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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7264   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:2019-004958-29
    Sponsor's Protocol Code Number:CHUBX2019/25
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-06-04
    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 ConcernedFrance - ANSM
    A.2EudraCT number2019-004958-29
    A.3Full title of the trial
    EFFECT OF POLYETHYLENE GLYCOL TREATMENT ON INTESTINAL INFLAMMATION ASSOCIATED WITH CYSTIC FIBROSIS IN CHILDREN
    EFFET D’UN TRAITEMENT PAR POLYETHYLENE GLYCOL SUR L’INFLAMMATION INTESTINALE ASSOCIEE A LA MUCOVISCIDOSE CHEZ L’ENFANT
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Polyethylene glycol and intestinal inflammation in cystic fibrosis
    Polyéthylène glycol et inflammation digestive dans la mucoviscidose
    A.3.2Name or abbreviated title of the trial where available
    MUCOLAX
    A.4.1Sponsor's protocol code numberCHUBX2019/25
    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 SponsorCHU de Bordeaux
    B.1.3.4CountryFrance
    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 supportAOI 2019 CHU de Bordeaux
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCHU de Bordeaux
    B.5.2Functional name of contact pointClinical Trials Manager
    B.5.3 Address:
    B.5.3.1Street Address12 rue Dubernat
    B.5.3.2Town/ cityTalence
    B.5.3.4CountryFrance
    B.5.4Telephone number33557820877
    B.5.5Fax number33556794926
    B.5.6E-mailaurore.capelli@chu-bordeaux.fr
    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 FORLAX 4 g, poudre pour solution buvable en sachet FORLAX 10 g, poudre pour solution buvable en sachet-dose
    D.2.1.1.2Name of the Marketing Authorisation holderIPSEN PHARMA
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 Powder for oral solution in sachet
    D.3.4.1Specific paediatric formulation Yes
    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
    Cystic fibrosis
    Mucoviscidose
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis
    Mucoviscidose
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Study the impact of a 3-month polyethylene glycol treatment on intestinal inflammation associated with cystic fibrosis in children aged 4 to 18 years, as measured by the fecal calprotectin assay.
    Evaluer chez l’enfant de 4 à 18 ans, l’efficacité d’un traitement de 3 mois par polyéthylène glycol sur l’inflammation intestinale associée à la mucoviscidose, évaluée par le dosage de la calprotectine fécale.
    E.2.2Secondary objectives of the trial
    Study the impact of a 3-month treatment with polyethylene glycol:
    - On digestive symptoms and quality of life,
    - On the intestinal microbiota (bacterial and fungal)
    Study the co-evolution of inflammation between the gut and lungs ("the gut-lung axis") during the 3 months of polyethylene glycol treatment.
    Etudier l’efficacité à 3 mois d’un traitement par polyéthylène glycol :
    - Sur les symptômes digestifs et la qualité de vie,
    - Sur le microbiote intestinal (bactérien et fongique) et notamment le Microbial Dysbiosis Index1.
    Etudier la co-évolution de l’inflammation entre l’intestin et les poumons (« l’axe intestin-poumon ») durant les 3 mois de traitement par polyéthylène glycol.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age ≥ 4 years old and <18 years old ;
    - Patient with cystic fibrosis (sweat test > 60 mmol/l and/or molecular biology identifying mutations in the CFTR gene) with associated pancreatic insufficiency (fecal elastase <100 µg/g);
    - With a rapid calprotectin assay result via the IBDoc test (Bühlmann®) superior or equal to 250 µg/g;
    - Person affiliated or benefiting from a social security scheme;
    - Free, informed and written consent signed by the holders of parental authority and the investigator before any examination required by the research and oral and/or written consent by the participant (depending on his/her age).
    - Âge ≥ 4 ans et <18 ans ;
    - Patient atteint de mucoviscidose (test de la sueur > 60 mmol/l et/ou biologie moléculaire identifiant des mutations du gène CFTR) avec insuffisance pancréatique associée (élastase fécale <100 µg/g) ;
    - Présentant un résultat de dosage rapide de calprotectine via le test IBDoc (Bühlmann®) ≥ 250 µg/g ;
    - Personne affiliée ou bénéficiaire d’un régime de sécurité sociale ;
    - Consentement libre, éclairé et écrit signé par les titulaires de l’autorité parentale et l’investigateur avant tout examen nécessité par la recherche et assentiment oral et/ou écrit par le participant (en fonction de son âge).
    E.4Principal exclusion criteria
    - Ongoing processing that can modulate the functionality of the CFTR (such as lumacaftor-ivacaftor protein therapy);
    - Patient already on polyethylene glycol or other laxative within 3 months before the inclusion visit;
    - Patient with diarrhea at inclusion (diarrhea will be defined as the presence of 3 or more stools / day in the 7 days prior to the inclusion visit);
    - Acute viral or bacterial diarrhea in the month prior to the inclusion visit (associated with fever);
    - Cure of oral or intravenous antibiotics or antifungals in the month preceding the collection of samples;
    - Change in background treatment in the month prior to the inclusion visit (oral or inhaled corticosteroid therapy, azithromycin, inhaled antibiotic therapy, inhaled antifungal agent, proton pump inhibitors);
    - Taking probiotics in the month before the inclusion visit;
    - Transplanted patient (on immunosuppressants);
    - Patient with IBD or celiac disease;
    - Patient with digestive perforation or risk of digestive perforation;
    - Patient with ileus or suspicion of intestinal obstruction, symptomatic stenosis;
    - History of hypersensitivity to macrogol or any of the excipients
    - Holders of parental authority enjoying judicial protection.
    - Traitement en cours qui peut moduler la fonctionnalité du CFTR (thérapie protéique de type lumacaftor-ivacaftor) ;
    - Patient déjà traité par polyéthylène glycol ou autre laxatif dans les 3 mois précédents la visite d’inclusion ;
    - Patient présentant une diarrhée lors de l’inclusion (la diarrhée sera définie par la présence de 3 selles ou plus / jour dans les 7 jours précédents la visite d’inclusion) ;
    - Diarrhée aigüe virale ou bactérienne dans le mois précédent la visite d’inclusion (associée à de la fièvre) ;
    - Cure d’antibiotiques ou d’antifongiques oraux ou intraveineux dans le mois précédent le recueil des échantillons ;
    - Modification du traitement de fond dans le mois précédent la visite d’inclusion (corticothérapie orale ou inhalée, azithromycine, antibiothérapie inhalée, antifongique inhalé, inhibiteurs de pompe à proton) ;
    - Prise de probiotiques dans le mois précédent la visite d’inclusion ;
    - Patient greffé (sous immunosuppresseurs) ;
    - Patient atteint d’une Maladies Inflammatoires Chroniques de l’Intestin (MICI) ou d’une maladie cÅ“liaque ;
    - Patient ayant une perforation digestive ou risque de perforation digestive ;
    - Patient ayant un iléus ou suspicion d’occlusion intestinale, une sténose symptomatique ;
    - Antécédent d’hypersensibilité au macrogol ou à l’un des excipients
    - Titulaires de l’autorité parentale bénéficiant d’une mesure de protection judiciaire.
    E.5 End points
    E.5.1Primary end point(s)
    proportion of patients with faecal calprotectin <250 µg / g measured by an ELISA test 3 months after initiation of treatment with polyethylene glycol, testifying to the absence of intestinal inflammation or slight inflammation.
    proportion de patients avec une calprotectine fécale < 250 µg/g mesurée par un test ELISA à 3 mois de l’initiation du traitement par polyéthylène glycol, témoignant de l’absence d’inflammation intestinale ou d’une inflammation légère.
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months after initiation of treatment with polyethylene glycol
    à 3 mois de l’initiation du traitement par polyéthylène glycol
    E.5.2Secondary end point(s)
    The evolution between the initiation of treatment (D0) and 3 months of treatment with polyethylene glycol (M3):
    - Digestive symptoms and quality of life, assessed respectively by the validated questionnaire JenAwinen CF score and CFQ-R;
    - The digestive inflammatory response, assessed by:
    - The dosage of fecal calprotectin, interpreted as a quantitative variable (measured by an ELISA test),
    - Analysis of the expression of genes involved in the inflammatory and pro-oncogenic response, using NanoString Technology and the nCounters® PanCancer Immune Profiling Panel and the production of the main cyto / chemokines produced by the digestive mucosa in the stool samples ..
    - The composition of the bacterial and fungal intestinal microbiota, with:
    - High-speed sequencing and phylogenetic assignment of the bacterial flora (on MiSeq, from Illumina®)
    - High-speed sequencing of the region and phylogenetic affectation of the fungal flora (on MiSeq, from Illumina®)
    - Confirmation by quantitative PCR of the species or genera whose relative abundance will be significantly modified
    - Calculation of the MD-Index dysbiosis score for the intestinal microbiota.
    - Pulmonary inflammation, assessed by the dosage of calprotectin in the sputum.
    L’évolution entre l’initiation du traitement (J0) et 3 mois de traitement par polyéthylène glycol (M3) :
    - Des symptômes digestifs et de la qualité de vie, évalués respectivement par le questionnaire validé JenAbdomen CF score et CFQ-R ;
    - De la réponse inflammatoire digestive, évaluée par :
    - Le dosage de la calprotectine fécale, interprété en tant que variable quantitative (mesurée par un test ELISA),
    - L’analyse de l’expression des gènes impliqués dans la réponse inflammatoire et pro oncogénique, à l’aide de la Technologie NanoString et du panel nCounters® PanCancer Immune Profiling Panel et de la production des principales cyto/chimiokines produites par la muqueuse digestive dans les échantillons de selles..
    - De la composition du microbiote intestinal bactérien et fongique, avec :
    - Séquençage haut-débit et affectation phylogénétique de la flore bactérienne (sur MiSeq, de chez Illumina®)
    - Séquençage haut-débit de la région et affectation phylogénétique de la flore fongique (sur MiSeq, de chez Illumina®)
    - Confirmation par PCR quantitative des espèces ou genres dont l’abondance relative sera significativement modifiée
    - Calcul du score de dysbiose MD-Index pour le microbiote intestinal.
    - De l’inflammation pulmonaire, évaluée par le dosage de la calprotectine dans les crachats.
    E.5.2.1Timepoint(s) of evaluation of this end point
    at the initiation of treatment (D0) and at 3 months of treatment with polyethylene glycol (M3):
    à l’initiation du traitement (J0) et à 3 mois de traitement par polyéthylène glycol (M3)
    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 No
    E.8.1.1Randomised No
    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 Yes
    E.8.1.7.1Other trial design description
    Essai bicentrique, non comparatif, prospectif, de phase II selon un schéma de Fleming.
    Bicentric, non-comparative, prospective, phase II trial according to a Fleming scheme.
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months24
    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 Yes
    F.1.1Number of subjects for this age range: 23
    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: 11
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    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 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 state23
    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 Decision2020-04-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-30
    P. End of Trial
    P.End of Trial StatusOngoing
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