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    Summary
    EudraCT Number:2017-000684-33
    Sponsor's Protocol Code Number:OC5-DB-02
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-04-30
    Trial results View 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 number2017-000684-33
    A.3Full title of the trial
    A phase III double-blind, randomized study to evaluate the long-term
    efficacy and safety of Oxabact® in patients with primary hyperoxaluria
    Étude de phase III randomisée, en double aveugle, évaluant l’efficacité et la sécurité à long terme d’Oxabact® chez des patients atteints d’hyperoxalurie primitive.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to find out of if Oxabact is an effective and safe treatment for patients with primary hyperoxaluria compared to placebo
    A.3.2Name or abbreviated title of the trial where available
    ePHex
    A.4.1Sponsor's protocol code numberOC5-DB-02
    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 SponsorOxThera Intellectual Property AB
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportOxThera Intellectual Property AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOxThera Intellectual Property AB
    B.5.2Functional name of contact pointChief Operating Officer
    B.5.3 Address:
    B.5.3.1Street Addressc/o Ekonomi Klara Papper, Box 1062
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post codeSE-101 39
    B.5.3.4CountrySweden
    B.5.4Telephone number004686600223
    B.5.6E-mailelisabeth.lindner@oxthera.com
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/06 354 and EMA/OD/095/05
    D.3 Description of the IMP
    D.3.1Product nameOxabact_Oxalobacter formigenes strain HC-1
    D.3.2Product code OC5 (enteric coated capsules)
    D.3.4Pharmaceutical form Capsule
    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 INNOC5
    D.3.9.3Other descriptive nameOXALOBACTER FORMIGENES, STRAIN HC 1
    D.3.9.4EV Substance CodeSUB31050
    D.3.10 Strength
    D.3.10.1Concentration unit million CFU million colony forming units
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number1000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    D.3.11.13.1Other medicinal product typelive bacteria (natural, commensal, intestinal bacteria) - Oxalobacter formigenes
    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 placeboCapsule
    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
    Primary Hyperoxaluria (PH)
    Hyperoxalurie Primitive (HP)
    E.1.1.1Medical condition in easily understood language
    PH is a genetic disease associated with high levels of oxalate in the blood, the urine and throughout the body. The high oxalate levels damage the body in different ways (e.g. kidney stones)
    HP est une maladie congénitale associée à la présence de taux élévés d’oxalate dans le sang et les urines, générant des complications à différents niveaux du corps (Ex : calculs renaux).
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10020702
    E.1.2Term Hyperoxalemia
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of Oxabact® following 52 weeks treatment in
    patients with maintained kidney function, but below the lower limit of the normal range (eGFR < 90 ml/min/1.73 m2) and a total plasma oxalate concentration ≥ 10 μmol/L at baseline
    Évaluer l’efficacité d’Oxabact® après 52 semaines de traitement chez des patients dont la fonction rénale est maintenue mais se situe sous la limite inférieure de la normale (DFGe < 90 ml/min/1,73 m2) et dont la concentration plasmatique d’oxalate total est ≥ 10 μmol/l à l’inclusion.
    E.2.2Secondary objectives of the trial
    To obtain additional safety data from 52 weeks continuous treatment
    with Oxabact®
    Obtenir des données de sécurité supplémentaires sur le traitement continu d’une durée de 52 semaines par Oxabact®.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent (as applicable for the age of the subject).
    2. A diagnosis of PH (as determined by standard diagnostic methods).
    3. eGFR < 90 ml/min/1.73 m2. The Schwartz formula will be used to estimate GFR for children (age below 18), and CKD-EPI formula will be used for adults (age 18 or above).
    4. Plasma oxalate concentration ≥10 μmol/L in total plasma oxalate.
    5. Male or female patients ≥ 2 years of age.
    6. Patients receiving vitamin B6 must be receiving a stable dose for at least 3 months prior to screening and must not change the dose during the study. Patients not receiving vitamin B6 at study entry must be willing to refrain from initiating pyridoxine during study participation.
    1.Consentement éclairé signé (selon le cas en fonction de l'âge du sujet).
    2. Diagnostic de HP (tel qu'établi par les méthodes standards de diagnostic).
    3. DFGe < 90 ml/min/1,73 m2. On utilisera la formule de Schwartz pour estimer le
    DFG chez les enfants (âgés de moins de 18 ans), et la formule CKD-EPI chez les adultes (âgés de 18 ans ou plus).
    4. Concentration plasmatique d’oxalate ≥ 10 μmol/l dans l’oxalate plasmatique total.
    5. Patients de sexe masculin ou féminin âgés de ≥ 2 ans.
    6. Les patients sous vitamine B6 doivent recevoir une dose stable pendant au moins
    3 mois avant la sélection et la dose ne doit pas être modifiée au cours de l’étude. Les sujets ne recevant pas de vitamine B6 au début de l'étude doivent se déclarer prêts à ne pas commencer à prendre de la pyridoxine au cours de leur participation à l'étude.
    E.4Principal exclusion criteria
    7. Inability to swallow size 4 capsules.
    8. Subjects that have undergone transplantation (solid organ or bone marrow).
    9. Patients requiring dialysis or at immediate risk for kidney failure or expected to be in need of dialysis during the study period.
    10. The existence of secondary hyperoxaluria, e.g. hyperoxaluria due to bariatric surgery or chronic gastrointestinal diseases such as cystic fibrosis, chronic inflammatory bowel disease and short-bowel syndrome.
    11. Use of antibiotics to which O. formigenes is sensitive, including current antibiotic use, or antibiotics use within 14 days of initiating study medication.
    12. Current treatment with a separate ascorbic acid preparation.
    13. Pregnant women (or women who are planning to become pregnant) or lactating women.
    14. Women of childbearing potential who are not using adequate contraceptive precautions.
    15. Presence of a medical condition that the Investigator considers likely to make the subject susceptible to adverse effect of study treatment or unable to follow study procedures or any condition that is likely to interfere with the study drug mechanism of action (such as abnormal GI function).
    16. Participation in any interventional study of another investigational product, biologic, device, or other agent within 60 days prior to the first dose of OC5 or not willing to forego other forms of investigational treatment during this study.
    7. Patients incapables d’avaler des gélules de calibre 4.
    8. Sujets ayant subi une transplantation (organe solide ou moelle épinière).
    9. Patients devant subir une dialyse ou à risque immédiat d’insuffisance rénale ou dont on anticipe qu’ils devront subir une dialyse pendant la période de l’étude.
    10. Existence d'une hyperoxalurie secondaire, par exemple une hyperoxalurie due à une chirurgie bariatrique ou à des troubles gastro-intestinaux chroniques comme la mucoviscidose, la maladie inflammatoire chronique de l’intestin et le syndrome de l'intestin court.
    11. Utilisation d’antibiotiques auxquels O. formigenes est sensible, notamment la prise actuelle ou au cours des 14 jours précédant le début du traitement par le médicament à l’étude d’antibiotiques.
    12. Traitement en cours par une préparation distincte à base d'acide ascorbique.
    13. Femmes enceintes (ou femmes prévoyant de débuter une grossesse) ou
    allaitantes.
    14. Femmes en âge de procréer ne prenant aucune précaution contraceptive
    adéquate.
    15. Présence d’une pathologie jugée par l’investigateur comme susceptible de
    provoquer chez le patient un effet indésirable du traitement à l’étude ou de le rendre incapable de suivre les procédures de l’étude, ou toute affection susceptible de perturber le mécanisme d’action du médicament à l’étude (telle qu’une anomalie de la fonction gastro-intestinale).
    16. La participation à toute étude interventionnelle portant sur un autre produit expérimental, une substance biologique, un dispositif ou tout autre agent dans les 60 jours précédant l’administration de la première dose d’OC5, ou le refus de renoncer à d'autres formes de traitements expérimentaux pendant la durée de la présente étude.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in plasma oxalate concentration after 52 weeks of treatment (total plasma oxalate concentration).
    Variation de la concentration d’oxalate plasmatique entre l’inclusion et après 52 semaines de traitement (concentration plasmatique d’oxalate total).
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 52 weeks of treatment
    après 52 semaines de traitement
    E.5.2Secondary end point(s)
    Key secondary endpoint:
    1. Change from baseline in kidney function (eGFR) after 52 weeks of
    treatment.
    2. Frequency of kidney stones events after 52 weeks of treatment. Stone
    events are defined as (i) Patient- or investigator reported symptoms, or
    (ii) Stone passages or removals, or (iii) Increase in number of stones
    assessed by ultrasound.
    Other endpoints
    3. Change from baseline in myocardial function in the heart as measured
    by Speckle Tracking Echocardiography (STE) and traditional
    echocardiography.
    4. Change from baseline in free plasma oxalate concentration after 52
    weeks of treatment.
    5. Change from baseline in urinary oxalate excretion after 52 weeks of
    treatment.
    6. Change from baseline in grade of nephrocalcinosis as assessed by
    Ultrasound.
    7. Change in number of O. formigenes in stool.
    8. Correlation between change in number of O. formigenes in stool and
    change in total plasma oxalate concentration.
    9. Change from baseline in score of Quality of Life questionnaire.
    10. Change from baseline in markers for renal tubular capacity and
    inflammation:
    Urine: magnesium, phosphorus, citrate, calcium, glycolate, creatinine,
    urea, calcium oxalate crystals, pH, osmolality and urinary volume.
    Blood: magnesium, phosphorus, citrate, calcium, glycolate, BUN, ALP,pH, bicarbonate, CRP, WBC, creatinine and cystatine C
    Principaux critères d’évaluation secondaires :
    1.Variation de la fonction rénale (DFGe) entre l’inclusion et après 52 semaines de traitement.
    2. Fréquence des événements de calculs rénaux après 52 semaines de traitement. Les événements de calculs sont définis comme (1) symptômes rapportés par le patient ou l’investigateur, ou (2) passages ou retraits de calculs, ou (3) augmentation du nombre de calculs évalué par échographie
    Autres critères d’évaluation
    3. Variation de la fonction myocardique par rapport à l’inclusion mesurée par l’échocardiographie de suivi des marqueurs acoustiques et l’échocardiographie classique.
    4. Variation de la concentration d’oxalate plasmatique libre rapport à l’inclusion après 52 semaines de traitement.
    5. Variation de l’excrétion d’oxalate dans l’urine par rapport à l’inclusion après 52 semaines de traitement.
    6. Variation par rapport à l’inclusion du grade de néphrocalcinose évalué par échographie.
    7. Variation du nombre d’O. formigenes dans les selles.
    8. Corrélation entre la variation du nombre d’O. formigenes dans les selles et la variation de la concentration plasmatique d’oxalate total.
    9. Variation par rapport à l’inclusion du score au questionnaire sur la qualité de vie.
    10. Variation des marqueurs de la capacité tubulaire et de l’inflammation rénales par rapport à l’inclusion : Urine : magnésium, phosphore, citrate, calcium, glycolate, créatinine, urée, cristaux d’oxalate de calcium, pH, osmolalité et volume urinaire. Sang : magnésium, phosphore, citrate, calcium, glycolate, AUS, PAL, pH, bicarbonate, CRP, globules blancs, créatinine et cystatine C.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. after 52 weeks of treatment
    2. after 52 weeks of treatment
    3. after 48 weeks of treatment
    4. after 52 weeks of treatment
    5. after 52 weeks of treatment
    6. after 48 weeks of treatment
    7. after 52 weeks of treatment
    8. after 52 weeks of treatment
    9. Quality of life will be taken once during baseline and this is compared
    to the results of week 8, 24, 40 and 52.
    10. after 52 weeks of treatment.
    1. après 52 semaines de traitement
    2. après 52 semaines de traitement
    3. après 48 semaines de traitement
    4. après 52 semaines de traitement
    5. après 52 semaines de traitement
    6. après 48 semaines de traitement
    7. après 52 semaines de traitement
    8. après 52 semaines de traitement
    9.Les questionnaire sur la qualité de vie seront complétés deux fois
    pendant la période d'inclusion et la moyenne sera comparée aux
    résultats collectés au Jour 0, Semaine 8, 24, 40 et 52.
    10.après 52 semaines de traitement

    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 Yes
    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 Yes
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA7
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    France
    Germany
    Netherlands
    Spain
    Tunisia
    United Kingdom
    United States
    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 years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 16
    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: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children are recruited for this study. Informed Consent will be obtained from their parents or legal guardians as appropriate
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 22
    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)
    The patients will be offered to continue in an open-label follow-up protocol with Oxabact® treatment directly after the 52 weeks treatment period.
    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 Decision2017-06-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-04-15
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