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    Summary
    EudraCT Number:2017-001215-37
    Sponsor's Protocol Code Number:RG_16_211
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-10-10
    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 number2017-001215-37
    A.3Full title of the trial
    A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome
    Międzynarodowe, randomizowane badanie kliniczne, typu pivotal, prowadzone metodą podwójnie ślepej próby, oceniające skuteczność i bezpieczeństwo walproinianu sodu u dzieci i dorosłych z zespołem Wolframa.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Trial of a Treatment to Slow Disease Progression Compared to Usual Standard of Care in Children and Adults with Wolfram
    Badanie kliniczne terapii spowalniającej postęp choroby w porównaniu do standardowego leczenia u dzieci i dorosłych z zespołem Wolframa
    A.3.2Name or abbreviated title of the trial where available
    Treat Wolfram
    Treat Wolfram
    A.4.1Sponsor's protocol code numberRG_16_211
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN10176118
    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 SponsorUniversity of Birmingham
    B.1.3.4CountryUnited Kingdom
    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 Council
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedical Univeristy of Lodz
    B.5.2Functional name of contact pointDepartment of Paediatrics
    B.5.3 Address:
    B.5.3.1Street AddressSporna 36/50
    B.5.3.2Town/ cityLodz
    B.5.3.3Post code91-738
    B.5.3.4CountryPoland
    B.5.4Telephone number+4842617 77 91
    B.5.6E-mailwojciech.mlynarski@umed.lodz.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 Sodium valproate 200 mg gastro-resistant tablets
    D.2.1.1.2Name of the Marketing Authorisation holderWockhardt UK Ltd
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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.3.11 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 placeboTablet
    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
    Wolfram syndrome
    Zespół Wolframa
    E.1.1.1Medical condition in easily understood language
    Genetic form of diabetes, blindness and neurodegeneration
    Genetycznie uwarunkowana choroba neurodegeneracyjna, objawiająca się cukrzycą i ślepotą
    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
    To determine the effectiveness of Sodium Valproate treatment in delaying the loss of vision in Wolfram syndrome
    Określenie skuteczności leczenia walproinianem sodu w procesie hamowania utraty wzroku, jaki towarzyszy w przebiegu zespołu Wolframa
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of sodium valproate.
    To determine the efficacy of sodium valporoate with respect to clinical parameters other than visual acuity.
    To evaluate the effects of sodium valproate on functional activities of daily living and quality of life.
    Ocena bezpieczeńtwa i tolerancji leczenia walproinianem sodu.
    Ocena skuteczności walproinianu sodu w odniesieniu do parametrów klinicznych innych niż ostrość widzenia.
    Ocena wpływu walproinianu sodu na sprawnośc funkcjonalną i jakość życia codziennego.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be eligible for enrolment:
    1. The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
    a) Documented diabetes mellitus diagnosed under 16 completed years according to the World Health Organization (WHO) or American Diabetes Association (ADA) criteria and/or documented optic atrophy diagnosed under 16 completed years
    AND
    b) Documented functionally relevant mutations on one or both alleles of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
    2. The patient is aged 6 years or older and weighing at least 20 kg.
    3. The patient’s visual acuity assessed as either the right eye or left eye having a LogMAR score of
    1.6 or better on an ETDRS chart, with or without corrected vision.
    4. Written informed consent (and assent as required).
    5. Females of child bearing potential will only be included after a negative pregnancy test as per national Valproate pregnancy prevention programme or equivalent. If sexually active, they must agree to use a highly effective contraception measure and to pregnancy testing at each clinic follow up visit.
    6. Sexually active men with a female partner of childbearing potential must agree to the use of condoms and the use of a highly effective method of contraception by the female partner.
    7. Patient willing and able to meet all protocol defined visits for the duration of the Trial.
    Pacjenci muszą spełniać wszystkie poniższe kryteria, aby kwalifikować się do rejestracji:
    1. Pacjent/ka ma rozpoznany zespół Wolframa, określony przez:
    a) udokumentowaną cukrzycę zdiagnozowaną przed ukończonym 16 rokiem życia, zgodnie z kryteriami Światowej Organizacji Zdrowia (WHO) lub Amerykańskiego Towarzystwa Diabetologicznego (ADA) i/lub udokumentowaną atrofię nerwu wzrokowego, zdiagnozowaną przed ukończeniem 16 roku życia ORAZ
    b) udokumentowane istotne funkcjonalnie mutacje na jednym lub obu allelach genu WFS1 na podstawie zapisanych w historii choroby wyników badań (jeśli są dostępne) lub wyników z certyfikowanego laboratorium, zweryfikowanych podczas wizyty przesiewowej.
    2) Pacjent/ka jest w wieku 6 lat lub starszy/a i waży co najmniej 20 kg.
    3) Wynik testu ostrości wzroku LogMAR na wykresie ETDRS ≥ 1.6 z korekcją lub bez korekcji (lewe lub prawe oko).
    4) Pisemna świadoma zgoda (i zgoda, jeśli jest wymagana).
    5) Pacjentki w wieku rozrodczym zostaną włączone do badania wyłącznie po uzyskaniu ujemnego wyniku testu ciążowego zgodnie z krajowym lub równoważnym programem dedykowanym zapobieganiu ekspozycji na walproinian w okresie ciąży. Jeśli są aktywne seksualnie, muszą wyrazić zgodę na stosowanie wysoce skutecznych metod antykoncepcji oraz muszą wyrazić zgodę na wykonywanie testów ciążowych podczas każdej wizyty kontrolnej.
    6) Aktywni seksualnie mężczyźni z partnerką w wieku rozrodczym muszą wyrazić zgodę na stosowanie prezerwatyw i wysoce skutecznych metod antykoncepcji przez partnerkę.
    7) Pacjent/ka chętny/a i zdolny/a do realizacji wszystkich wizyt zdefiniowanych protokołem na czas trwania badania.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria are not eligible for this Trial:
    1. The patient has clinically significant non-Wolfram related Central Nervous System (CNS) involvement, which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
    2. The patient has a diagnosis of a mitochondrial myopathy
    3. The patient has active liver disease, has a personal or family history of liver dysfunction related to known genetic disorders, or patient has porphyria.
    4. The patient has received treatment with any investigational drug within the 30 days prior to study entry.
    5. The patient is currently taking sodium valproate; or has a known hypersensitivity to sodium valproate or its excipients.
    6. Any other acute or chronic medical, psychiatric, social situation or laboratory result that, based on investigator's judgment, would jeopardize patient safety during trial participation, cause inability to comply with the protocol, or affect the study outcome.
    7. The patient is currently breastfeeding.
    8. The patient has known urea cycle disorders.
    9. The patient has one of he following disorders: Lactose intorelance, the Lapp lactase deficiency, or glucose-galactose malabsorption.
    Pacjenci, którzy spełniają którekolwiek z poniższych kryteriów, nie kwalifikują się do włączenia do badania:
    1. Pacjent/ka cierpi na klinicznie istotną, współwystepujacą chorobę układu nerwowego nie związaną z zespołem Wolframa, która może mieć wpływ na realizację i interpretację wyników poszczególnych badań określonych w protokole.
    2. Pacjent/ka ma rozpoznaną miopatię mitochondrialną.
    3. Pacjent/ka ma rozpoznaną czynną chorobę wątroby, u pacjenta/ki lub w wywiadzie rodzinnym stwierdzono występowanie dysfunkcji wątroby, lub ma rozpoznaną porfirię.
    4. Pacjent/ka przyjmował/a dowolny badany lek w ciągu 30 dni przed rozpoczęciem badania.
    5. Pacjent/ka aktualnie przyjmuje walproinian sodu lub ma stwierdzoną nadwrażliwość na walproinian sodu lub substancje pomocnicze.
    6. Każde inne ostre lub przewlekłe zdarzenie medyczne zaobserwowane u pacjenta/ki, stan psychiczny pacjenta/ki, sytuacja społeczna, w której znajduje się pacjent/ka lub wyniki jego badań laboratoryjnych, które na podstawie oceny badacza, zagrażałyby jego bezpieczeństwu podczas udziału w badaniu oraz powodowałyby niezdolność do przestrzegania protokołu lub mogłyby wpłynąć na wynik badania.
    7. Pacjentka w trakcie karmienia piersią.
    8. Pacjent/ka ma rozpoznane zaburzenia cyklu mocznikowego.
    9. Pacjent/ka ma rozpoznane jedno z następujących zaburzeń: nietolerancję laktozy, niedobór laktazy typu Lappa lub zespół złego wchłaniania glukozy-galaktozy.
    E.5 End points
    E.5.1Primary end point(s)
    Rate of change in visual acuity (using EDTRS chart)
    Wskaźnik zmiany ostrości wzroku (wykres EDTRS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Planned interim analysis after 24 months of intervention and placebo.

    Planned final analysis within 6 months of the final assessment of the final patient.

    Visual acuity will be assessed at screening, and then at day 0 (baseline), day 180, day 360, day 540, day 720, day 900 and day 1080.
    Planowana tymczasowa analiza po 24 miesiącach stosowanej interwencji i placebo.

    Planowana końcowa analiza w ciągu 6 miesięcy od ostatecznej wizyty ostatniego pacjenta.

    Ostrość wzroku będzie oceniana podczas badań przesiewowych, a następnie podczas wizyt w dniach 0, 180, 360, 540, 720, 900 i 1080.
    E.5.2Secondary end point(s)
    Safety of sodium valproate administration.
    Bezpieczeństwo podawania walproinianu sodu.
    E.5.2.1Timepoint(s) of evaluation of this end point
    We will assess the safety of sodium valproate by comparing the proportion of patients who experience an adverse event of specific interest in the first 3 months of treatment.
    SAE's will be monitored and evaluated throughout the trial.
    Ocenimy bezpieczeństwo walproinianu sodu, porównując odsetek pacjentów, u których wystąpi niepożądane zdarzenie o szczególnym znaczeniu w pierwszych 3 miesiącach leczenia.
    SAE będą monitorowane i oceniane przez cały okres trwania badania.
    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 No
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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 Yes
    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 (last visit of the last subject)
    LVLS (ostatnia wizyta ostatniego pacjenta)
    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 months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    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: 4
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 70
    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)
    If the study findings are that the treatment was effective, then doctors will be able to prescribe the study medicine 'off licence'. This is because sodium valproate is already licensed for use in children and adults, but for epilepsy, not Wolfram syndrome.
    Jeśli wyniki badania wskażą, że leczenie było skuteczne, lekarze będą mogli przepisać lek badany "pozarejestracyjnie, off-label", ponieważ walproinian sodu jest już dopuszczony do stosowania u dzieci i dorosłych w przypadku padaczki, a nie w zespole Wolframa.
    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 Decision2020-02-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-10-08
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