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    Summary
    EudraCT Number:2017-004554-42
    Sponsor's Protocol Code Number:TAMDMD
    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:2018-08-17
    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 number2017-004554-42
    A.3Full title of the trial
    Tamoxifen in Duchenne muscular dystrophy - TAMDMD
    A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
    Le tamoxifène dans la dystrophie musculaire de Duchenne – TAMDMD
    Étude de phase 3 multicentrique, randomisée, en double aveugle, contrôlée
    contre placebo visant à évaluer la sécurité et l'efficacité pendant 48
    semaines
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.
    L'étude porte sur des garçons atteints de dystrophie musculaire de
    Duchenne. Nous menons cette étude pour étudier l'effet et la tolérance du
    tamoxifène dans cette maladie.
    A.4.1Sponsor's protocol code numberTAMDMD
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03354039
    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 SponsorUniversity of Basel Children's Hospital, Division of Neuropediatrics
    B.1.3.4CountrySwitzerland
    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 supportSwiss National Science Foundation (SNSF)
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportScientific and Technological Research Council of Turkey (TÜBÄ°TAK)
    B.4.2CountryTurkey
    B.4.1Name of organisation providing supportFrench National Research Agency (ANF)
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportDuchenne UK
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportDuchenne Parent Project Netherlands
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportAssociation Monegasque contre les myopathies
    B.4.2CountryMonaco
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationmulti-service-monitoring
    B.5.2Functional name of contact pointDr. Josef Reisinger
    B.5.3 Address:
    B.5.3.1Street AddressAhornweg 8
    B.5.3.2Town/ cityBeratzhausen
    B.5.3.3Post code93176
    B.5.3.4CountryGermany
    B.5.4Telephone number+4994939592966
    B.5.5Fax number+4994939592967
    B.5.6E-mailjosef.reisinger@multi-service-monitoring.de
    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 Tamoxifen 20mg Hexal® Filmtabletten
    D.2.1.1.2Name of the Marketing Authorisation holderHexal Ag, Industristraße 25, 83607 Holzkirchen
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical form Film-coated tablet
    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 INNTamoxifen
    D.3.9.1CAS number 54965-24-1
    D.3.9.3Other descriptive nameTAMOXIFEN CITRATE
    D.3.9.4EV Substance CodeSUB04672MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 placeboFilm-coated tablet
    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
    Duchenne muscular dystrophy
    dystrophie musculaire de Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    dystrophie musculaire de Duchenne
    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 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).
    To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 10-16 years old non-ambulant DMD patients not treated with glucocorticoids (using the MFM D2 subscore as primary endpoint.)
    Comparé au placebo, le tamoxifène réduit d'au moins 50 % la progression de la
    maladie chez les patients atteints de DMD ambulatoires âgés de 6,5 à 12 ans (en
    utilisant le sous-score MFM D1 comme critère d’évaluation clinique principal chez
    les patients du groupe A).
    Comparé au placebo, le tamoxifène réduit d'au moins 50 % la progression de la
    maladie chez les patients atteints de DMD ambulatoires âgés de 10 à 16 ans (en
    utilisant le sous-score MFM D2 comme critère d’évaluation clinique
    principal.)
    E.2.2Secondary objectives of the trial
    Secondary clinical outcomes to assess muscle function:

    1. MFM total score, the D2, and D3 MFM subscores, North Star Ambulatory Assessment, proximal upper limb function from baseline to week 48 under TAM treatment compared to placebo.

    2. Timed function tests (6 minute walking distance in meter, 10 meter walking time in seconds, time to rise from lying on the floor / supine up in seconds,) from baseline to week 48 under TAM treatment compared to placebo.

    Secondary clinical outcomes to assess muscle force:

    3. Quantitative muscle testing (using Grip force) from baseline to week 48 under TAM treatment compared to placebo.

    Secondary surrogate marker to assess muscle degeneration:

    4. Quantitative muscle MRI including muscle fat fraction (MFF) and T2 times of thigh muscles visualised by MRI from baseline to week 48 under TAM treatment compared to placebo.
    1.Résultat total de MFM, les sous-résultats D2 et D3 de MFM, North Star Ambulatory Assessment, fonction proximale des membres supérieurs, du point de référence à la semaine 48 sous traitement TAM comparé à un placebo.
    2.Tests de fonction chronométrés (distance parcourue en mètres en marchant pendant 6 minutes, temps de marche sur 10 mètres en secondes, temps pour se lever d’une position allongée au sol / couchée sur le dos en secondes), du point de référence à la semaine 48 sous traitement TAM comparé à un placebo.
    Critères d’évaluation cliniques secondaires de la force musculaire :
    3.Test quantitatif musculaire, du point de référence à la semaine 48 sous traitement TAM comparé à un placebo. Marqueur de substitut secondaire pour l’évaluation de la dégénération musculaire :
    4.IRM quantitative musculaire, y compris fraction lipidique du muscle (MFF) et temps T2 des muscles des cuisses visualisés par IRM, du point de référence à la semaine 48 sous traitement TAM comparé à un placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Group A (ambulant patients)
    - Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
    - Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
    - Male gender
    - 6.5 to 12 years of age at time of screening
    - weight >20kg
    - ambulant patients
    - able to walk at least 350 meters in 6 minute walking distance test without assistance at screening
    - MFM D1 subdomain of the MFM scale >40% at screening
    - Ability to provide informed consent and to comply with study requirements
    - Patients harbouring a nonsense mutation treatable with the
    approved drug ataluren should be under stable ataluren treatment
    for at least 3 months or in case of nontolerance being off ataluren
    treatment for at least 3 months before screening

    Group B (non-ambulant patients)
    - Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
    - patients that have stopped steroid treatment due to side effects
    or intolerance for at least 6 months before screening
    - Male gender
    - non-ambulant patients (walking distance less than 10 meters)
    - 10 to 16 years of age at time of screening
    - Ability to provide informed consent and to comply with study requirements
    Groupe A (patients ambulatoires)
    - Diagnostic documenté de DMD par analyse de la mutation au niveau du gène de la dystrophine ou en lien avec des niveaux substantiellement réduits de la protéine dystrophine (c’est-à-dire absence totale ou <5 % par rapport à la normale) par Western blot ou par immunocoloration
    - Traitement stable par glucocorticoïdes pendant >6 mois (aucun changement significatif de dosage (>0,2 mg/kg)) à la date de la sélection ; des adaptations du dosage en fonction des variations de poids sont autorisées.
    - Sexe masculin
    - 6,5 à 12 ans à la date de la sélection
    - Poids >20 kg
    - Patients ambulatoires
    - Capables de marcher au moins 350 mètres dans le cadre du test de marche de 6 minutes sans assistance à la date de la sélection
    - Sous-score MFM-D1 sur l’échelle MFM >40 % à la date de la sélection
    - Capacité à fournir un consentement éclairé et à respecter les exigences de l’essai
    - Les patients présentant une mutation non-sens traitable par le médicament approuvé ataluren doivent suivre un traitement stable par ataluren depuis au moins 3 mois ou, en cas de non-tolérance, avoir arrêté le traitement par ataluren depuis au moins 3 mois avant la sélection

    Groupe B (patients hospitalisés)
    - Diagnostic documenté de DMD par analyse de la mutation au niveau du gène de la dystrophine ou en lien avec des niveaux substantiellement réduits de la protéine dystrophine (c’est-à-dire absence totale ou <5 % par rapport à la normale) par Western blot ou par immunocoloration
    - - Patients ayant arrêté une corticothérapie en raison de ses effets indésirables ou d’une intolérance depuis au moins 6 mois avant la sélection
    - Sexe masculin
    - Patients hospitalisés (capacité à la marche inférieure à 10 mètres)
    - 10 à 16 ans à la date de la sélection
    - Capacité à fournir un consentement éclairé et à respecter les exigences de l’essai
    E.4Principal exclusion criteria
    - Known individual hypersensitivity or allergy to tamoxifen or other
    ingredients/excipients of IMP
    - Female gender
    - Use of tamoxifen or testosterone within the last 3 months
    - Known or suspected malignancy
    - Other chronic disease or clinically relevant limitation of renal, liver
    or heart function
    - Known or suspected non-compliance
    - Any injury which may impact functional testing, e.g. upper or lower limb fracture
    - Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapid progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 month prior to screening.
    - Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders of the participant/parents (as judged by the investigator)
    - Concomitant participation in any other interventional trial (and up to 3 months prior to screening)
    - Use of CYP2D6 inhibitors or of CYP3A4 inducers, platelet aggregation inhibitors and coumarin-type anti-coagulants
    - Use of drugs metabolized by CYP2C9, such as phenprocoumon,
    phenytoin, warfarin, celecoxib, fluvastatin, ginko biloba, St. John’s
    wort and sulfamethoxazol
    -Galactosemia (lack of galactose-1-phosphat-uridylyltransferase or
    UDP-galactose-4-epimerase or galactokinase; Fanconi-Bickelsyndrome); congenital lack of lactase; glucose-galactose
    malabsorption
    - Presence of one or more of the following eye disorders: cataract,
    retinopathia, optic neuropathy, alteration of the cornea
    - Presence of one or more of the following laboratory abnormalities:
    anaemia, thrombocytopenia, leukopenia, neutropenia or
    agranulocytosis
    Group A:
    - Glucocorticoid naïve patients
    - Start of glucocorticoid treatment or change in dosage <6 month prior to screening (dosing adaptations according to weight change are allowed)

    Group B:
    - Glucocorticoid treated patients or patients that stopped steroid treatment <6 month prior to screening
    - Assisted ventilation of any kind necessary
    - Hypersensibilité individuelle ou allergie connue au tamoxifène ou à d’autres ingrédients/excipients du médicament expérimental.
    - Sexe féminin
    - Utilisation de tamoxifène ou de testostérone au cours des 3 derniers mois
    - Malignité connue ou suspectée
    - Autre maladie chronique ou insuffisance rénale, hépatique ou cardiaque cliniquement pertinente
    - Non-conformité connue ou suspectée
    - Toute blessure susceptible de fausser les tests fonctionnels, par ex. une fracture d’un membre supérieur ou inférieur
    - Spondylodèse programmée ou attendue durant la période de l’essai (à l’appréciation du chercheur ; par ex. en raison d’une scoliose évoluant rapidement), un antécédent de spondylodèse ne constitue pas de contre-indication si elle a eu lieu plus de 6 mois avant la sélection.
    - Incapacité à suivre les procédures de l’essai, par ex. en raison de problème de compréhension de la langue, de troubles psychologiques du participant/de ses parents (à l’appréciation du chercheur)
    - Participation concomitante à un autre essai interventionnel (et jusqu’à 3 mois avant la sélection)
    - Utilisation d’inhibiteurs CYP2D6 ou d’inducteurs CYP3A4 (en dehors des glucocorticoïdes), d’inhibiteurs d’agrégation plaquettaire et d’anticoagulants de type coumarine
    - Utilisation de médicaments métabolisés par l’enzyme CYP2C9, par exemple le phenprocoumone, la phénytoïne, la warfarine, le célécoxib, la fluvastatine, le ginko biloba, le millepertuis perforé et le sulfaméthoxazole
    - Galactosémie (anomalie du métabolisme du galactose-1-phosphate-uridylyltransférase ou UDP-galactose-4-epimérase ou galactokinase ; syndrome de Fanconi-Bickel) ; déficit congénital en lactase ; malabsorption du glucose et du galactose
    - Présence de l’un ou de plusieurs des troubles oculaires suivants : cataracte, rétinopathie, neuropathie oculaire, altération de la cornée
    - Présence de l’une ou de plusieurs des anomalies de laboratoire
    suivantes : anémie, thrombocytopénie, leucopénie, neutropénie ou agranulocytose

    Groupe A :
    - Patients naïfs de traitement par glucocorticoïdes
    - Instauration du traitement par glucocorticoïdes ou changement de dosage <6 mois avant la sélection (des adaptations du dosage en fonction d’une variation de poids sont autorisées)

    Groupe B :
    - Patients traités par glucocorticoïdes ou patients ayant arrêté un traitement par glucocorticoïdes <6 mois avant la sélection
    - Ventilation assistée de toutes sortes obligatoire
    E.5 End points
    E.5.1Primary end point(s)
    For ambulant DMD patients (group A) the motor function measure (MFM) subscore D1 (standing and transfer) is the primary outcome. In the second patient population (non-ambulant patients, group B) the MFM D2
    subscore is the primary endpoint, allowing extrapolation of D2 data from the group A (ambulant patient) population.
    Secondary outcomes include the total MFM and subscores D2 and D3, the North Star Ambulatory Assessment (NSAA), timed function tests (TFT) including 6 minute walking distance (6MWD), proximal upper limb function (PUL), and quantitative muscle testing (Grip force). In addition, to investigate whether longterm 48-week TAM treatment can slow muscle degeneration, quantitative thigh muscle magnetic resonance imaging will be performed. Finally, patient reported outcome measures will be
    collected: Personal-Adjustment and Role Skills-Scale (PARS-III).
    Chez les patients atteints de DMD ambulatoires (groupe A), le sous-score D1 (position debout et transferts) de la mesure de la fonction musculaire (MFM) constitue le résultat principal. Dans la deuxième population de patients (patients hospitalisés, groupe B), le sous-score D2 de la MFM
    constitue le critère de jugement principal, ce qui permet l'extrapolation des données D2 à partirde la population du groupe A (patients ambulatoires).
    Les résultats secondaires comprennent le score total MFM ainsi que ses sous-scores D2 et D3, l’échelle d’évaluation North Star Ambulatory Assessment (NSAA), des tests fonctionnels chronométrés (TFT), incluant le score de 6 minutes de marche (6MWD), le score de l’Upper Limb Performance (PUL), ainsi que la mesure quantitative de la force musculaire (grip test). De plus, afin de déterminer si le traitement par TAM à long terme sur 48 semaines permet de ralentir la dégénérescence musculaire, un examen d’imagerie par résonance magnétique sera effectué pour évaluer le volume du muscle de la cuisse. Enfin, les mesures des résultats relevés chez les patients seront
    compilés : Personal-Adjustment and Role Skills-Scale (PARS-III).

    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, week 12, week 24, week 36, week 48
    Sélection, Jour 0, Semaine 12, Semaine 24, Semaine 36, Semaine 48
    E.5.2Secondary end point(s)
    Secondary clinical outcomes to assess muscle function:
    - MFM total score, the D2, and D3 MFM subscores, North Star Ambulatory Assessment from baseline to week 48 under tamoxifen treatment compared to placebo.
    - Timed function tests (6 minute walking distance in meter, 10 meter walking time in seconds, time to rise from lying on the floor / supine up in seconds,) from baseline to week 48 under tamoxifen treatment compared to placebo.
    Secondary clinical outcomes to assess muscle force:
    - Quantitative muscle testing (using Grip force) from baseline to week 48 under tamoxifen treatment compared to placebo.
    Secondary surrogate marker to assess muscle degeneration:
    - Quantitative muscle MRI including muscle fat fraction (MFF) and T2 times of thigh muscles visualised by MRI from baseline to week 48 under tamoxifen treatment compared to placebo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    MRI: Baseline, week 24, week 48
    Timed Function test, MFM: Screening, Baseline, week 12, week 24, week 36, week 48
    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 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) 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 trial4
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Switzerland
    Turkey
    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
    Visite du dernier patient (LPLV)
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 100
    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: 80
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) No
    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 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
    Boys between 6,5 years and 16 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 100
    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)
    Depending on the safety and efficacy data obtained during the study, the sponsor plans an open-label extension study to provide tamoxifen access to all DMD patients enrolled in this study.
    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 Decision2019-10-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-10-13
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