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   43873   clinical trials with a EudraCT protocol, of which   7293   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:2020-004995-17
    Sponsor's Protocol Code Number:NL7507301820
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-03-08
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2020-004995-17
    A.3Full title of the trial
    Low dose iron chelation as TReatment of Oxidative stress in Sickle cell disease; TROS study
    Lage dosis ijzer chelatie therapie als behandeling van oxidatieve stress bij patienten met sikkel cel ziekte; de TROS studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Iron lowering treatment to reduce tissue damage in sickle cell disease
    Ijzer verlagende behandeling om weefselschade bij sikkelcel ziekte te verminderen
    A.3.2Name or abbreviated title of the trial where available
    TROS study
    TROS studie
    A.4.1Sponsor's protocol code numberNL7507301820
    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 SponsorAMsterdam UMC-AMC
    B.1.3.4CountryNetherlands
    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 supportAMC foundation
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportSHOW foundation
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmsterdam UMC-AMC
    B.5.2Functional name of contact pointErfan Nur
    B.5.3 Address:
    B.5.3.1Street AddressMeibergdreef
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105 AZ
    B.5.3.4CountryNetherlands
    B.5.6E-maile.nur@amsterdamumc.nl
    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 Exjade
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameExjade
    D.3.2Product code EMEA/H/C/000670
    D.3.4Pharmaceutical form Dispersible 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 INNDEFERASIROX
    D.3.9.1CAS number 201530-41-8
    D.3.9.4EV Substance CodeSUB21981
    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
    Sickle cell disease (an hereditary hemoglobinopathy)
    Sikkel cel ziekte, een erfelijke hemoglobinopatie.
    E.1.1.1Medical condition in easily understood language
    Sickle cell disease
    Sikkelcel ziekte
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of the study is to investigate the safety and efficacy (effect on point of sickling en pain) of deferasirox in patients met SCD without iron overload.
    De veiligheid en effectiviteit (effect op point of sickling en pijn) van deferasirox bij patienten met SCD zonder ijzer overload.


    E.2.2Secondary objectives of the trial
    In addition, the effect on neutrophil activity and phenotype, adhesion of neutrophils and red blood cells, fatigue questionairres, and other exploratory endpoints.
    Hiernaast zullen we kijken naar neutrofiel activiteit en fenotype, adhesie van neutrofielen en rode bloedcellen, vermoeidheids vragenlijsten, en andere exploratory eindpunten.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Sickle cell disease diagnosis: HbSS, or HbSβ0-thalassemia genotype
    2. Age 18-65 years
    3. Willing and able to provide written informed consent
    1. Sikkelcel diagnose: HbSS, of HbSβ0-thalassemia genotype
    2. Leefttijd 18-65 jaar
    3. Bereid en in staat om het toestemmingsformulier voor studie deelname te ondertekenen
    E.4Principal exclusion criteria
    1. Blood transfusion in the preceding four months
    2. Already using iron chelation due to iron overload
    3. Ferritin levels of <50 µg/L and/or transferrin saturation of < 0.20.
    4. LDH of < 300 U/L
    5. Pregnancy or the desire to get pregnant in the following 6 months
    6. Impaired renal function of GFR < 60 ml/min/1,73m2 (CKD-EPI).
    7. Known allergic reaction to deferasirox.
    8. Other somatic or cognitive condition disturbing adherence to study treatment
    1. Bloedtransfusie in de 4 maanden voorafgaand aan inclusie
    2. Reeds gebruik van ijzerchelatie therapie voor hoog ijzer
    3. Ferritine waarde <50 µg/L en/of transferrin saturatie < 0.20
    4. LDH < 300 U/L
    5. Zwangerschap of wens tot zwangerschap in de komende 6 maanden
    6. verminderde nierfunctie of een eGFR < 60 ml/min/1,73m2 (bepaald middels CKD-EPI)
    7. Eerdere allergische reactie op basis van behandeling met exjade
    8. Bekende somatische of cognitieve aandoening die de therapietrouw tijdens de studie mogelijk verstoren
    E.5 End points
    E.5.1Primary end point(s)
    The main endpoints of this study are safety and efficacy.
    -We will evaluate safety by analysis of adverse events, medication use and physical and laboratory examinations.
    -The primary efficacy endpoint will be the effect of deferasirox on sickling of red blood cells, measured as changes in Point of Sickling (PoS), as quantified by Oxygenscan.
    Other efficacy endpoints include:
    - the proportion of patients with decreases in hemoglobin S (HbS) %
    - oxidative stress as reflected by advanced glycation end-products (AGEs)
    Het belangrijkste eindpunt van de studie is om de veiligheid en effectiviteit van deferasirox te beoordelen voor deze indicatie.
    -De veiligheid zullen we beoordelen aan de hand van de adverse events, anamnese over het medicatie gebruik en de lichamelijke en bloed beoordelingen.
    De effectiviteit zullen we beoordelen aan de hand van verschillende effectiviteits eindpunten:
    -Kan deferasirox het sikkelen tegengaan?: beoordeling van point of sickling van rode bloedcellen (PoS)
    -Hemoglobine S (HbS) % (we verwachten dat dit vermindert)
    -Oxidative stress gemeten middels 'advanced glycation eind-producten (AGEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    T0: before start of treatment
    T1: after 2 weeks of treatment
    T2: after 4 weeks of treatment
    T3: after 6 weeks of treatment
    T4: 4 weeks after completing treatment (week 10)
    T0: voor start behandeling
    T1: na 2 weken behandeling
    T2: na 4 weken behandeling
    T3: na 6 weken behandeling
    T4: 4 weken na afronden van de behandeling (week 10)
    E.5.2Secondary end point(s)
    - To evaluate RBC degradation as expressed by phosphatidylserine (PS) exposure on the outer surface of RBC membrane and markers of hemolysis (cell-free heme, lactate dehydrogenase (LDH), bilirubin, reticulocytes and hemoglobin
    - To evaluate the effect of deferasirox on RBC HbS percentage
    - Effect of deferasirox on levels of non-transferrin bound iron (NTBI) and labile plasma iron (LPI)
    - To evaluate the effect of deferasirox on oxidative stress as expressed by intracellular metabolomics
    - To evaluate the effect of deferasirox on in vitro adhesion of RBCs
    - To evaluate the effect of deferasirox on endothelial activation as reflected by plasma levels of soluble vascular adhesion molecule-1 (sVCAM-1) and von Willebrand factor antigen (VWF:Ag)
    - To evaluate the effect of deferasirox on neutrophil activation as measured with flow cytometry and in vitro NET formation
    Het evalueren van erythrocyte degradatie (gemeten middels phosphatidylserine (PS) blootstelling op het buitenmembraan van de erythrocyte en markers van hemolyse, zoals cell vrij heem, lactaat dehydrogenase (LDH) bilirubine, reticulocyten aantal en hemoglobine.
    - Het effect van deferasirox of het niet-transferrine gebonden ijzer en labiel plasma ijzer (NTBI en LPI resp.)
    - Oxidatieve stress levels weergegeven middels metabolomics op erythrocyten.
    - Endotheel activatie, gemeten middels plasma levels van soluble adhesie molecuul-1 (sVCAM-1) en van willebrand factor antigen (VWF-ag)
    - In vitro adhesie van neutrofielen en rode bloedcellen
    - Neutrofiel activatiue gemeten middels flow cytometrie en in vitro NETS formatie
    E.5.2.1Timepoint(s) of evaluation of this end point
    T0: before start of treatment
    T1: after 2 weeks of treatment
    T2: after 4 weeks of treatment
    T3: after 6 weeks of treatment
    T4: 4 weeks after completing treatment (week 10)
    T0: voor start behandeling
    T1: na 2 weken behandeling
    T2: na 4 weken behandeling
    T3: na 6 weken behandeling
    T4: 4 weken na afronden van de behandeling (week 10)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) 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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee 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
    The last visit of the last subject undergoing the trial
    Het laatste bezoek van de laatste patient die geincludeerd is in de studie
    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 months0
    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 months0
    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: 30
    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 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 state30
    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
    Geen
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-11
    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-Tue May 07 19:58:37 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA