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    Summary
    EudraCT Number:2020-005786-14
    Sponsor's Protocol Code Number:7830
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-07-19
    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 number2020-005786-14
    A.3Full title of the trial
    A randomized and controlled phase II national protocol in non NF1 pediatric and AYA (Adolescent and Young Adults) patients bearing a wild type BRAF gene newly diagnosed comparing a daily oral MEK inhibitor (Trametinib) versus weekly vinblastine during 18 months

    PLGG – MEKTRIC (Pediatric Low Grade Glioma – MEKinhibitor TRIal vs Chemotherapy)
    Protocole national de phase II des patients pédiatriques et AJA (Adolescents et Jeunes Adultes) non NF1 ayant un gliome de bas grade BRAF non muté en première ligne comparant un traitement par MEK inhibiteur (Trametinib) en prise orale quotidienne versus vinblastine IV hebdomadaire pendant 18 mois
    PLGG - MEKTRIC (Gliome de bas grade pédiatrique - essai des inhibiteurs de MEK vs chimiothérapie)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A randomized and controlled phase II national protocol in non NF1 pediatric and AYA (Adolescent and Young Adults) patients bearing a wild type BRAF gene newly diagnosed comparing a daily oral MEK inhibitor (Trametinib) versus weekly vinblastine during 18 months

    PLGG – MEKTRIC (Pediatric Low Grade Glioma – MEKinhibitor TRIal vs Chemotherapy)
    Protocole national de phase II des patients pédiatriques et AJA (Adolescents et Jeunes Adultes) non NF1 ayant un gliome de bas grade BRAF non muté en première ligne comparant un traitement par MEK inhibiteur (Trametinib) en prise orale quotidienne versus vinblastine IV hebdomadaire pendant 18 mois
    A.3.2Name or abbreviated title of the trial where available
    PLGG – MEKTRIC (Pediatric Low Grade Glioma – MEKinhibitor TRIal vs Chemotherapy)
    PLGG - MEKTRIC (Gliome de bas grade pédiatrique - essai des inhibiteurs de MEK vs chimiothérapie)
    A.4.1Sponsor's protocol code number7830
    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.1.2
    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 pointEric DEMONSANT
    B.5.3 Address:
    B.5.3.1Street Address1 place de l'hopital
    B.5.3.2Town/ citySTRASBOURG
    B.5.3.3Post code67091
    B.5.3.4CountryFrance
    B.5.4Telephone number003388115266
    B.5.5Fax number003388115494
    B.5.6E-maildpidrci@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 MEKINIST
    D.2.1.1.2Name of the Marketing Authorisation holderNOVARTIS
    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 nameMEKINIST
    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.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 VELBE
    D.2.1.1.2Name of the Marketing Authorisation holderEG LABO - LABORATOIREES EUROGENERICS
    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.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous 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.IMP: 3
    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 nameTrametinib 5mg poudre pour solution buvable
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation Yes
    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
    Histologically proven grade 1 glioma/mixed glio-neuronal tumors or pleomorphic xanthoastrocytoma (PXA) confirmed
    Gliome de grade 1 / tumeurs glio-neuronales mixtes ou xanthoastrocytome pléomorphe (PXA) histologiquement prouvé et confirmé
    E.1.1.1Medical condition in easily understood language
    Pediatric Low Grade Glioma
    Gliome de bas grade pédiatrique
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    A 3-year PFS (Progression Free Survival) between the standard treatment (e.g. weekly intra-veinous vinblastine) in the group of non-NF1 PLGG, characterized by a wild-type BRAF gene, to the experimental arm (e.g. daily oral MEK inhibitor, Trametinib (Mekinist©) during 18 courses (e.g. 72 weeks).
    Comparaison de la PFS (survie sans progression) sur 3 ans entre le traitement standard (par exemple, vinblastine intraveineuse hebdomadaire) dans le groupe de PLGG non-NF1, caractérisé par un gène BRAF de type sauvage, et le bras expérimental (par exemple, inhibiteur MEK quotidien par voie orale, Trametinib (Mekinist©) pendant 18 cures (par exemple 72 semaines).
    E.2.2Secondary objectives of the trial
    1. To evaluate tumor objective response rate (ORR) at 24 and 72 weeks
    2. Estimation of overall survival (OS) for each treatment arm
    3. Toxicity and safety of the new experimental compound comparatively to the control arm during treatment and until 40 days after last administration of experimental drug
    4. Comparing the quality of life (QoL) assessment between a daily oral compound and an IV weekly administration of chemotherapy
    5. Homogeneity of treatment effect across molecular strata in each group of treatment
    6. Exploratory correlation between visual outcome and the response treatment in patients with optic pathway glioma (OPG)
    7. To collect additional data on survival and security for patient benefiting from the switch from the standard arm to Trametinib supply in case of relapse/progression
    1. Évaluer le taux de réponse objective de la tumeur (ORR) à 24 et 72 semaines
    2. Estimation de la survie globale (OS) pour chaque groupe de traitement
    3. Toxicité et sécurité du nouveau composé expérimental par rapport au groupe témoin pendant le traitement et jusqu’à 40 jours après la dernière administration du traitement
    4. Comparaison de l'évaluation de la qualité de vie (QoL) entre un composé oral quotidien et une administration hebdomadaire de chimiothérapie par voie intraveineuse
    5. Homogénéité de l'effet du traitement entre les strates moléculaires dans chaque groupe de traitement
    6. Corrélation exploratoire entre le résultat visuel et la réponse au traitement chez les patients atteints d'un gliome de la voie optique (OPG)
    7. Recueillir des données supplémentaires sur la survie et la sécurité des patients bénéficiant du passage du bras standard à l'approvisionnement en tramétinib en cas de rechute/progression
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age: ≥ 1 month to ≤ 25 years
    • Signed written informed consent prior to study participation of the legal representatives and the patient if the patient is able to understand the impact of clinical trial and to give consent. For patients above 18 years, their written informed consent will be obtained.
    • Patient could be under guardianship or limited guardianship (for patient under legal guardianship, authorization is given by the legal representative of the patient under guardianship. For patient under limited guardianship, consent will be obtained from the adult under limited guardianship assisted by his or her guardian.
    • Histologically proven grade 1 glioma/mixed glio-neuronal tumors or pleomorphic xanthoastrocytoma (PXA) confirmed by local referee and/or regional RENOCLIP referee and/or national referees in neuropathology (RENOCLIP-LOC panel)
    • Determination of a negative BRAFv600 mutation by immunohistochemistry and/or molecular methods
    • Determination of 7q34 duplication status additionally to routinely done FGFR1 and MYB/MYBL1 abnormalities’ research
    • Midline tumors without proven histone H3 mutations
    • Tumor without IDH1 mutation
    • Fresh frozen tumor tissues and/or paraffin-embedded samples for further molecular biomarker testing
    • Sus-tentorial, optic pathway, midline and spine locations allowed
    • Karnofsky or Lansky ≥ 50%
    • Criteria for post-surgical treatment: severe visual or neurological symptoms at diagnosis, clinical deterioration of visual or neurological symptoms or radiological progression. The radiological progression is defined as an increase of solid part of the tumor of more than 25% compared to the pre-baseline MRI-imaging over a period of at least 3 months or the occurrence of new metastatic lesions.
    • Infants below one year of age with chiasmatic and/or hypothalamic tumor will be treated immediately after surgery, independently from neurological and/or visual progression
    • Females of childbearing potential must be willing to practice highly effective contraception during all treatment and until 6 months after the last dose of study drugs’ administration. Additionally, females of childbearing potential must have a negative serum pregnancy test within 7 days prior to start of study drugs. Boys with reproductive potential must be willing to use condom and consider contraception for partner women of childbearing potential during treatment and until 4 months after the last study drugs’ administration.
    • Patients must have adequate bone marrow function defined as: absolute neutrophil count (ANC) ≥ 1500/µL; platelets ≥ 100,000/µL and hemoglobin ≥ 9.0 g/dL
    • Patients must have adequate liver function within 7 days prior to screening: bilirubin (sum of unconjugated and conjugated) ≤ 1.5 ULN for age, ALT and AST ≤ 2.5 x upper limit of normal, alkaline phosphatase ≤ 4 x upper limit of normal, INR/PTT < 1.5 x upper limit of normal,
    • Patients must have adequate renal function within 7 days prior to screening: serum creatinine < 1.5 x upper limit of normal for age and a creatinine clearance > 60 ml/min for 1.73 m2
    • Cardiac function defined as a corrected QT (QTcF) interval < 480 msec, LVEF (left-ventricular-ejection-fraction) ≥ lower limit of normal (LLN) by echocardiogram (ECHO)
    • Adequate blood pressure control (smaller or equal to the 95th percentile for patient's age, height and gender)
    • Patients are willing and able to comply with scheduled visits, treatment plan, laboratory tests and study procedures
    • Guardians (in case of patients under 18 years) or patient if above 18 years must be affiliated to or a beneficiary of health insurance system.
    • Âge : ≥ 1 mois à ≤ 25 ans
    • Consentement éclairé écrit et signé avant la participation à l'étude par les représentants légaux et le patient si ce dernier est en mesure de comprendre l'impact de l'essai clinique et de donner son consentement. Pour les patients de plus de 18 ans, leur consentement éclairé écrit sera obtenu.
    • Le patient peut être sous tutelle ou sous curatelle (pour les patients sous tutelle légale, l'autorisation est donnée par le représentant légal du patient sous tutelle. Pour les patients sous curatelle, le consentement sera obtenu de l'adulte sous tutelle limitée assisté de son curateur
    • Gliome de grade 1 / tumeurs glio-neuronales mixtes ou xanthoastrocytome pléomorphe (PXA) histologiquement prouvé et confirmé par un arbitre local et/ou régional RENOCLIP et/ou des arbitres nationaux en neuropathologie (panel RENOCLIP-LOC)
    • Détermination d'une mutation BRAFv600 négative par immunohistochimie et/ou méthodes moléculaires
    • Détermination du statut de duplication 7q34 en plus des recherches de routine sur les anomalies FGFR1 et MYB/MYBL1
    • Tumeurs médianes sans mutations avérées de l'histone H3
    • Tumeur sans mutation IDH1
    • Tissus tumoraux frais congelés et/ou échantillons enrobés de paraffine pour des tests de biomarqueurs moléculaires supplémentaires
    • Les emplacements des tentes, des voies optiques, de la ligne médiane et de la colonne vertébrale sont autorisés
    • Karnofsky ou Lansky ≥ 50
    • Critères de traitement postopératoire : symptômes visuels ou neurologiques graves au moment du diagnostic, détérioration clinique des symptômes visuels ou neurologiques ou progression radiologique. La progression radiologique est définie comme une augmentation de la partie solide de la tumeur de plus de 25 % par rapport à l'imagerie IRM de référence sur une période d'au moins 3 mois ou l'apparition de nouvelles lésions métastatiques.
    • Les nourrissons de moins d'un an atteints d'une tumeur chiasmatique et/ou hypothalamique seront traités immédiatement après l'opération, indépendamment de la progression neurologique et/ou visuelle
    • Les femmes en âge de procréer doivent pratiquer une contraception efficace pendant tout le traitement et jusqu'à 6 mois après l'administration de la dernière dose des médicaments à l'étude. En outre, les femmes en âge de procréer doivent avoir un test de grossesse sérique négatif dans les 7 jours précédant le début du traitement. Les garçons ayant un potentiel reproductif doivent être prêts à utiliser des préservatifs et à envisager une contraception pour leur partenaire en âge de procréer pendant le traitement et jusqu'à 4 mois après l'administration de la dernière dose du médicament à l'étude.
    • Les patients doivent avoir une fonction adéquate de la moelle osseuse, définie comme suit : numération absolue des neutrophiles (NAN) ≥ 1500/µL ; plaquettes ≥ 100 000/µL et hémoglobine ≥ 9,0 g/dL
    • Les patients doivent avoir une fonction hépatique adéquate dans les 7 jours précédant le dépistage : bilirubine (somme des éléments non conjugués et conjugués) ≤ 1,5 ULN pour l'âge, ALT et AST ≤ 2,5 x limite supérieure de la normale, phosphatase alcaline ≤ 4 x limite supérieure de la normale, INR/PTT < 1,5 x limite supérieure de la normale,
    • Les patients doivent avoir une fonction rénale adéquate dans les 7 jours précédant le dépistage : créatinine sérique < 1,5 x la limite supérieure de la normale pour l'âge et une clairance de la créatinine > 60 ml/min pour 1,73 m2
    • Fonction cardiaque définie comme un intervalle QT corrigé (QTcF) < 480 msec, LVEF (left-ventricular-ejection-fraction) ≥ limite inférieure de la normale (LLN) par échocardiogramme (ECHO)
    • Contrôle adéquat de la pression artérielle (inférieur ou égal au 95e percentile selon l'âge, la taille et le sexe du patient)
    • Les patients sont disposés et capables de se conformer aux visites prévues, au plan de traitement, aux tests de laboratoire et aux procédures d'étude
    • Les tuteurs (dans le cas de patients de moins de 18 ans) ou le patient s'il a plus de 18 ans doivent être affiliés au système d'assurance maladie ou en être bénéficiaires
    E.4Principal exclusion criteria
    Patients presenting a NF1 congenital disease
    • Pure optic nerve glioma
    • Completely resected tumors
    • Previous treatment except tumor surgery
    • Pregnancy and lactation
    • Participation in other clinical trials during all protocol
    • Prior non-surgical therapy for this indication
    • Diffuse intrinsic pontine glioma (DIPG), even if histologically diagnosed as WHO grade II
    • Subependymal giant astrocytoma (SEGA) in patients with TSC
    • Patient having a known diagnosis of human immunodeficiency virus (HIV) infection, hepatitis B or C
    • Known hypersensitivity to drugs or excipients
    • History of another malignancy
    • History of current uncontrolled infection
    • Patients présentant une maladie congénitale de type neurofibromatose de type 1 (NF1)
    • Gliome isolé du nerf optique
    • Des tumeurs complètement réséquées sans résidu ou évolutivité
    • Traitement antérieur, sauf chirurgie tumorale
    • Grossesse et allaitement
    • Participation à d'autres essais cliniques pendant toute la durée du protocole
    • Traitement non chirurgical préalable pour cette indication
    • Gliome pontine intrinsèque diffus (DIPG), même s'il est histologiquement diagnostiqué comme étant de grade II selon l'OMS
    • Astrocytome géant sous-épendymaire (SEGA) chez les patients atteints de Sclérose Tubéreuse de Bourneville
    • Patient ayant un diagnostic connu d'infection par le virus de l'immunodéficience humaine (VIH), d'hépatite B ou C
    • Hypersensibilité connue aux médicaments ou aux excipients
    • Antécédant d’une lesion maligne
    • Infection évolutive non contrôlée
    E.5 End points
    E.5.1Primary end point(s)
    3-year progression free survival (PFS) rates comparing both arms (standard vs experimental). The analysis will be based on PFS measured from time of 1st treatment administration up to an event, during the first 3 years of follow-up.
    Taux de survie sans progression (PFS) sur 3 ans en comparant les deux bras (standard et expérimental). L'analyse sera basée sur les taux de survie sans progression mesurés à partir de la première administration du traitement jusqu'à un événement, pendant les trois premières années de suivi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 years
    3 ans
    E.5.2Secondary end point(s)
    • Tumor response at 24 and 72 weeks based on the international and recognized RANO criteria
    • 3-year OS rates for both arms. The OS calculation will take into account the survival measured from time of 1st treatment administration up to death.
    • Frequency and description of AE/SAE/SUSAR (Adverse Event/Serious Adverse Event) based on CTCAE criteria focusing on visual disturbances, skin, intestinal and cardiac side effects in the experimental arm compared with the standard control arm
    • QoL based on specific questionnaires at 24 weeks, at the end of treatment and 3 years after 1st treatment administration in both arms. This analysis is based on PEDsQL questionnaires
    • 3-year PFS and OS rates according to molecular biomarkers obtained at the entry of the study and based on stratification done at randomization of patients in both arms
    • 3-year PFS and OS rates according to visual outcome with an analysis of visual function (LogMAR scale) at baseline, at 24 weeks, at the end of treatment and 3 years after 1st treatment administration (both arms are planned to be stratified on locations)
    • Survival rate (OS, PFS), response rate and AE/SAE/SUSAR rate in the group where a switch from the standard arm to the experimental drug is done at relapse/progression
    • Réponse aux tumeurs à 24 et 72 semaines sur la base des critères internationaux et reconnus du RANO
    • Taux de OS sur 3 ans pour les deux bras. Le calcul de l'OS prendra en compte la survie mesurée à partir de l'administration du premier traitement jusqu'au décès.
    • Fréquence et description des EI/EIG/SUSAR (Evénement Indésirable/Evénement Indésirable Grave/ Effets Indésirables Graves Inattendus) sur la base des critères CTCAE axés sur les troubles visuels, les effets secondaires cutanés, intestinaux et cardiaques dans le bras expérimental par rapport au bras de contrôle standard
    • Qualité de vie basée sur des questionnaires spécifiques à 24 semaines, à la fin du traitement et 3 ans après l'administration du premier traitement dans les deux bras. Cette analyse est basée sur les questionnaires PEDsQL
    • Taux de PFS et de OS sur 3 ans selon les biomarqueurs moléculaires obtenus à l'entrée de l'étude et basés sur une stratification faite au hasard des patients dans les deux bras
    • Taux de PSF et de SG à 3 ans selon le résultat visuel avec une analyse de la fonction visuelle (échelle LogMAR) au départ, à 24 semaines, à la fin du traitement et 3 ans après la première administration du traitement (il est prévu de stratifier les deux bras sur les lieux)
    • Taux de survie (OS, PFS), taux de réponse et taux EI/EIG/SUSAR dans le groupe qui passera du bras standard au médicament expérimental en cas de rechute/progression
    E.5.2.1Timepoint(s) of evaluation of this end point
    3 years
    3 ans
    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 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) 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 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
    Pour les patients randomisés dans le bras standard et présentant une progression ou une rechute dans
    For patients randomized in the standard arm and experiencing a progression or a relapse on therapy (
    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
    Vinblastine
    Vinblastine
    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 concerned25
    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
    last visit of the last subject undergoing the trial
    last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years9
    E.8.9.1In the Member State concerned months1
    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: 134
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 134
    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: 134
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 134
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 134
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 134
    F.1.3Elderly (>=65 years) No
    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 No
    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 state134
    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 Decision2021-10-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-30
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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