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    Summary
    EudraCT Number:2020-003168-22
    Sponsor's Protocol Code Number:2020/0206/HP
    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:2021-06-01
    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 number2020-003168-22
    A.3Full title of the trial
    Multicenter, uncontrolled pilot study evaluating the efficacy of eculizumab in the treatment of gemcitabine-induced thrombotic microangiopathies.
    Etude pilote, multicentrique, non contrôlée, évaluant l’efficacité de l’eculizumab dans le traitement des microangiopathies thrombotiques induites par la gemcitabine
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study evaluating the efficacy of eculizumab in the treatment of gemcitabine-induced thrombotic microangiopathies.
    Etude évaluant l’efficacité de l’eculizumab dans le traitement des microangiopathies thrombotiques induites par la gemcitabine
    A.3.2Name or abbreviated title of the trial where available
    GEMECULI
    GEMECULI
    A.4.1Sponsor's protocol code number2020/0206/HP
    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 SponsorRouen University Hospital
    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 supportRouen University Hospital
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity hospital center of Rouen
    B.5.2Functional name of contact pointBLOT
    B.5.3 Address:
    B.5.3.1Street Address1, rue de Germont
    B.5.3.2Town/ cityRouen
    B.5.3.3Post code76031
    B.5.3.4CountryFrance
    B.5.4Telephone number0033232886265
    B.5.5Fax number003302 32 88 82 87
    B.5.6E-mailJulien.Blot@chu-rouen.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 SOLIRIS®
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    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.3.1Product nameSOLIRIS®
    D.3.2Product code EU/1/07/393/001
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEculizumab
    D.3.9.1CAS number 219685-50-4
    D.3.9.4EV Substance CodeSUB25187
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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 Yes
    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
    Thrombotic microangiopathies induced by gemcitabine
    Microangiopathies thrombotiques induites par la gemcitabine
    E.1.1.1Medical condition in easily understood language
    Thrombotic microangiopathies induced by gemcitabine
    Microangiopathies thrombotiques induites par la gemcitabine
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10043645
    E.1.2Term Thrombotic microangiopathy
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective is to study the evolution of renal function (occurrence of partial or total renal remission) in patients with gemcitabine-induced thrombotic microangiopathy treated with eculizumab.
    L'objectif principal est d’étudier l’évolution de la fonction rénale (survenue d’une rémission rénale partielle ou totale) des patients présentant une microangiopathie thrombotique induite par la gemcitabine, traitée par eculizumab
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are :
    1. to describe the clinical and biological evolution (time to hematological and renal remission) of gemcitabine-induced thrombotic microangiopathy
    2. to clarify the role of the alternating complement pathway by assaying C5 and C5b9 in plasma
    3. to clarify the role and incidence of the mutations involved in the alternate complement pathway
    4. to evaluate the tolerance to eculizumab treatment in this indication
    5. To evaluate the quality of life of patients treated with eculizumab in this indication.
    Les objectifs secondaires de l’étude sont :
    1. de décrire l’évolution, sur le plan clinique et biologique (délais jusqu’à une rémission hématologique, rénale) des microangiopathie thrombotique induites par la gemcitabine
    2. de préciser le rôle de la voie alterne du complément en dosant le C5 et le C5b9 dans le plasma
    3. de préciser le rôle et l’incidence des mutations impliquées dans la voie alterne du complément
    4. d’évaluer la tolérance au traitement par eculizumab dans cette indication
    5. d’évaluer la qualité de vie des patients traités par eculizumab dans cette indication
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years old
    2. Previous treatment with gemcitabine within the last 18 months (duration of treatment ≥ 3 consecutive months and cumulative dose should be ≥ 10 grams).
    3. Neoplasia in remission or not in remission but with an estimated life expectancy > 6 months
    4. Acute renal failure defined by 1 of the following 2 criteria : Creatinine > 2 times baseline creatinine and/or diuresis < 0.5 ml/kg/h for 12 hours.
    5. Clinical and biological criteria for thrombotic microangiopathy: mechanical hemolytic anemia and/or tThrombocytopeniaPatient affiliated with a social security system,
    6. Adult patient or representative of the adult patient who has read and understood the information letter and signed the consent form.
    7. Woman of childbearing age with effective contraception as defined by the WHO (estrogen-progestin or intrauterine device or tubal ligation) for more than one month and a negative -HCG pregnancy test at baseline, for the duration of the study and for at least 5 months after the end of the treatment, or Postmenopausal woman (non-medically induced amenorrhea for at least 12 months prior to the baseline visit) or For men, use of protection during sexual intercourse for the duration of the study and for at least 60 days after the end of study treatment
    1. Age ≥ 18 ans
    2. Traitement antérieur par gemcitabine dans les 18 derniers mois (durée de traitement ≥ 3 mois consécutifs et dose cumulée doit être ≥ 10 grammes).
    3. Néoplasie en rémission ou non en rémission mais avec une espérance de vie estimée > 6 mois
    4. Insuffisance rénale aiguë définie par 1 des 2 critères suivants : Créatininémie > 2 fois la créatininémie de base et/ou diurèse < 0,5 ml/kg/h pendant 12 heures
    5. Critères cliniques et biologiques de microangiopathie thrombotique : anémie hémolytique mécanique et/ou tThrombopéniePatient affilié à un régime de sécurité sociale,
    6. Patient majeur ou représentant du patient majeur ayant lu et compris la lettre d’information et signé le formulaire de consentement
    7. 7. Femme en âge de procréer ayant une contraception efficace selon la définition de l’OMS (oestro-progestatifs ou dispositif intra-utérin ou ligature de trompes) depuis plus d’un mois et un test de grossesse par beta-HCG négatif à l’inclusion, pendant la durée de l’étude et pendant au moins 5 mois après la fin du traitement à l’étude ou Femme ménopausée (aménorrhée non médicalement induite depuis au moins 12 mois avant la visite d’inclusion)
    Ou
    Pour les hommes, utilisation d’un moyen de protection lors des rapports sexuels pendant la durée de l’étude et pendant au moins 60 jours après la fin du traitement à l’étude
    E.4Principal exclusion criteria
    1. Progressive neoplasia with a life expectancy of <6 months
    2. Patient with a contraindication to the administration of the treatment experienced: SOLIRIS® 300 mg concentrate for solution for infusion
    3. Contraindication to antibiotic prophylaxis
    4. Thrombotic microangiopathy associated with cancer (metastatic adenocarcinoma with bone marrow invasion, erythromyelemia, disseminated intravascular coagulation)
    5. Active systemic bacterial infection, untreated or confirmed sepsis (positive blood cultures within 7 days of patient inclusion and not treated with effective antibiotic therapy)
    6. Unresolved meningococcal infection
    7. Patient not vaccinated against meningococcal infection
    8. Pregnant or breastfeeding woman or proven lack of contraception
    9. Known systemic lupus erythematosus
    10. Person deprived of liberty by an administrative or judicial decision or person placed under judicial protection, under guardianship or curatorship
    11. Patient participating in another interventional clinical trial / having participated in another interventional clinical trial within 1 month
    1. Néoplasie évolutive avec une espérance de vie < 6 mois
    2. Patient présentant une contre-indication à l’administration du traitement expérimenté : SOLIRIS® 300 mg solution à diluer pour perfusion
    3. Contre-indication à l’antibioprophylaxie
    4. Microangiopathie thrombotique associée au cancer (adénocarcinome métastatique avec envahissement médullaire, érythromyélémie, coagulation intravasculaire disséminée)
    5. Infection bactérienne systémique active, non traitée ou sepsis confirmé (hémocultures positives dans les 7 jours suivant l’inclusion du patient et non traité par antibiothérapie efficace)
    6. Infection méningococcique non résolue
    7. Patient non vacciné contre une infection à méningocoques
    8. Femme enceinte ou allaitante ou absence de contraception avérée
    9. Lupus érythémateux systémique connu
    10. Personne privée de liberté par une décision administrative ou judiciaire ou personne placée sous sauvegarde de justice, sous-tutelle ou curatelle
    11. Patient participant à un autre essai clinique interventionnel / ayant participé à un autre essai clinique interventionnel dans un délai de 1 mois
    E.5 End points
    E.5.1Primary end point(s)
    Time from initiation of eculizumab treatment to renal remission
    Délai entre le début du traitement par eculizumab et la rémission rénale
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the 12 months of participation
    durant les 12 mois de participation
    E.5.2Secondary end point(s)
    1- Number of patients in haematological remission:
    • Normalization of the platelet count> 150 giga / L and decrease in LDH, on 2 successive samples over a period of 4 weeks.
    • Time between diagnosis of TAD secondary to gemcitabine and start of treatment with eculizumab
    • Time between the start of treatment with eculizumab and haematological remission: normalization of the platelet count> 150 giga / L and decrease in LDH, on 2 successive samples over a period of 4 weeks.
    • Value of biological parameters: Haptoglobin, schizocytes, Hematocrit, Hemoglobin, reticulocytes, serum creatinine at M0, M1, M2, M3, M6 and M12
    2- Number of patients with overexpression of C5b9 on the renal biopsy (if performed during treatment) and percentage of patients with overexpression of C5 and C5b9 in plasma
    3- Number of patients with a mutation involved in the alternate complement pathway
    4- Tolerance will be assessed by:
    • Number of red blood cells transfused before and after treatment with eculizumab
    • Duration of hospitalization in an intensive care unit / Resuscitation and total duration of hospitalization
    • Number of EvI and EvIG
    5- Quality of life assessed by the SF-36 questionnaire
    1- Nombre de patient en rémission hématologique :
    • Normalisation du taux de plaquettes > 150 giga/L et décroissance des LDH, sur 2 prélèvements successifs sur une période de 4 semaines.
    • Délai entre le diagnostic de MAT secondaire à la gemcitabine et le début du traitement par eculizumab
    • Délai entre le début du traitement par eculizumab et la rémission hématologique : normalisation du taux de plaquettes > 150 giga/L et décroissance des LDH, sur 2 prélèvements successifs sur une période de 4 semaines.
    • Valeur des paramètres biologiques : Haptoglobine, schizocytes, Hématocrite, Hémoglobine, réticulocytes, créatininémie à M0, M1, M2, M3, M6 et M12
    2- Nombre de patients présentant une surexpression de C5b9 sur la biopsie rénale (si réalisée au cours du soin) et pourcentage de patients présentant une surexpression de C5 et C5b9 dans le plasma
    3- Nombre de patient ayant une mutation impliquée dans la voie alterne du complément
    4- La tolérance sera évaluée par :
    • Nombre de culots globulaires transfusés avant et après le traitement par eculizumab
    • Durée d’hospitalisation en unité de soins intensifs / Réanimation et durée totale d’hospitalisation
    • Nombre d’EvI et EvIG
    5- La qualité de vie évaluée par le questionnaire SF-36
    E.5.2.1Timepoint(s) of evaluation of this end point
    During the 12 months of participation
    durant les 12 mois de participation
    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 No
    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 No
    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.8.2.4Number of treatment arms in the trial1
    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 concerned16
    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
    The end of the trial is at the LVLS, 364 days after the inclusion visit.
    La fin de l'essai est définis par la dernière visite du dernier patient, à 364 jours après la visite d'inclusion.
    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 months36
    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: 10
    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 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 state10
    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 Decision2021-09-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-26
    P. End of Trial
    P.End of Trial StatusOngoing
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