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    Summary
    EudraCT Number:2021-004548-64
    Sponsor's Protocol Code Number:RaRETS
    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:2022-12-08
    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 number2021-004548-64
    A.3Full title of the trial
    Multicenter, randomized, double-blind, placebo controlled study to assess the efficacy and safety of Rapamycin in drug Resistant Epilepsy associated with TSC (RaRE-TS)
    Wieloośrodkowe, randomizowane, podwójnie zaślepione, kontrolowane placebo badanie oceniające skuteczność i bezpieczeństwo rapamycyny w lekoopornej padaczce związanej ze stwardnieniem guzowatym
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Placebo controlled study to assess the efficacy and safety of rapamycin in drug resistant epilepsy associated with tuberous sclerosis complex
    Kontrolowane placebo badanie oceniające skuteczność i bezpieczeństwo rapamycyny w lekoopornej padaczce związanej ze stwardnieniem guzowatym
    A.3.2Name or abbreviated title of the trial where available
    RaRE-TS
    RaRE-TS
    A.4.1Sponsor's protocol code numberRaRETS
    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 pointKatarzyna Kotulska-Jóźwiak
    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.kotulska@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.3.4Pharmaceutical 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral liquid
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosis
    stwardnienie guzowate, padaczka, guzy narządowe związane ze stwardnieniem guzowatym
    E.1.1.1Medical condition in easily understood language
    tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosis
    stwardnienie guzowate, padaczka, guzy narządowe związane ze stwardnieniem guzowatym
    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 RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).
    Podstawowym celem badania RaRE-TS jest określenie bezpieczeństwa, tolerancji i skuteczności rapamycyny w porównaniu z placebo w padaczce lekoopornej związanej ze stwardnieniem guzowatym.
    E.2.2Secondary objectives of the trial
    The secondary aim of the study is to ascertain the impact of rapamycin on the TSC- Associated Neuropsychiatric Disorders (TAND) and TSC-associated tumors (brain, kidney, retina and skin lesions) in comparison to placebo.
    Drugorzędowym celem badania jest ustalenie wpływu rapamycyny na związane z TSC zaburzenia neuropsychiatryczne (TAND) i guzy (zmiany w mózgu, nerkach, siatkówce i skórze) w porównaniu z placebo.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    fMRI substudy - imaging brain structures and its activities related to stimulus,
    holter EEG substudy - assessment of bioelectrical brain activities during 24h,
    pharmacokinetics profile (AUC) substudy - drug concentration in blood assessment in different timepoints after dosing
    podbadanie funkcjonalny rezonans magnetyczny - obrazowanie struktur mózgu oraz jego czynności w reakcji na różne bodźce
    podbadanie holter EEG - ocena czynności bioelektrycznej mózgu w ciągu całej doby
    podbadanie AUC – profil farmakokinetyczny - określenie zmian stężenia leku we krwi jakie zachodzą w różnych odstępach czasu po jego podaniu
    E.3Principal inclusion criteria
    -male or female aged from 3 months up to 50 years at the day of randomization
    -patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study
    -patients/parents/caregivers are willing to and able to comply with all study requirements
    -definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)
    -drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks
    - mężczyzna lub kobieta w wieku od 3 miesięcy do 50 lat w dniu randomizacji
    - pacjenci/rodzice/opiekunowie chcą i są w stanie wyrazić świadomą zgodę na udział w badaniu
    - pacjenci/rodzice/opiekunowie chcą i są w stanie spełnić wszystkie wymagania badania
    - jednoznaczne rozpoznanie TSC według kryteriów Consensus (Northrup, 2013)
    - padaczka lekooporna związana z TSC z co najmniej 8 napadami w ciągu 4 tygodni
    E.4Principal exclusion criteria
    - history of treatment with mTOR inhibitor in the three months prior to screening,
    - history of pseudo-epileptic seizures,
    - history of progressive CNS disease other than TSC
    - recent surgery within 2 weeks prior to the screening
    - severe infection within 2 weeks prior to the screening
    - use of the cannabis derivatives
    - contraindications for MRI or general anesthesia
    - occurrence of the serious comorbidities which, in the opinion of the investigator, may either put a patient at significant risk associated with the
    participation in the study or may influence the results of the study the investigator
    - pregnancy
    - historia leczenia inhibitorem mTOR w ciągu trzech miesięcy przed badaniem przesiewowym,
    - historia napadów rzekomopadaczkowych,
    - postępująca choroba ośrodkowego układu nerwowego (OUN) w wywiadzie inna niż TSC
    - niedawna operacja przebyta w ciągu 2 tygodni przed badaniem
    - ciężka infekcja przebyta w ciągu 2 tygodni przed badaniem
    - stosowanie pochodnych konopi
    - przeciwwskazania do wykonania rezonansu magnetycznego lub znieczulenia ogólnego
    - wystąpienie poważnych chorób współistniejących, które w ocenie badacza mogą narazić pacjenta na znaczne ryzyko związane z udziałem w badaniu
    lub mogą mieć wpływ na wyniki badania
    - ciąża
    E.5 End points
    E.5.1Primary end point(s)
    - comparison of the number of patients with at least 50% reduction of seizures per week in the last month of the core blinded phase in comparison to screening phase in the rapamycin vs placebo group
    - number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
    - porównanie liczby pacjentów z co najmniej 50% redukcją tygodniowych napadów w ostatnim miesiącu głównej fazy zaślepienia w porównaniu z fazą przesiewową w grupie rapamycyny w porównaniu do grupy placebo
    - liczba zdarzeń niepożądanych (wg klasyfikacji CTCAE) w grupie rapamycyny vs placebo podczas głównej, zaślepione fazy badania
    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)
    -comparison of the number of seizures per week and the number of days free of seizures in the rapamycin vs placebo group, during 12-week treatment in double-blind core phase
    - severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group
    -porównanie liczby napadów w tygodniu i liczby dni wolnych od napadów w grupie rapamycyny w porównaniu z placebo podczas 12-tygodniowego leczenia w głównej, zaślepione fazie badania
    - nasilenie zdarzeń niepożądanych (wg CTCAE) oraz liczba pacjentów wycofanych z badania z powodu zdarzeń niepożądanych w grupie rapamycyny w porównaniu do grupy placebo
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    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 concerned3
    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 years1
    E.8.9.1In the Member State concerned months0
    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: 160
    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: 50
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 70
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 40
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2022-12-08. Yes
    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 state200
    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 Decision2022-06-03
    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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