycyny w leczeniu rzadkich
    i ultrarzadkich schorzeń związanych z patologią szlaku mTOR
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age at inclusin from 4 months to 18 years.
    2. Diagnosis of drug-resistant epilepsy as defined by ILAE (Kwan 2010), with focal onset (ICD-10: G40.2), with confirmed coexistence or suspicion of mTORopathy in MRI, including: FCD, hemimegalencephaly, LEATs; active epilepsy at study entry, at least 8 seizures in the prior 4 weeks prior to study entry
    3. Informed consent to the participation in the study by the patient's legal caregivers and the patient himself, if he is 13 years of age or older.
    4. Negative result of the pregnancy test performed within 2 weeks prior to the start of the study in patients of reproductive age.
    5. Written consent to the use of effective contraception in sexually active patients or an obligation to abstaining from sexual activity during and for 3 months after its treatment.
    1. Wiek pacjenta od ukończenia 4 miesiąca do 18 roku życia.
    2. Rozpoznanie padaczki lekoopornej zgodnie z definicją ILAE (Kwan 2010), o ogniskowym początku (ICD-10: G40.2), z potwierdzonym współistnieniem lub podejrzeniem mTORopatii w MRI, w tym: FCD, hemimegalencefalii, LEATs; w momencie włączenia do badania aktywna padaczka- co najmniej 8 napadów w okresie poprzedzających 4 tygodni przed włączeniem do badania
    3. Udzielenie świadomej zgody na udział w badaniu przez opiekunów prawnych pacjenta i samego pacjenta jeżeli ukończył 13 lat,
    4. Negatywny wynik testu ciążowego, wykonanego w ciągu 2 tygodni poprzedzających rozpoczęcie badania u pacjentek w wieku rozrodczym.
    5. Pisemna zgoda na stosowanie skutecznej antykoncepcji u pacjentów aktywnych seksualnie lub zobowiązanie zachowania wstrzemięźliwości seksualnej w trakcie leczenia i przez 3 miesiące po jego zakończeniu.
    E.4Principal exclusion criteria
    1. Severe coexisting diseases, e.g. renal failure, immunodeficiency,
    2. Clinically significant disease of the cardiovascular system, e.g. arrhythmia,
    3. Earlier diagnosis of neoplasm <5 years requiring antitumor treatment,
    4. Active infection requiring general treatment,
    5. Contraindications to use, including the inability to take or known hypersensitivity to the drug used in the study,
    6. Simultaneous administration of drugs that are strong inhibitors of CYP3A or PgP,
    7. Radiological features of active CNS bleeding except for asymptomatic resolving changes after biopsy or point bleeding in the tumor,
    8. Major surgery, open biopsy <28 days prior to treatment initiation,
    9. Minor surgical procedures <2 days before the start of treatment, e.g. implantation of a vascular port,
    10. Non-healing wound, unhealed fracture,
    11. Radiation therapy of the CNS <28 days,
    12. Pregnancy, breastfeeding,
    13. Active participation in another research program or completed <30 days,
    14. Systemic treatment with an mTOR inhibitor from other indications in the 3 months preceding study enrollment,
    15. Use of marijuana or its derivatives
    16. Live vaccine intake <6 weeks prior to study inclusion,
    17. Receipt of inactivated vaccine or mRNA vaccine <4 weeks prior to study inclusion.
    18. Tuberous sclerosis
    1. Ciężkie współistniejące choroby, np. niewydolność nerek, niedobory odporności,
    2. Klinicznie istotna choroba układu sercowo-naczyniowego, np. arytmia,
    3. Wcześniejsze <5 lat rozpoznanie nowotworu, wymagającego leczenia przeciwnowotworowego,
    4. Aktywna infekcja wymagająca leczenia ogólnego,
    5. Przeciwwskazania do stosowania, w tym niemożność przyjęcia lub znana nadwrażliwość na lek stosowany w badaniu,
    6. Równoczesne przyjmowanie leków będących silnymi inhibitorami CYP3A lub PgP,
    7. Radiologiczne cechy aktywnego krwawienia w OUN poza bezobjawowymi ustępującymi zmianami po biopsji lub punktowym krwawieniem w nowotworze,
    8. Poważny zabieg chirurgiczny, otwarta biopsja < 28 dni przed rozpoczęciem leczenia,
    9. Drobne zabiegi chirurgiczne < 2 dni przed rozpoczęciem leczenia np. implantacja portu naczyniowego,
    10. Niegojąca się rana, nie zagojone złamanie,
    11. Radioterapia OUN < 28 dni,
    12. Ciąża, karmienie piersią,
    13. Aktywny udział w innym programie badawczym lub zakończony < 30 dni,
    14. Leczenie systemowe inhibitorem mTOR z innych wskazań w okresie 3 miesięcy poprzedzających włączenie do badania,
    15. Stosowanie marihuany lub jej pochodnych
    16. Przyjęcie szczepionki żywej < 6 tygodni przed włączeniem do badania.
    17. Przyjęcie szczepionki inaktywowanej lub szczepionki mRNA <4 tygodni przed włączeniem do badania.
    18. Stwardnienie guzowate.
    E.5 End points
    E.5.1Primary end point(s)
    The primary safety endpoint of rapamycin will be the assessment of the incidence of adverse reactions (according to the CTCAE classification) during the treatment phase and follow-up of patients with drug-resistant epilepsy

    The primary efficacy endpoint will be the proportion of patients achieving a ≥50% reduction in seizures
    Pierwszorzędowym punktem końcowym dotyczącym bezpieczeństwa rapamycyny będzie ocena częstości występowania działań niepożądanych (zgodnie z klasyfikacją CTCAE) w czasie fazy leczenia i obserwacji pacjentów z padaczką lekooporną

    Pierwszorzędowym punktem końcowym dotyczącym skuteczności leczenia będzie odsetek chorych osiągających redukcja liczby napadów padaczkowych o ≥50%
    E.5.1.1Timepoint(s) of evaluation of this end point
    -interim analysis
    -final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
    -analiza okresowa
    -ostateczna analiza po formalnym zamknięciu bazy danych, planowana w ciągu miesiąca od ostatniej wizycie ostatniego pacjenta w badaniu
    E.5.2Secondary end point(s)
    A secondary safety endpoint will be the CTCAE severity of adverse reactions and the number of patients experiencing adverse reactions requiring exclusion from the study or premature termination of participation during the therapeutic dose period. The effect of treatment on the results of laboratory tests will also be analyzed.

    The secondary endpoint of the trial will be maintenance of short-term effects, improvement of quality of life in ≥50% of patients / families.
    Drugorzędowym punktem końcowym dotyczącym bezpieczeństwa będzie ciężkość działań niepożądanych wg klasyfikacji CTCAE oraz liczba pacjentów doświadczających działań niepożądanych wymagających wykluczenia z badania lub przedwczesnego zakończenia uczestnictwa w okresie podawania terapeutycznej dawki leku. Analizie poddany zostanie także wpływ leczenia na wyniki badań laboratoryjnych.

    Drugorzędowym punktem końcowym badania będzie utrzymanie efektów krótkoterminowych, poprawa jakości życia u ≥50% pacjentów /rodzin.
    E.5.2.1Timepoint(s) of evaluation of this end point
    -interim analysis
    -final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
    -analiza okresowa
    -ostateczne analizy po formalnym ostatecznym zamknięciu bazy danych, planowane w ciągu miesiąca od ostatniej wizyty pacjenta w badaniu
    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 Yes
    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 Yes
    E.8.2.3.1Comparator description
    leczenie standardowe (SOC)
    standard of care (SOC)
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned 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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    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) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    the study will include underage and incapacitated patients - their
    parents/caregivers will be asked for their consent
    do badania włączani będą pacjenci niepełnoletni i ubezwłasnowolnieni
    – o zgodę zostaną poproszeni opiekunowie/rodzice
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    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)
    Patients will be offered standard treatment of that condition
    Pacjentom zostanie zaproponowane standardowe leczenie w tym
    schorzeniu
    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 Decision2022-08-16
    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 StatusTrial now transitioned
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