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    Summary
    EudraCT Number:2015-001716-36
    Sponsor's Protocol Code Number:6100
    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:2015-06-25
    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 number2015-001716-36
    A.3Full title of the trial
    LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)
    EFFET A LONG TERME ET TOLERANCE DE L’ACETATE D’ULIPRISTAL DANS LA MALADIE DE CHARCOT-MARIE-TOOTH DE TYPE 1A (UPACOMT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)
    EFFET A LONG TERME ET TOLERANCE DE L’ACETATE D’ULIPRISTAL DANS LA MALADIE DE CHARCOT-MARIE-TOOTH DE TYPE 1A (UPACOMT)
    A.3.2Name or abbreviated title of the trial where available
    UPACOMT
    UPACOMT
    A.4.1Sponsor's protocol code number6100
    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 SponsorHÔPITAUX UNIVERSITAIRES DE STRASBOURG
    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 supportHôpitaux universitaires de Strasbourg
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHôpitaux universitaires de Strasbourg
    B.5.2Functional name of contact pointDRCI
    B.5.3 Address:
    B.5.3.1Street Address1 place de l'hôpital
    B.5.3.2Town/ citystrasbourg
    B.5.3.3Post code67000
    B.5.3.4CountryFrance
    B.5.4Telephone number0033388115266
    B.5.5Fax number00333 88 11 52 40
    B.5.6E-mailDRCI@chru-strasbourg.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 elleOne
    D.2.1.1.2Name of the Marketing Authorisation holderHRA Pharma
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameULIPRISTAL ACETATE
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    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
    Charcot-Marie-Tooth disease type 1A
    maladie de CharcOt-Marie-Tooth de type 1A
    E.1.1.1Medical condition in easily understood language
    Charcot-Marie-Tooth disease type 1A
    maladie de CharcOt-Marie-Tooth de type 1A
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10008414
    E.1.2Term Charcot-Marie-Tooth disease
    E.1.2System Organ Class 100000004850
    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 biological effect of ulipristal acetate, a selective modulator of the progesterone receptor in CMT1A in humans, on the mRNA expression of the PMP22 in a skin biopsy.
    Evaluer l’effet biologique de l’acétate d’ulipristal, un modulateur sélectif des récepteurs à la progestérone, dans le CMT1A chez l’homme, sur l’expression de l’ARNm de la PMP22 dans une biopsie cutanée.



    E.2.2Secondary objectives of the trial
    - To evaluate the clinical efficacy of ulipristal acetate
    - To assess the biological effect of ulipristal acetate on serum markers of oxidative stress and in skin biopsies
    - Evaluate the clinical and biological tolerance
    - Assess the neurophysiological effects of ulipristal acetate
    - To evaluate the pharmacokinetics of ulipristal acetate
    - Evaluer l’efficacité clinique de l’acétate d’ulipristal
    - Evaluer l’effet biologique de l’acétate d’ulipristal sur des marqueurs du stress oxydant sériques et dans les biopsies de peau
    - Evaluer la tolérance clinique et biologique
    - Evaluer l’effet neurophysiologique de l’acétate d’ulipristal
    - Evaluer la pharmacocinétique de de l’acétate d’ulipristal
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male from 18 to 70 years
    Signed informed consent
    CMT1A proven genetically (17p11.2 duplication) and symptomatic
    Non-severe axonal damage
    Belong to a social security scheme subject
    Having been informed about the results of the medical examination prior enrollment
    Subject contacted with a valid phone number
    agree to use with a double partner effective method of contraception at least one barrier for the duration of the study and during the 2 weeks following the end of treatment
    Homme de 18 à 70 ans
    Consentement éclairé signé
    CMT1A prouvé génétiquement (duplication 17p11.2) et symptomatique
    Atteinte axonale non sévère
    Sujet affilié à un régime de sécurité sociale
    Sujet ayant été informé des résultats de la visite médicale préalable
    Sujet joignable avec un numéro de téléphone valide
    accepter d’utiliser avec la partenaire une méthode double de contraception efficace dont au moins une barrière pendant toute la durée de l’étude et pendant les 2 semaines qui suivent la fin du traitement
    E.4Principal exclusion criteria
    Major protected by law, under guardianship
    • Hypersensitivity to the active substance / excipient
    • Another cause of neuropathy: Chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulins, B12 deficiency, hepatitis B / C, HIV, Lyme or poliomyelitis
    • Hepatic and renal impairment
    • Lapp lactase deficiency, malabsoprtion syndrome glucose / galactose
    • Decision-going drug / plant interacting with CYP3A4 and / or inhibitor of proton pump
    • History of hypersensitivity to any product or device that can be used before, during and after the biopsy;
    • In the biopsy site: surgery, skin disease or local infection
    • Immunosuppression innate or acquired
    • Malfunction of the innate or acquired coagulation
    • a recognized addiction of alcoholism or drug addiction;
    • known healing disorders: keloids or delay healing.
    • biological control of active viral disease such as positive serology for HIV, hepatitis B or C ... (withdrawals made prior to inclusion);
    • Majeurs protégés par la loi, sous tutelle ou curatelle
    • Hypersensibilité à la substance active/excipient
    • Autre cause de neuropathie : intoxication éthylique chronique, chimiothérapie, diabète, insuffisance rénale, gammapathie monoclonale, cryoglobuline, déficit en B12, hépatite B/C, VIH, Lyme ou poliomyelite
    • Insuffisance hépatique et rénale
    • Déficit en Lapp lactase, syndrome de malabsoprtion glucose/galactose
    • Prise au long cours de médicament/plante interagissant avec le CYP3A4 et/ou d’inhibiteur de la pompe à protons
    • Antécédents d’hypersensibilité à tout produit ou dispositif susceptible d’être utilisé avant, pendant et après la biopsie ;
    • Au site de biopsie : intervention chirurgicale, maladie de peau ou infection locale
    • Immunosuppression innée ou acquise
    • Anomalie de la coagulation innée ou acquise
    • une addiction reconnue d’éthylisme ou de toxicomanie ;
    • des troubles connus de la cicatrisation : chéloïdes ou retard à la cicatrisation.
    • des témoins biologiques d’une affection virale évolutive telle que des sérologies positives pour le VIH, l’hépatite B ou C… (prélèvements réalisés avant inclusion) ;
    E.5 End points
    E.5.1Primary end point(s)
    measure in skin biopsy the variation of mRNA expression of PMP22 after 1 year of treatment with ulipristal acetate.
    Mesure dans une biopsie cutanée la variation d’expression de l’ARNm de la PMP22 après 1 an de traitement par acétate d’ulipristal.
    E.5.1.1Timepoint(s) of evaluation of this end point
    one year after randomization
    un an après le début du traitement
    E.5.2Secondary end point(s)
    1. production of markers of oxidative stress in skin biopsy
    2. clinical efficacy as measured by the QMT (Quantified Muscular Testing), the CMTNS2 (Charcot-Marie-Tooth Neuropathy Score Version 2), the T10MW (Ten-meter timed walking), the 9NHPT (Nine-hole peg test) the ONLS (Overall Neuropathy Limitations Scales), EVA (analogue rating Scale) pain / fatigue and quality of life (SF36).
    3. the biological effectiveness measured by serum samples in search of markers of oxidative stress.
    4. clinical and biological tolerance.
    1. la production de marqueurs du stress oxydant dans la biopsie de peau
    2. l’efficacité clinique mesurée par le QMT (Quantified Muscular Testing), le CMTNS2 (Charcot-Marie-Tooth Neuropathy Score version 2), le T10MW (Ten-meter timed walking), le 9NHPT (Nine-hole peg test), l’ONLS (Overall Neuropathy Limitations Scales), les EVA (Echelle d’évaluation analogique) douleur/fatigue et la qualité de vie (SF36).
    3. l’efficacité biologique mesurée par des prélevements sériques à la recherche des marqueurs du stress oxydant.
    4. la tolérance clinique et biologique .
    E.5.2.1Timepoint(s) of evaluation of this end point
    Clinical evaluations will be conducted during the screening visit, randomization and repeated at 1, 3, 6, 9, 12 and 13 months after randomization.
    Les évaluations cliniques seront réalisées pendant la visite de screening, de randomisation et répétées à 1, 3, 6, 9, 12 et 13 mois après randomisation.
    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 Yes
    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) 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 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 trial3
    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 concerned5
    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
    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 years
    E.8.9.1In the Member State concerned months
    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: 45
    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 state45
    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
    Non
    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 Decision2015-08-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-02-26
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