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    Summary
    EudraCT Number:2020-004723-17
    Sponsor's Protocol Code Number:UZBSK001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-18
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2020-004723-17
    A.3Full title of the trial
    Clinical usefulness and influence on oesophageal motility of pyridostigmine
    for dysphagia in systemic sclerosis.
    Klinische meerwaarde van pyridostigmine bij patiënten met
    systeemsclerose die dysfagie ervaren, met meting van de invloed op de
    slokdarm samentrekkingen.
    Valeur clinique de la pyridostigmine chez les patients atteints de
    sclérodermie systémique souffrant de troubles de la déglutition, avec
    mesure des contractions de l'oesophage.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to evaluate the improvement of swallowing problems
    experienced by patients suffering from systemic sclerosis by
    pyridostigmine.
    Deze klinische studie wordt uitgevoerd om het onderzochte geneesmiddel
    of "studiegeneesmiddel", pyridostigmine, te evalueren voor de behandeling
    van slikstoornissen bij patiënten die lijden aan systeemsclerose.
    Une étude clinique pour évaluer le médicament à l'étude, la pyridostigmine
    pour le traitement des troubles de la déglutition chez le patient atteint de
    la sclérodermie systémique.
    A.3.2Name or abbreviated title of the trial where available
    PySys
    A.4.1Sponsor's protocol code numberUZBSK001
    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 SponsorUZ Brussel
    B.1.3.4CountryBelgium
    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 supportUZ Brussel
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUZ Brussel
    B.5.2Functional name of contact pointProf. Dr. S. Kindt
    B.5.3 Address:
    B.5.3.1Street AddressLaarbeeklaan 101
    B.5.3.2Town/ cityJette
    B.5.3.3Post code1090
    B.5.3.4CountryBelgium
    B.5.4Telephone number003224776811
    B.5.5Fax number003224776810
    B.5.6E-mailsebastien.kindt@uzbrussel.be
    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 Mestinon (pyridostigmine bromide)
    D.2.1.1.2Name of the Marketing Authorisation holderMylan EPD bvba/sprl
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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 INNPYRIDOSTIGMINE BROMIDE
    D.3.9.1CAS number 101-26-8
    D.3.9.4EV Substance CodeSUB10167MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    Dysphagia by patients suffering from systemic sclerosis
    Dysfagie bij patienten met systeemsclerose
    Dysphagie chez les patients atteints de sclérodermie systémique
    E.1.1.1Medical condition in easily understood language
    Swallowing problems by patients suffering from systemic sclerosis
    Slikstoornissen bij patienten met systeemsclerose
    Troubles de déglutition chez les patients atteints de sclérodermie
    systémique
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    This study aims to evaluate the improvement of dysphagia experienced
    by patients suffering from systemic sclerosis by pyridostigmine.
    Deze studie heeft tot doel de verbetering van dysfagie te evalueren die
    wordt ervaren door patienten die lijden aan systemische sclerose door
    pyridostigmine.
    Cette étude vise à évaluer l'amélioration de la dysphagie subie par des
    patients souffrant de sclérose systémique par la pyridostigmine
    E.2.2Secondary objectives of the trial
    Secondarily, as esophageal dysmotility results in impaired clearance of
    refluxate, the influence of pyridostigmine on the severity of symp-toms
    of gastro-esophageal reflux disease will be assessed.
    In de tweede plaats, aangezien oesofageale dysmotiliteit resulteert in
    een verminderde klaring van refluxaat, zal de invloed van pyridostigmine
    op de ernst van symptomen van gastro-oesofageale refluxziekte worden
    beoordeeld.
    Deuxièmement, comme la dysmotilité oesophagienne entraîne une
    diminution de la clairance du reflux, l'influence de la pyridostigmine sur
    la sévérité des symptômes du reflux gastro-oesophagien sera évaluée.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients aged 18 years or older;
    - Known systemic sclerosis;
    - Dysphagia with Eckardt score 5 or more despite twice daily high dose
    PPI (omeprazole 40 mg b.i.d., esomeprazole 20 mg b.i.d., pantoprazole
    40 mg b.i.d., lansoprazole 30 mg b.i.d.).
    - Patiënten van 18 jaar of ouder;
    - Bekende systemische sclerose;
    - Dysfagie met Eckardt-score 5 of meer ondanks tweemaal daags hoge
    dosis PPI (omeprazol 40 mg tweemaal daags, esomeprazol 20 mg
    tweemaal daags, pantoprazol 40 mg tweemaal daags, lansoprazol 30 mg
    tweemaal daags).
    Patients âgés de 18 ans ou plus;
    - Sclérose systémique connue;
    - Dysphagie avec un score Eckardt de 5 ou plus malgré une dose élevée
    d'IPP deux fois par jour (oméprazole 40 mg b.i.d., esoméprazole 20 mg
    b.i.d., pantoprazole 40 mg b.i.d., lansoprazole 30 mg b.i.d.).
    E.4Principal exclusion criteria
    - Presence of reflux oesophagitis grade C or more, eosinophilic
    oesophagitis, organic stenosis (whether peptic or malignant);
    - Prior oesophageal surgery, endoscopic resections, Radiofre-quency
    Ablation for Barrett's mucosa or POEM;
    - Prior gastric surgery;
    - Prior diagnosis of major oesophageal motor disorder such as
    oesophageal spasm, achalasia, EGJ(esophagogastric junction) outflow
    obstruction
    Prevention of major side effects by exclusion of certain patient groups:
    - Diarrhoea (defined as Bristol Stool Scale > 5) for more than 2 days
    per week in the past 2 weeks;
    - Uncontrolled asthma, exacerbation of COPD in the last 4 weeks;
    - Known ischaemic heart condition or myocardial infarction in the last 4
    weeks;
    - Resting heart rate < 60 bpm;
    - AV-block 2 or 3 (except after implantation of a pacemaker), or
    bradycardia < 60 bpm;
    - Systolic blood pressure > 100 mmHg;
    - Impaired renal function with glomerular filtration rate < 30 ml/min;
    - Previous treatment with pyridostigmine in the past 4 weeks;
    - Treatment with anticholinergic agents in the past 4 weeks;
    - Pregnancy. Women of childbearing age will undergo a preg-nancy test
    before starting treatment and will be required to use at least 2 highly
    effective anticonception methods.
    - Aanwezigheid van reflux-oesofagitis graad C of hoger, eosinofiele
    oesofagitis, organische stenose (zowel peptisch als kwaadaardig);
    - Eerdere slokdarmoperaties, endoscopische resecties, radiofrequentieablatie
    voor Barrett's mucosa of POEM;
    - Voorafgaande maagoperatie;
    - Voorafgaande diagnose van een ernstige motorische aandoening van
    de slokdarm, zoals slokdarmkrampen, achalasie, obstructie van de EGJuitstroom
    Preventie van ernstige bijwerkingen door uitsluiting van bepaalde
    patiëntengroepen:
    - Diarree (gedefinieerd als Bristol Stool Scale> 5) gedurende meer dan 2
    XML File Identifier: EGrRyed7fGTihnrlFgEnneJa5Po=
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    dagen per week in de afgelopen 2 weken;
    - Ongecontroleerde astma, verergering van COPD in de afgelopen 4
    weken;
    - Bekende ischemische hartaandoening of myocardinfarct in de
    afgelopen 4 weken;
    - Hartslag in rust <60 slagen per minuut;
    - AV-blok 2 of 3 (behalve na implantatie van een pacemaker), of
    bradycardie <60 spm;
    - systolische bloeddruk> 100 mmHg;
    - verminderde nierfunctie met glomerulaire filtratiesnelheid <30 ml /
    min;
    - Eerdere behandeling met pyridostigmine in de afgelopen 4 weken;
    - Behandeling met anticholinergica in de afgelopen 4 weken;
    - Zwangerschap. Vrouwen in de vruchtbare leeftijd zullen een
    zwangerschapstest ondergaan voordat ze met de behandeling beginnen,
    en zullen minimaal 2 zeer effectieve anticonceptiemethoden moeten
    gebruiken.
    - Présence d'oesophagite par reflux de grade C ou plus, oesophagite à
    éosinophiles, sténose organique (qu'elle soit peptique ou maligne);
    - Chirurgie oesophagienne préalable, résections endoscopiques, Ablation
    par radiofréquence de la muqueuse de Barrett ou POEM;
    - Chirurgie gastrique antérieure;
    - Diagnostic préalable d'un trouble moteur oesophagien majeur tel que
    spasme oesophagien, achalasie, obstruction à l'écoulement de l'EGJ
    Prévention des effets secondaires majeurs par exclusion de certains
    groupes de patients:
    - Diarrhée (définie par le Bristol Stool Scale> 5) pendant plus de 2 jours
    par semaine au cours des 2 dernières semaines;
    - Asthme incontrôlé, exacerbation de la BPCO au cours des 4 dernières
    semaines;
    - Cardiopathie ischémique connue ou infarctus du myocarde au cours des
    4 dernières semaines;
    - Fréquence cardiaque au repos <60 bpm;
    - bloc AV 2 ou 3 (sauf après implantation d'un stimulateur cardiaque), ou
    bradycardie <60 bpm;
    - Tension artérielle systolique> 100 mmHg;
    - Insuffisance rénale avec débit de filtration glomérulaire <30 ml / min;
    - Traitement antérieur par pyridostigmine au cours des 4 dernières
    semaines;
    - Traitement avec des agents anticholinergiques au cours des 4 dernières
    semaines;
    - Grossesse. Les femmes en âge de procréer subiront un test de
    grossesse avant de commencer le traitement et devront utiliser au moins
    2 méthodes anticonceptionnelles hautement efficaces.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the number of patients with improvement of
    Eckardt score to 3 or less (which is widely accepted as absence of significant
    dysphagia).
    Het primaire eindpunt is het aantal patiënten met een verbetering van de
    Eckardt-score tot 3 of minder (wat algemeen wordt aanvaard als
    afwezigheid van significante dysfagie).
    Le critère d'évaluation principal est le nombre de patients avec une
    E.5.1.1Timepoint(s) of evaluation of this end point
    4 weeks
    4 weken
    4 semaines
    E.5.2Secondary end point(s)
    - Changes of Eckardt score from baseline will be compared be-tween
    both treatment arms;
    - Changes in HRM (high resolution manometry) parameters from
    baseline: Increase in Distal Contractility Integral, in-crease in
    Integrative Relaxatory Pressure 4s, increase in the percentage of
    successful bolus clearance, decrease in number of failed peristalsis,
    increase in the percentage of normal peri-stalsis;
    - Decrease in GERD-FSSG;
    - Improvement in the score of the SF-36 QOL questionnaire;
    - Comparison of the occurrence of side effects.
    - Veranderingen van de Eckardt-score ten opzichte van de
    uitgangswaarde zullen worden vergeleken tussen beide behandelarmen;
    - Veranderingen in HRM-parameters (hoge resolutie manometrie) ten
    opzichte van de uitgangswaarde: toename van de distale contractiliteit
    integraal, toename van de integratieve relaxerende druk 4 seconden,
    toename van het percentage succesvolle bolusklaring, afname van het
    aantal mislukte peristaltiek, toename van het percentage normale peristalsis;
    - Afname van GERD-FSSG;
    - Verbetering van de score van de SF-36 QOL-vragenlijst;
    - Vergelijking van het optreden van bijwerkingen.
    - Les changements du score Eckardt par rapport à la ligne de base seront
    comparés entre les deux bras de traitement;
    - Modifications des paramètres HRM (manométrie à haute résolution)
    par rapport à la ligne de base: augmentation de la contractilité distale
    intégrale, augmentation de la pression relaxante intégrative 4s,
    augmentation du pourcentage de clairance du bolus réussie, diminution
    du nombre d'échecs de péristaltisme, augmentation du pourcentage de
    la normale péri-stalsie;
    - Diminution du GERD-FSSG;
    - Amélioration du score du questionnaire SF-36 QOL;
    - Comparaison de la survenue d'effets secondaires.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the end of the study
    Aan het einde van de studie
    A la fin de l'étude
    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 No
    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 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 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 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state25
    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)
    none
    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 Decision2021-02-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-17
    P. End of Trial
    P.End of Trial StatusOngoing
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