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    EudraCT Number:2019-000883-40
    Sponsor's Protocol Code Number:TP0004
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-12-20
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-000883-40
    A.3Full title of the trial
    An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
    Studio di estensione in aperto per valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di rozanolixizumab in soggetti con trombocitopenia immune (ITP) primaria persistente o cronica
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the long-term safety, tolerability, and efficacy of Rozanolixizumab in study participants with persistent or chronic primary immune thrombocytopenia (ITP)
    Studio per valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di rozanolixizumab in soggetti con ITP primaria
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberTP0004
    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 SponsorUCB Biopharma SRL
    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 supportUCB Biopharma SRL
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB Biosciences GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Str. 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post code40789
    B.5.4Telephone number000000
    B.5.5Fax number0000000
    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/3/18/2131
    D.3 Description of the IMP
    D.3.1Product nameRozanolixizumab
    D.3.2Product code [UCB7665]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1584645-37-3
    D.3.9.2Current sponsor codeUCB7665
    D.3.9.4EV Substance CodeSUB166282
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number140
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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 Yes
    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
    Primary Immune Thrombocytopenia
    Trombocitopenia immune primaria
    E.1.1.1Medical condition in easily understood language
    Primary immune thrombocytopenia is an autoimmune disease which is characterized by an isolated low platelet count (thrombocytopenia) and the absence of other causes of thrombocytopenia.
    La trombocitopenia immune primaria è una malattia autoimmune che è caratterzzata da un'isolata bassa conta piastrinica (trombocitopenia) e dall'assenza di altre cause di trombocitopenia.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10043554
    E.1.2Term Thrombocytopenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only)
    Valutare la sicurezza e la tollerabilità a lungo termine del trattamento ripetuto con rozanolixizumab (solo Braccio di trattamento di mantenimento)
    E.2.2Secondary objectives of the trial
    -Assess the long-term clinical efficacy of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only)
    -Assess effect of rozanolixizumab on health-related quality of life (HRQoL)
    -Assess the pharmacodynamic (PD) effect of rozanolixizumab (Maintenance Treatment Arm only)
    -Assess the effect of rozanolixizumab on patient-reported outcomes (PROs)
    -Assess the reduction in use of steroids in study participants receiving rozanolixizumab
    - Valutare l’efficacia clinica a lungo termine del trattamento ripetuto con rozanolixizumab (solo Braccio di trattamento di mantenimento)
    - Valutare l’effetto di rozanolixizumab sulla qualità di vita correlata alla salute (HRQoL)
    - Valutare l’effetto farmacodinamico (PD) di rozanolixizumab (solo Braccio di trattamento di mantenimento)
    - Valutare l’effetto di rozanolixizumab sugli esiti riferiti dai pazienti (PRO)
    - Valutare la riduzione nell’uso di steroidi nei partecipanti allo studio che ricevono rozanolixizumab
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Study participants who have consented can participate in the pharmacogenomics sub-study

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: I partecipanti allo studio che hanno acconsentito possono partecipare al sotto-studio di farmacogenomica
    E.3Principal inclusion criteria
    Study participant completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 28 (Week 25) and, in the opinion of the
    investigator and sponsor, has been compliant with the TP0003 or TP0006 study assessments
    -The study participant is considered reliable and capable of adhering to the protocol, visit schedule, or medication intake according to the
    judgment of the investigator
    -If taking allowed concomitant medications, study participant must have been on stable doses
    - Study participants may be male or female:
    a) A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study
    treatment and refrain from donating sperm during this period
    b) A female participant is eligible to participate if she is not pregnant as confirmed by a negative urine pregnancy test or not planning to get
    pregnant during the breastfeeding, and at least one of the following conditions applies:
    Not a woman of childbearing potential (WOCBP)
    A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the final dose of study
    Il partecipante allo studio ha completato lo studio TP0003 [NCT04200456] o TP0006 [NCT04224688] fino alla visita 28 (settimana 25) e, secondo il parere del investigatore e sponsor, è conforme alle valutazioni dello studio TP0003 o TP0006
    -Il partecipante allo studio è considerato affidabile e in grado di aderire al protocollo, al programma delle visite o all'assunzione del farmaco secondo il
    giudizio dell'investigatore
    -Se si assumono farmaci concomitanti consentiti, i partecipanti allo studio devono assumere dosi stabili
    - I partecipanti allo studio possono essere maschi o femmine:
    a) Un partecipante di sesso maschile deve accettare di usare la contraccezione durante il periodo di trattamento e per almeno 3 mesi dopo la dose finale del trattamento in studio e di astenersi dal donare lo sperma durante questo periodo
    b) Una donna partecipante ha diritto a partecipare se non è incinta, come confermato da un test di gravidanza negativo sulle urine o se non ha intenzione di pianificare una gravidanza durante l'allattamento e almeno una delle seguenti condizioni si applica:
    non è una donna con potenziale di gravidanza (WOCBP)
    Un WOCBP che accetta di seguire la guida contraccettiva durante il periodo di trattamento e per almeno 3 mesi dopo la dose finale del trattamento di studio
    E.4Principal exclusion criteria
    -Study participant has any ongoing investigational medicinal product (IMP)-related serious adverse event (SAE) or ongoing severe IMPrelated
    treatment-emergent adverse event (TEAE) experienced during TP0003 or TP0006
    -Study participant had any relevant resolved IMP-related SAE or severe IMP-related TEAE experienced during TP0003 or TP0006 that was not
    discussed and approved as acceptable for enrollment into open-label extension (OLE) study by Medical Monitor or designee
    -Study participant has, at last available assessment of TP0003 or TP0006, 3.0x upper limit of normal (ULN) of any of the following: alanine
    aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP)
    -Il partecipante allo studio ha in corso un evento avverso grave (SAE) correlato al medicinale sperimentale (IMP) o un evento avverso serio emergente dal trattamento (TEAE) correlato al medicinale sperimentale (IMP) insorto durante le sperimentazioni TP0003 o TP0006
    -Il partecipante allo studio ha avuto un qualsiasi SAE rilevante e correlato a IMP che si è risolto o un grave TEAE correlato all'IMP sperimentaale durante TP0003 o TP0006 che non è stato discusso e approvato come accettabile per la partecipazione dello studio di estensione in aperto (OLE) da parte del Medical Monitor o designato-
    -Il partecipante allo studio ha infine una valutazione disponibile al TP0003 o TP0006, il limite superiore di 3.0x del normale (ULN) di uno dei seguenti elementi: alanina aminotransferasi (ALT), aspartato aminotransferasi (AST) o alcalino fosfatasi (ALP)
    E.5 End points
    E.5.1Primary end point(s)
    1 Manifestarsi di eventi avversi durante il trattamento (TEAE)
    2 Manifestarsi di TEAE che determinano l’interruzione di rozanolixizumab
    1. Occurrence of treatment-emergent adverse events (TEAEs)
    2. Occurrence of TEAEs leading to withdrawal of rozanolixizumab
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. and 2. From Baseline to end of Safety Follow-Up Period (up to Week 61)
    1. e 2. Dal basale sino alla fine del periodo di follow-up di sicurezza (fino alla settimana 61)
    E.5.2Secondary end point(s)
    1. Stable Clinically Meaningful Response without rescue therapy at > o = 70% of the visits starting after the second dose of rozanolixizumab until Week 54
    2. Change from Baseline in European Quality of Life-5 Dimension 5 Levels Assessment (EQ5D-5L)
    3. Change from Baseline in Short-Form 36-Item (SF-36) Survey
    4. Change from Baseline in ITP-Patient Assessment Questionnaire (ITPPAQ)
    5. Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
    6. Minimum value in total serum immunoglobulin G (IgG) concentration over time
    7. Maximum decrease from Baseline in total serum IgG concentration over time
    8. Change from Baseline in serum IgG subclasses concentration over time
    9. Change from Baseline in serum Ig concentrations (IgA, IgE, IgM) over time
    10. Change from Baseline in Physical Fatigue Instrument
    11. Change from Baseline in Patient Global Impression of Severity (PGIS)
    12. Change from Baseline in Patient Global Impression of Change (PGIC)
    13. Area under the curve (AUC) of the oral steroid dose over time
    1 Risposta clinicamente significativa senza ricorso alla terapia di emergenza al > o = 70% delle visite a partire dalla seconda dose di rozanolixizumab fino alla Settimana 54
    2 Variazione rispetto al Basale al questionario EQ-5D-5L (European Quality of Life-5 Dimensions 5 Levels Assessment)
    3 Variazione rispetto al Basale al questionario SF-36 (Short-Form 36-item Survey)
    4 Variazione rispetto al basale al questionario ITP-PAQ (ITP-Patient Assessment Questionnaire)
    5 Risposta definita come variazione dal Basale a un livello pari o superiore alla soglia definita per il punteggio relativo ai sintomi del questionario ITP-PAQ
    6 Valore minimo della concentrazione sierica totale di IgG nel tempo
    7 Massima riduzione rispetto al Basale della concentrazione sierica totale di IgG nel tempo
    8 Variazione rispetto al Basale della concentrazione sierica delle sottoclassi di IgG nel tempo
    9 Variazione rispetto al Basale delle concentrazioni sieriche di immunoglobuline (IgA, IgE, IgM) nel tempo
    10 Variazione rispetto al Basale al Physical Fatigue Instrument
    11 Variazione rispetto al Basale dell’indice PGI-S (Patient Global Impression of Severity)
    12 Variazione rispetto al Basale dell’indice PGI-C (Patient Global Impression of Change)
    13 AUC della dose di steroidi per via orale nel tempo
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. After second dose of rozanolixizumab until Week 54 (up to Week 54) 2.-7.; 12.; 13. From Baseline during the Treatment Period (up to Week
    55) 8.-11. From Baseline during the Treatment Period (up to Week 53)
    1. Dopo la seconda dose di rozanolixizumab fino alla settimana 54 (fino alla settimana 54) 2.-7 .; 12 .; 13. Dal basale durante il periodo di trattamento (fino alla settimana 55) 8.-11. Dal basale durante il periodo di trattamento (fino alla settimana 53)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability, Immunogenicity
    Tollerabilità Immunogenicità
    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. 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 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 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.8.2.4Number 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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA90
    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
    Hong Kong
    Moldova, Republic of
    Russian Federation
    United Kingdom
    United States
    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 (LVLS)
    Ultima visita ultimo soggetto ( 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 months8
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days15
    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: 138
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 72
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 210
    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)
    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 Decision2020-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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