Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-000192-20
    Sponsor's Protocol Code Number:PL3397-A-U4003
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-10-21
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-000192-20
    A.3Full title of the trial
    A Phase 4, multicenter study to evaluate the discontinuation and re treatment in subjects with tenosynovial giant cell tumor (TGCT) previously treated with pexidartinib
    Studio di Fase 4, multicentrico, volto a valutare l’interruzione e la ripetizione del trattamento in soggetti con tumore tenosinoviale a cellule giganti (TGCT) precedentemente trattati con pexidartinib
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 4, multicenter study to evaluate the discontinuation and re treatment in subjects with tenosynovial giant cell tumor (TGCT) previously treated with pexidartinib
    Studio di Fase 4, multicentrico, volto a valutare l’interruzione e la ripetizione del trattamento in soggetti con tumore tenosinoviale a cellule giganti (TGCT) precedentemente trattati con pexidartinib
    A.3.2Name or abbreviated title of the trial where available
    --
    --
    A.4.1Sponsor's protocol code numberPL3397-A-U4003
    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 SponsorDAIICHI SANKYO INC.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDAIICHI SANKYO, INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDaiichi Sankyo , Inc.
    B.5.2Functional name of contact pointClinical Trial Information Contact
    B.5.3 Address:
    B.5.3.1Street Address211 Mt. Airy Road
    B.5.3.2Town/ cityBasking Ridge
    B.5.3.3Post codeNJ 07920
    B.5.3.4CountryUnited States
    B.5.4Telephone number0019089926400
    B.5.5Fax number000000
    B.5.6E-maileu_cta@dsi.com
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1457
    D.3 Description of the IMP
    D.3.1Product namePexidartinib
    D.3.2Product code [PLX3397]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPEXIDARTINIB
    D.3.9.1CAS number 1029044-16-3
    D.3.9.2Current sponsor codePLX3397
    D.3.9.3Other descriptive namePLX3397 HCl
    D.3.9.4EV Substance CodeSUB185585
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Tenosynovial giant cell tumor (TGCT) is a group of neoplasms including pigmented villonodular synovitis (PVNS) and giant cell tumor of the tendon sheath (GCT-TS)
    Il tumore tenosinoviale a cellule giganti (TGCT) è un gruppo di neoplasie che include la sinovite villonodulare pigmentosa [PVNS] ed il tumore a cellule giganti della guaina tendinea (GCT-TS)
    E.1.1.1Medical condition in easily understood language
    Tenosynovial giant cell tumour is a condition where the tissue surrounding the joints and tendons, called the synovial lining or synovium, expands abnormally forming outgrowths of the joint.
    Il TGCT è una condizione in cui il tessuto che circonda le articolazioni e i tendini, chiamato rivestimento sinoviale o sinovia, si espande in modo anomalo formando escrescenze dell'articolazione.
    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 LLT
    E.1.2Classification code 10062856
    E.1.2Term Giant cell tumour of tendon sheath benign
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Proportion of subjects who remain treatment-free.
    Percentuale di soggetti che rimangono liberi da trattamento
    E.2.2Secondary objectives of the trial
    - Change from Baseline in Patient Reported Outcomes (PROs) (PROMIS PF, EQ-5D-5L)
    - Safety: Total number of subjects in the safety analysis set with any AE collected between Screening and start of re-treatment or final database lock (whichever occurs first)
    - Tumor Assessment: Investigator evaluation of tumor
    - Variazione rispetto al basale negli Esiti riferiti dal paziente (PRO) (PROMIS PF, EQ-5D-5L)
    - Sicurezza: Numero totale di soggetti nella serie di analisi di sicurezza con qualsiasi EA osservato tra lo screening e l’avvio della ripetizione del trattamento o la chiusura finale della banca dati (a seconda di quale evento si verifichi prima)
    - Valutazione del tumore: Valutazione del tumore da parte dello sperimentatore
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria to be eligible for enrollment into the study:
    1. Currently enrolled and have not been discontinued from pexidartinib treatment in one of the following studies: Study PLX108-10 (ENLIVEN), Study PLX108-01, Study PL3397-A-A103, or Study PL3397-A-U126.
    2. Willing and able to complete the PROMIS (Physical Function Scale) and EQ-5D-5L (European Quality of Life) throughout the study.
    3. Willing and able to provide written informed consent form (ICF) prior to any study-related procedures and to comply with all study requirements.
    4. Females of reproductive potential must have a negative urine pregnancy test at Screening/Baseline (to be confirmed by a serum pregnancy test taken on the End-of-Treatment visit of their prior study) and should be advised to use an effective, non-hormonal method of contraception during treatment with pexidartinib and for 1 month after the last dose. Males with female partners of reproductive potential should be advised to use an effective method of contraception during treatment with pexidartinib and for 1 month after the last dose. Female partners of male patients should concurrently use effective contraceptive methods (hormonal or non-hormonal).
    Note: A female is considered of reproductive potential following menarche and until becoming postmenopausal (no menstrual period for a minimum of 12 months) unless permanently sterile (undergone a hysterectomy, bilateral salpingectomy or bilateral oophorectomy) with a confirmed by follicle stimulating hormone (FSH) test level >40 mIU/mL.
    5. Male subjects must not freeze or donate sperm starting at Screening and throughout the study period, and for at least 5 half-lives or 1 month after the final study drug administration, whichever is longer.
    Female subjects must not donate, or retrieve for their own use, ova from the time of Screening and throughout the study treatment period, and for at least 1 month or 5 half-lives after the final study drug administration, whichever is longer.
    1. Essere attualmente arruolati e non aver sospeso il trattamento con pexidartinib in uno dei seguenti studi: Studio PLX108-10 (ENLIVEN), studio PLX108-01, studio PL3397-A-A103 o studio PL3397-A-U126.
    2. Essere disposti e in grado di compilare la scala di funzionalità fisica PROMIS e l’EQ-5D-5L durante tutto lo studio.
    3. Essere disposti e in grado di fornire un consenso informato scritto prima di qualsiasi procedura correlata allo studio e di soddisfare tutti i requisiti dello studio.
    4. I soggetti di sesso femminile potenzialmente fertili devono presentare un risultato negativo al test di gravidanza sulle urine allo screening/basale (che deve essere confermato da un test di gravidanza sul siero prelevato alla visita dell’ultimo trattamento del precedente studio) e devono essere avvisati di utilizzare un metodo di contraccezione efficace non ormonale durante il trattamento con pexidartinib e per 1 mese dopo l’ultima dose. I soggetti di sesso maschile con compagne potenzialmente fertili devono essere avvisati di utilizzare un metodo contraccettivo efficace durante il trattamento con pexidartinib e per 1 mese dopo l’ultima dose. Le compagne dei pazienti di sesso maschile, contestualmente, devono utilizzare metodi contraccettivi efficaci (ormonali oppure non ormonali).
    Nota: una donna è considerata potenzialmente fertile dopo il menarca e fino alla postmenopausa (assenza di ciclo mestruale per un minimo di 12 mesi) a meno che permanentemente sterile (sottoposta a isterectomia, salpingectomia bilaterale oppure ovariectomia bilaterale) con un livello del test dell’ormone follicolo stimolante (FSH) >40 mUI/ml confermato.
    5. I soggetti di sesso maschile non devono congelare o donare sperma a partire dallo screening e per tutto il periodo dello studio e per almeno 5 emivite o 1 mese dall’ultima somministrazione del farmaco dello studio, a seconda di quale periodo sia più lungo.
    I soggetti di sesso femminile non devono donare, o recuperare per il loro utilizzo, gli ovuli dal momento dello screening e per l’intera durata del periodo di trattamento dello studio, e per almeno 1 mese o 5 emivite dall’ultima somministrazione del farmaco dello studio, a seconda di quale periodo sia più lungo.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria are NOT eligible for enrollment into the study
    1. Subject has a clinically significant abnormality identified by the Investigator at Screening on physical examination, laboratory tests, or electrocardiogram which, in the judgement of the Investigator, would preclude the subject’s safe completion of the study.
    2. Exposure to another investigational drug or current participation in other therapeutic investigational procedures, besides pexidartinib studies, within 1 month prior to start of study treatment. Any known contraindication to treatment with, including hypersensitivity to, the study drug(s) or excipients in pexidartinib.
    1. Il soggetto presenta un’anomalia clinicamente significativa identificata dallo sperimentatore allo screening tramite esame obiettivo, esami di laboratorio o elettrocardiogramma (ECG) che, secondo il parere dello sperimentatore, precluderebbe al soggetto il completamento in sicurezza dello studio.
    2. Esposizione a un altro farmaco sperimentale o attuale partecipazione ad altre procedure terapeutiche sperimentali, oltre agli studi su pexidartinib, entro 1 mese prima dell’inizio del trattamento in studio. Qualsiasi controindicazione nota al trattamento, inclusa ipersensibilità al/i farmaco/i dello studio o agli eccipienti di pexidartinib.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects who remain treatment-free at Month 12 and Month 24
    Percentuale di soggetti che rimangono liberi da trattamento al Mese 12 e al Mese 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months after last subject enrolled in the Cohort
    24 mesi dopo l'ultimo soggetto arruolato nella coorte
    E.5.2Secondary end point(s)
    Change from Baseline in Patient Reported Outcomes (PROs): Mean change from Baseline* for PROMIS PF and EQ-5D-5L quarterly for the treatment-free and Re-Treatment periods.
    *Note: Baseline is Screening values for Treatment Free Period Treatment Continuation Cohort. Baseline is reinitiated with subject entering Re-Treatment period.

    Safety: Incidence of AEs/TEAEs and SAEs, ECGs, and laboratory assessments

    Tumor Assessment: Qualitative assessment of the tumor (not applicable to Treatment-Free period)
    Variazione rispetto al basale nei PRO: Variazione media rispetto al basale* nei punteggi trimestrali di PROMIS PF e EQ-5D-5L per i periodi liberi da trattamento e di ripetizione del trattamento.
    *Nota: Il basale è costituito dai valori di screening per i periodi liberi da trattamento e di ripetizione del trattamento. Il basale riparte quando il soggetto rientra nel periodo di ripetizione del trattamento.

    Sicurezza: Incidenza di EA e SAE, ECG e valutazioni di laboratorio

    Valutazione del tumore: Valutazione qualitativa del tumore (non applicabile nel periodo libero da trattamento)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from Baseline in Patient Reported Outcomes (PROs): every 3 months

    Safety:
    - Incidence of AEs/TEAEs and SAEs: after subject signs the ICF and up to 30 days after the last dose of study medication
    - ECGs: at screening/baseline and at EOS/EOT/FU (and whenever deemed medically necessary)
    - laboratory assessments - from baseline, every study visit until EOS/EOT/FU; only for Treatment Free Period: at baseline and then every 6 months until EOS/EOT/FU
    Variazione rispetto al basale nei PRO: ogni 3 mesi

    Sicurezza:
    Incidenza di EA e SAE: dopo che il soggetto firma il consenso e fino a 30 giorni dopo l'ultima dose del farmaco in studio
    ECG: allo screening/basale e alla visita EOS/EOT/FU (e quando ritenuto necessario dal punto di vista medico)
    valutazioni di laboratorio: dal basale, ad ogni visita dello studio fino a EOS/EOT/FU; solo per il periodo libero da trattamento: al basale e poi ogni 6 mesi fino a EOS/EOT/FU
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    Studio di continuazione
    Continuation study
    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 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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Hungary
    Italy
    Netherlands
    Spain
    Taiwan
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days0
    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: 46
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 17
    F.4.2.2In the whole clinical trial 49
    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)
    Study Drug Continuation After the End of Study
    A post-trial access plan, delineating options for access to pexidartinib, will be available for subjects after completing their study participation if the subject is benefiting from the study drug.
    Continuazione dei farmaci di studio dopo la fine dello studio
    Un piano di accesso post-studio, che delinea le opzioni per l'accesso a pexidartinib, sarà disponibile per i soggetti dopo aver completato la loro partecipazione allo studio se il soggetto stia beneficiando del farmaco in studio.
    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 Decision2020-08-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-22
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Wed May 01 15:34:15 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA