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    Summary
    EudraCT Number:2021-000171-36
    Sponsor's Protocol Code Number:C20-54
    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:2021-01-22
    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 number2021-000171-36
    A.3Full title of the trial
    PHASE I-II EFFICACY-TOXICITY OF ARTESUNATE IN FRIEDREICH ATAXIA
    Evaluation de l’Effet de l’Artésunate dans l’Ataxie de Friedreich (AF) Etude de Tolérance et d’Efficacité de Phase I/II
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    PHASE I-II EFFICACY-TOXICITY OF ARTESUNATE IN FRIEDREICH ATAXIA (FA)
    Evaluation de l’Effet de l’Artésunate dans l’Ataxie de Friedreich (AF) Etude de Tolérance et d’Efficacité de Phase I/II
    A.3.2Name or abbreviated title of the trial where available
    ESSAI ARTEMIS
    A.4.1Sponsor's protocol code numberC20-54
    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 SponsorINSERM
    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 supportAssociation Images
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportInserm
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportImagine Institute
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationINSERM
    B.5.2Functional name of contact pointHélène Espérou
    B.5.3 Address:
    B.5.3.1Street Address8 rue de la Croix jarry
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75013
    B.5.3.4CountryFrance
    B.5.4Telephone number33144236742
    B.5.5Fax number33144236710
    B.5.6E-mailrqrc.siege@inserm.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 No
    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 nameartesunate
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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
    Friedreich Ataxia
    Ataxie de Friedreich
    E.1.1.1Medical condition in easily understood language
    Friedreich Ataxia
    Ataxie de Friedreich
    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 10017374
    E.1.2Term Friedreich's ataxia
    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
    Search for the maximum tolerated and effective dose of oral artesunate (tablet of 25 mg) to regulate iron homeostasis and TfR1 immunofluorescence in PBMC
    Rechercher la dose maximale d’artésunate oral (comprimés de 25 mg) tolérée et efficace pour réguler l’homéostasie du fer et l’immunofluorescence de TfR1 dans les PBMC.
    E.2.2Secondary objectives of the trial
    Safety of artesunate in FA patients
    Impact of stopping the intake of an effective dose of artesunate on the regulation of iron homeostasis and TfR1 immunofluorescence
    Tolérance de l’Artésunate chez les patients AF
    Impact de l’arrêt de la prise d’une dose efficace d’artésunate sur la régulation de l’homéostasie du fer et l’immunofluorescence de TfR1
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients with FA confirmed by genetic analysis
    Male patients aged minimum 16 and maximum 65
    Weight ≥ 50 kg
    Compliant patients agreeing to come to all protocol visits
    Signature of consent form by patient or by parents of minor patient
    Patients who have taken no treatment within 30 days prior to first artesunate intake, apart from cardiac treatments (antithrombotics, anticoagulants, antihypertensive drugs, β-blockers)
    Affiliation to a social security scheme

    Patients atteints d’AF confirmée par étude génétique
    Patients de sexe masculin âgés de minimum 16 ans et maximum 65 ans
    Poids ≥ 50 kg
    Patients compliant acceptant de venir à toutes les visites prévues par le protocole
    Signature du consentement par le patient ou par les autorités parentales si patient mineur
    Patients n’ayant pris aucun traitement dans les 30 jours en dehors des traitements cardiaques (antithrombotiques, anticoagulants, anti-hypertenseur, ß-bloquants) avant la première prise d'artésunate
    Affiliation à un régime de sécurité sociale ou bénéficiaire d’un tel régime
    E.4Principal exclusion criteria
    Individuals placed under judicial protection
    Female patients
    Abnormal values ​​of renal function (creatinine, creatinine clearance, etc.), hepatic function (transaminases, bilirubin, etc.), CBC (red blood cells, leucocytes, platelets, etc.)
    Associated progressive disease (cancer, hepatitis, etc.)
    Treatment interfering with iron transport within 30 days before the first dose of artesunate
    Participation in another therapeutic trial
    Hypersensitivity to artesunate or to any component of the drug
    Personnes faisant l’objet d’une mesure de sauvegarde de justice
    Patients de sexe féminin
    Valeurs anormales de la fonction rénale (créatinine, clairance de créatinine …), hépatique (transaminases, bilirubine …), de la NFS (hématies, leucocytes, plaquettes …)
    Maladie associée évolutive (cancer, hépatites …)
    Traitement interférant avec le transport du fer dans les 30 jours avant la première prise d'artésunate
    Participation à un autre essai thérapeutique
    Hypersensibilité à l'artésunate ou à l'un des composants du médicament
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint evaluates the biological efficacy on an ex vivo marker in the absence of observed side effects. This is a binary endpoint established by comparison between compared measurements on blood samples taken before / after exposure of the subject to a 7-day intake of a given dose of the study product (artesunate).
    The effective dose of oral artesunate will be searched by detecting the biomarker (total cellular iron content) tested ex vivo in PBMC obtained by a simple blood test on EDTA.
    Each subject entering the study has a blood sample at time 0, a sample at the end of week 1 under treatment (level 1), then at the end of week 2 without treatment. Each cycle is repeated at increasing dose levels in the absence of side effect (steps 2, 3 and 4), and this as long as no reaction is observed on the in vitro test. A comparative evaluation of the slope of the regression of the intracellular iron concentration as a function of time will be calculated for each reached level: at D0 and D7 for level 1, at D14 and D21 for level 2, at D28 and D35 for level 3 and at D42 and D49 for level 4. A slope below 0.075 (or a division by 3 of the slope) will be considered as demonstrating a positive effect of the study product.
    If an effect is observed from the first dose used, the dose escalation will be stopped as an "ex vivo" efficacy on the primary endpoint selected will be observed. Otherwise, the test will be repeated for each dose.

    If an adverse effect, serious or not serious, for a voluntary patient is observed for a given dose, the maximum tolerated dose for this patient will be considered to be the dose immediately below the current dose. Since the dose escalation was justified by the absence of adverse reactions and biomarker detection at the previous dose, this patient terminates the trial.
    Le critère principal évalue l'efficacité biologique sur un marqueur ex vivo en absence d'effet secondaire observé. Il s'agit d'un critère binaire établi par comparaison entre des mesures comparées sur échantillons sanguins pris avant/ après une exposition du sujet à une prise de 7 jours d'une dose donnée de la substance étudiée (artésunate).
    La dose efficace d’artésunate per os sera ainsi recherchée par détection du biomarqueur (contenu cellulaire total en fer) testé ex vivo dans les PBMC obtenues par une simple prise de sang sur EDTA.
    Chaque sujet entrant dans l’étude bénéficie d’un prélèvement sanguin au temps 0 puis d’un prélèvement à la fin de la semaine 1 sous traitement (palier 1), puis à la fin de la semaine 2 sans traitement, chaque cycle est répété à des niveaux de dose croissants en absence d’effet secondaire (paliers 2, 3 et 4), et ce tant qu’aucun effet n’est observé sur le test in vitro. Une évaluation comparée de la pente de régression de la concentration de fer intracellulaire en fonction du temps sera calculée suivant les paliers atteints : à JO et J7 pour le palier 1, à J14 et J21 pour le palier 2, à J28 et J35 pour le palier 3 et à J42 et J49 pour le palier 4. Une pente inférieure à 0.075 (ou une division par 3 de la pente) sera considérée comme démontrant un effet positif de la substance testée.
    Si un effet est observé dès la première dose utilisée, on arrêtera l’escalade de dose dans la mesure où une efficacité « ex vivo » sur le critère primaire retenu sera observée. Dans le cas contraire, le test sera répété à chaque dose.

    Si un effet indésirable, grave ou non grave, pour un patient volontaire est observé pour une dose donnée, la dose maximale tolérée chez ce patient sera estimée être la dose immédiatement inférieure à la dose en cours. Dans la mesure où l’escalade de dose avait été justifiée par l’absence d’effet indésirable et de détection du biomarqueur à la dose précédente, l’essai s’arrête chez ce patient.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at D0 and D7 for level 1, at D14 and D21 for level 2, at D28 and D35 for level 3 and at D42 and D49 for level 4
    à JO et J7 pour le palier 1, à J14 et J21 pour le palier 2, à J28 et J35 pour le palier 3 et à J42 et J49 pour le palier 4
    E.5.2Secondary end point(s)
    Rate and description of adverse event dependng on administered doses of artesunate.
    Evolution of the response to treatment after one week without treatment in patients who presented a positive response: Comparison of the results of intracellular iron concentration in in vitro PBMC obtained with the sample at the end of the week without treatment with those obtained at the end of the week under treatment (D14 versus D7, D28 versus D21, D42 versus D35 and D56 versus D49).
    Taux et description des effets secondaires en fonction des doses administrées.
    Évolution de la réponse au traitement après une semaine sans traitement chez les patients ayant présenté une réponse positive : Comparaison des résultats de concentration de fer intracellulaire dans les PBMC in vitro obtenus avec le prélèvement à la fin de la semaine sans traitement à ceux obtenus à la fin de la semaine sous traitement (J14 versus J7, J28 versus J21, J42 versus J35 et J56 versus J49).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Rate and description of adverse event fo: Throughout the study
    Evolution of the response to treatment after one week without treatment in patients who presented a positive response: at D14 for level 1, D28 for level 2, D42 for level 3 and D56 for level 4
    Taux et description des effets secondaires: tout au long de l'étude
    Évolution de la réponse au traitement après une semaine sans traitement chez les patients ayant présenté une réponse positive : à J14 pour le palier 1, J28 pour le palier 2, J42 pour le palier 3 et J56 pour le palier 4
    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 Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Proof of concept study
    etude « preuve de concept »
    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.8.3 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 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
    LPLV
    Dernière visite du dernier patient
    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 months12
    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: 4
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 state22
    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 Decision2021-03-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-23
    P. End of Trial
    P.End of Trial StatusCompleted
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