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    Summary
    EudraCT Number:2017-002068-42
    Sponsor's Protocol Code Number:B14CS
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-10-09
    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 number2017-002068-42
    A.3Full title of the trial
    Open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and the acceptability of alkalising treatments at long-term in patients with cystinuria
    Etude multi-centrique, en ouvert, évaluant la sécurité d’emploi, la tolérabilité, l’efficacité, l’observance et l’acceptabilité de traitements alcalinisants à long terme chez des patients présentant une cystinurie.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study with for objective to evaluate the safety, tolerability, efficacy, compliance and acceptability of ADV7103 in patients with cystinuria
    Etude clinique avec pour objectif d'évaluer la sécurité d’emploi, la tolérabilité, l’efficacité, l’observance et l’acceptabilité de traitements alcalinisants à long terme chez des patients présentant une cystinurie.
    A.4.1Sponsor's protocol code numberB14CS
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04137978
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/252/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAdvicenne
    B.1.3.4CountryFrance
    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 supportAdvicenne
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAdvicenne
    B.5.2Functional name of contact pointClinical Project Manager
    B.5.3 Address:
    B.5.3.1Street Address21 Allée Boissy d'Anglas, ZAC De la Gare Centrale
    B.5.3.2Town/ cityNîmes
    B.5.3.3Post code30000
    B.5.3.4CountryFrance
    B.5.4Telephone number+33411 94 01 29
    B.5.6E-mailmortega@advicenne.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/3/19/2239
    D.3 Description of the IMP
    D.3.1Product nameADV7103
    D.3.4Pharmaceutical form Prolonged-release granules
    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 INNpotassium citrate monohydrate
    D.3.9.1CAS number 6100-05-5
    D.3.9.2Current sponsor codeADV7103-CK
    D.3.9.3Other descriptive namepotassium citrate
    D.3.9.4EV Substance CodeSUB14973MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number67
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpotassium hydrogen carbonate
    D.3.9.1CAS number 298-14-6
    D.3.9.2Current sponsor codeADV7103-BK
    D.3.9.3Other descriptive namepotassium bicarbonate, kalii hydrogenocarbonas, potassium acid carbonate, carbonic acid monopotassium salt, monopotassium carbonate
    D.3.9.4EV Substance CodeSUB12234MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number66
    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.IMP: 2
    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 URALYT-U (alkalinising products used as SOC)
    D.2.1.1.2Name of the Marketing Authorisation holderMeda Pharma
    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.3.1Product name alkalinising products used as SoC - Uralyt-U
    D.3.4Pharmaceutical form Granules for oral solution
    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 INNpotassium citrate
    D.3.9.3Other descriptive namePOTASSIUM CITRATE
    D.3.9.4EV Substance CodeSUB14973MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number46
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNsodium citrate
    D.3.9.3Other descriptive nameSODIUM CITRATE
    D.3.9.4EV Substance CodeSUB12582MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number39
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCitric acid
    D.3.9.3Other descriptive nameCitric Acid
    D.3.9.4EV Substance CodeSUB171900
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number14.8
    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
    Cystinuria
    Cystinurie
    E.1.1.1Medical condition in easily understood language
    Genetic disease resulting in high concentrations of the amino acid cystine in the urine, leading to the formation of cystine stones in the kidney.
    Maladie génétique provoquant des concentrations élevées d'un amino-acide, la cystine, dans les urines, entrainant la formation de cristaux puis de calculs dans le rein.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10011778
    E.1.2Term Cystinuria
    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
    To evaluate the safety and the tolerability of ADV7103 and standard of care (SoC) after a
    long-term treatment.

    Évaluer la sécurité d’emploi et la tolérabilité d’ADV7103 et de la norme de soin (NdS) après un traitement à long terme.
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of ADV7103 and SoC on urine pH during a long-term treatment
    (proportion/number of responders with urine pH values in the first morning urines and in
    pre-dose urines ≥ 7.0 and ≥ 7.5)
    • To evaluate the efficacy of ADV7103 and SoC on cystine crystalluria (including the volume
    of crystals of cystine - Vcys) and other other cystinuric parameters during a long-term
    treatment
    • To evaluate the efficacy of ADV7103 and SoC on other urine parameters during a longterm
    treatment
    • To evaluate the efficacy of ADV7103 and SoC on cystine calculi during a long-term
    treatment
    • To evaluate the time for any potential adding of a second line therapy with cystine
    chelators.
    • To evaluate the paraclinical and biological safety of ADV7103 and SoC during a long-term
    treatment.
    • Évaluer l'effet d’ADV7103 et de la NdS sur le pH urinaire pendant un traitement à long terme (proportion/nombre de répondeurs présentant des valeurs de pH urinaire des premières urines du matin et des urines en pré-dose ≥ 7.0 et ≥ 7.5)
    • Évaluer l'effet d'ADV7103 et de la NdS sur la cristallurie de la cystine (incluant le volume des cristaux de cystine - Vcys) et d'autres paramètres cystinuriques pendant un traitement à long terme.
    • Evaluer l'effet d'ADV7103 et de la NdS sur d'autres paramètres urinaires pendant un traitement à long terme.
    • Évaluer l'effet d’ADV7103 et de la NdS sur les calculs de cystine pendant un traitement à long terme.
    • Evaluer le temps d’ajout potentiel d’un traitement de seconde intention avec des produits chélateurs de cystine.
    • Evaluer la sécurité d’emploi paraclinique et biologique d'ADV7103 et de la NdS pendant un traitement à long terme.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ADV7103 Cohort :
    • Patient who has participated to and completed the previous B12CS Study or B13CS Study.

    SoC Cohort :
    • Patient who has a diagnosis of cystinuria based on medical diagnosis (at least one previous or current episode of calculus of cystine, and/or one previous or current episode of cystine crystalluria) or on genetic diagnosis (only for patients of Subset 4).
    • Patient treated with an alkalising treatment at a well-adapted dose (defined as a daily dose deemed by the investigator aiming to maintain overtime urinary pH value ≥ 7.0 and/or compatible with an acceptable safety profile and/or patient’s constraints or compliance).

    • Patient male or female, including child aged between 6 months and 17 years old and adult aged ≥ 18 years old up to 70 years old.
    Cohorte ADV7103 :
    • Patient qui a achevé l'étude précédente B12CS ou B13CS.

    Cohorte Norme de Soin (NdS) :
    • Patient qui a un diagnostic de cystinurie basé sur un diagnostic médical (au moins un précédent ou actuel épisode de calcul de cystine, et/ou un précédent ou actuel épisode de cristallurie de cystine) ou un diagnostic génétique (seulement pour les patients de la Sous-classe d’âge 4))
    • Patient traité avec un traitement alcalinisant à une dose bien adaptée (définie comme étant la dose quotidienne considérée par l’investigateur comme permettant de maintenir dans le temps une valeur de pH urinaire ≥ 7.0 et/ou compatible avec un profil de sécurité d’emploi acceptable et/ou avec les contraintes et l’observance du patient)

    • Patient homme ou femme, incluant les enfants âgés entre 6 mois et 17 ans et les adultes de 18 jusqu’à 70 ans.
    E.4Principal exclusion criteria
    ADV7103 Cohort :
    • Patient who has not participated to B12CS Study or B13CS Study
    • Patient for whom any safety issue could contraindicate her/his participation to the extension study.

    Soc Cohort :
    • Patient that is receiving the second line therapy -- cystine chelating agents (sulfhydryl compounds).
    • Patient who presents kalaemia > 5.0 mmol/L.
    • Patient who presents a moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 according to Schwartz formula for the children and both MDRDs and CKD-EPI for adults).
    Cohorte ADV7103 :
    • Patient who has not participated to B12CS Study or B13CS Study
    • Patient for whom any safety issue could contraindicate her/his participation to the extension study.

    Cohorte NdS :
    • Patient en cours de traitement de seconde intention - les agents chélateurs de cystine (composés sulfhydryles).
    • Patient qui présente une kaliémie > 5.0 mmol/L.
    • Patient qui présente une insuffisance rénale modérée ou sévère (Débit de filtration glomérulaire estimé (DFGe) < 45 mL/min/1.73 m2 selon la formule de Schwartz pour les enfants et les formules MDRDs et CKD-EPI pour les adultes).
    E.5 End points
    E.5.1Primary end point(s)
    Number and proportion of patients who have experienced treatment-emergent adverse events during the course of the study.
    Nombre et proportion de patients qui ont ressenti des événements indésirables relatif au traitement au cours de l'étude.
    E.5.1.1Timepoint(s) of evaluation of this end point
    all along the study
    tout au long de l'étude
    E.5.2Secondary end point(s)
    Main secondary efficacy endpoint:
    Values of the urine pH with a glass electrode pH-meter
    Critère d'évaluation secondaire principal d'efficacité:
    Valeurs du pH urinaire à mesurer avec un pH-mètre à électrode de verre
    E.5.2.1Timepoint(s) of evaluation of this end point
    - at each site visit from Visit 1 to Visit 7, in the fresh first and second morning urines
    - between site visits, at home, at least twice a week (total 4 time-points per week), including 2 time-points per day, in the morning and in the evening at approximately 12h-interval and before the treatment administration, in the first fresh morning urines and in the fresh evening urines, respectively
    - À chaque visite sur site, de la Visite 1 (M1, Jour 1) à la Visite 7 (M24), dans les premières et secondes urines fraîches du matin avant l'administration de traitement
    - Entre les visites sur site, au domicile, au moins 2 jours par semaine (au total 4 fois par semaine), 2 fois par jour, le matin et en soirée à approximativement 12h d’intervalle et avant l'administration du traitement, dans les premières urines fraîches du matin (t0) et dans les urines fraîches du soir (t12h), respectivement.
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Acceptability, compliance and quality of life
    Acceptabilité, observance et qualité de vie
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Contextuel avec un "bras comparateur"
    Contextual with a "comparator arm"
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    préparations officinales et hospitalières
    officinal and hospital preparations
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Dernière visite du dernier patient
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    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: 42
    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: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 12
    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) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children
    Enfants
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 97
    F.4.2.2In the whole clinical trial 97
    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
    aucun
    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 Decision2018-12-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-09-17
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