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   7294   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:2020-005289-32
    Sponsor's Protocol Code Number:GBT2104-133
    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:2021-10-12
    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 number2020-005289-32
    A.3Full title of the trial
    An Open-Label Extension Study to Evaluate the Long-Term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial
    Studio di estensione in aperto per valutare la sicurezza a lungo termine di inclacumab somministrato a partecipanti affetti da anemia falciforme che hanno partecipato a una sperimentazione clinica su inclacumab
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Long-Term Safety of Inclacumab Administered to Participants with Sickle Cell Disease
    Studio per valutare la sicurezza a lungo termine di inclacumab somministrato a partecipanti affetti da anemia falciforme
    A.3.2Name or abbreviated title of the trial where available
    A trial to evaluate the Long-Term Safety of inclacumab for VOC prevention
    Studio per valutare la sicurezza a lungo termine di inclacumab per la prevenzione delle VOC
    A.4.1Sponsor's protocol code numberGBT2104-133
    A.5.4Other Identifiers
    Name:US INDNumber:144073
    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 SponsorGLOBAL BLOOD THERAPEUTICS, 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 supportGlobal Blood Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlobal Blood Therapeutics, Inc.
    B.5.2Functional name of contact pointKalyan Obalampalli
    B.5.3 Address:
    B.5.3.1Street Address181 Oyster Point Blvd.
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number0019162840777
    B.5.5Fax number000000
    B.5.6E-mailkobalampalli@gbt.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameInclacumab
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNInclacumab
    D.3.9.1CAS number 1256258-86-2
    D.3.9.2Current sponsor code-
    D.3.9.3Other descriptive nameAnti P-selectin glycoprotein ligand-1 humanised monoclonal antibody
    D.3.9.4EV Substance CodeSUB205263
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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.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
    Sickle Cell Disease
    Anemia falciforme
    E.1.1.1Medical condition in easily understood language
    Sickle Cell Disease in participants experiencing Vaso-occlusive Crises
    Anemia falciforme in partecipanti che manifestano crisi vaso-occlusive
    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 21.0
    E.1.2Level PT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    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
    The primary objective of this study is to evaluate the long-term safety of every 12 week dosing of inclacumab in participants with sickle cell disease (SCD) that have completed a prior inclacumab clinical trial.
    L’obiettivo primario di questo studio è valutare la sicurezza a lungo termine della somministrazione della dose di inclacumab ogni 12 settimane in partecipanti affetti da anemia falciforme (AF) che hanno completato una precedente sperimentazione clinica su inclacumab.
    E.2.2Secondary objectives of the trial
    Additional objectives are to evaluate the incidence of vaso-occlusive crises (VOCs), hospitalizations, missed work/school days, red blood cell (RBC) transfusions, and quality of life (QoL) with long-term use of inclacumab.
    Ulteriori obiettivi sono valutare l’incidenza di crisi vaso-occlusive (CVO), ricoveri, giorni di lavoro/scuola persi, trasfusioni di globuli rossi (RBC) e qualità della vita (Quality of Life, [QOL]) con l’uso a lungo termine di inclacumab.
    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: Pharmacology Substudy (section 5.6 of the protocol)
    Samples collected for analysis of inclacumab PK, immunogenicity, or PD may additionally be used for further development and validation of the respective assay.
    Pharmacokinetics
    In approximately the first 30 participants enrolled, plasma samples will be collected for measurement of inclacumab concentrations on Day 1 (predose), at the Week 6 Visit, at Week 12 (predose), Week 24 (predose), Week 36 (predose), and Week 48 (predose). Population PK analysis using nonlinear mixed effects modeling will be performed to
    characterize inclacumab PK in plasma. If the Day 1 predose collection was performed on the same day as the last study visit in the originating GBT-Sponsored clinical study, the predose sample does not need to be collected. If Day 1 for this study is on a different day than the completion of the originating study, a predose sample will be obtained
    within 30 minutes prior to dose administration, per Appendix B.
    Anti-drug Antibodies
    In approximately the first 30 participants enrolled, plasma samples will be collected for assessment of ADA incidence at predose on Day 1, Week 12, and Week 48.
    Pharmacodynamics
    In approximately the first 30 participants enrolled, whole blood samples will be collected for assessment of non-activated and TRAP-activated PLA formation and platelet P-selectin expression. Serum samples will be collected for assessment of P-selectin inhibition by SPR. The relationships between PK, PD, clinical laboratory results, safety, and
    other outcomes will be explored.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sottostudio di farmacologia (sezione 5.6 del protocollo)
    I campioni raccolti per l’analisi di PK, immunogenicità o PD di inclacumab possono inoltre essere utilizzati per l’ulteriore sviluppo e convalida del rispettivo saggio.
    Farmacocinetica
    Approssimativamente nei primi 30 partecipanti arruolati, saranno raccolti campioni di plasma per la misurazione delle concentrazioni di inclacumab il Giorno 1 (pre-dose), alla Visita della Settimana 6, alla Settimana 12 (pre-dose), alla Settimana 24 (pre-dose), alla Settimana 36 (pre-dose) e alla Settimana 48 (pre-dose). Sarà eseguita un’analisi PK di popolazione utilizzando modelli a effetti misti non lineari per caratterizzare la PK di inclacumab nel plasma. Se il prelievo pre-dose al Giorno 1 è stato eseguito lo stesso giorno dell’ultima visita dello studio nello studio clinico originario sponsorizzato da GBT, il campione pre-dose non deve essere prelevato. Se il Giorno 1 di questo studio corrisponde a un giorno diverso da quello del completamento dello studio originario, sarà prelevato un campione pre-dose entro 30 minuti prima della somministrazione della dose, come da Appendice B.
    Anticorpi anti-farmaco
    Approssimativamente nei primi 30 partecipanti arruolati, saranno raccolti campioni di plasma per la valutazione dell’incidenza di anticorpi anti-farmaco (antidrug antibodies, [ADA]) pre-dose al Giorno 1, alla Settimana 12 e alla Settimana 48.
    Farmacodinamica
    Approssimativamente nei primi 30 partecipanti arruolati, saranno prelevati campioni di sangue intero per la valutazione della formazione di aggregati leucociti-piastrine, (Platelet-leukocyte aggregates, [PLA]) non attivati e attivati dal peptide attivante il recettore della trombina (thrombin receptor activating peptide, [TRAP]) e dell’espressione della P-selectina piastrinica. Saranno prelevati campioni di siero per la valutazione dell’inibizione della P-selectina mediante risonanza plasmonica di superficie (Surface Plasmon Resonance, [SPR]). Saranno esplorate le relazioni tra PK, PD, esami clinici di laboratorio, sicurezza ed altri esiti.
    E.3Principal inclusion criteria
    1. Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study.
    2. Participant has completed the originating inclacumab study within 30 days prior to the Day 1 Visit. Participants who discontinued study drug in the originating study due to a non-study drug-related adverse event (AE), but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with inclacumab.
    3. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study.
    4. If sexually active, female participants of childbearing potential must use highly effective methods of contraception until 165 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 165 days after the last dose of study drug.
    5. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirement.
    1. Partecipante ambosesso affetto da AF che ha partecipato e ricevuto il farmaco dello studio in uno studio clinico su inclacumab sponsorizzato da GBT.
    2. Il partecipante ha completato lo studio originario su inclacumab nei 30 giorni precedenti la visita del Giorno 1. I partecipanti che hanno interrotto il farmaco dello studio nello studio originario a causa di un evento avverso (EA) non correlato al farmaco dello studio, ma che sono rimasti nello studio, possono essere idonei al trattamento in questo studio, a condizione che l’EA non costituisca un rischio per il trattamento con inclacumab.
    3. I partecipanti di sesso femminile in età fertile devono presentare un test di gravidanza sulle urine negativo prima della somministrazione della dose il Giorno 1. Nota: le partecipanti che diventano fertili durante lo studio devono essere disposte a sottoporsi a un test di gravidanza sulle urine il cui risultato deve essere negativo per poter rimanere nello studio.
    4. Se sessualmente attive, le partecipanti in età fertile devono utilizzare metodi contraccettivi altamente efficaci fino a 165 giorni dopo l’ultima dose del farmaco dello studio. Se sessualmente attivi, i partecipanti di sesso maschile devono utilizzare metodi contraccettivi di barriera fino a 165 giorni dopo l’ultima dose del farmaco dello studio.
    5. Il partecipante ha fornito il consenso informato/l’assenso scritto. Per i partecipanti minorenni, sia il consenso del rappresentante legale o tutore legale del partecipante sia l’assenso del partecipante (ove pertinente) devono essere acquisiti in base ai requisiti locali.
    E.4Principal exclusion criteria
    1. Female participant who is breastfeeding or pregnant.
    2. Participant had an infusion-related reaction (IRR) in the originating inclacumab clinical study.
    3. Participant withdrew consent from the originating inclacumab clinical study.
    4. Participant was lost to follow-up from the originating inclacumab clinical study.
    5. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
    1. Partecipanti di sesso femminile che allattano al seno o sono in stato di gravidanza.
    2. Il partecipante ha manifestato una reazione correlata all’infusione (Infusion-Related Reaction, [IRR]) nello studio clinico originario di inclacumab.
    3. Il partecipante ha ritirato il consenso dallo studio clinico originario di inclacumab.
    4. Il partecipante è risultato perso al follow-up nell’ambito dello studio clinico originario di inclacumab.
    5. Il partecipante presenta una condizione medica, psicologica, di sicurezza o comportamentale che, a giudizio dello sperimentatore, potrebbe confondere l’interpretazione della sicurezza, interferire con la conformità o precludere il consenso informato.
    E.5 End points
    E.5.1Primary end point(s)
    Not applicable
    Non applicabile
    E.5.1.1Timepoint(s) of evaluation of this end point
    Not applicable
    Non applicabile
    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 No
    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 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 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
    aperto
    open
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Colombia
    Dominican Republic
    Egypt
    Ghana
    Kenya
    Lebanon
    Nigeria
    Oman
    Saudi Arabia
    Tanzania, United Republic of
    Turkey
    United States
    Zambia
    France
    Germany
    Italy
    United Kingdom
    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
    The study will continue until all participants are no longer participating in the study or until the Sponsor determines that the study should be discontinued early due to safety reasons or participants may receive study drug if they continue to receive clinical benefit that outweighs risk as determined by the Investigator or until the participant has access to inclacumab from an alternative source (eg, through commercialization or a managed-access program).
    Lo studio continuerà fino a quando tutti i partecipanti termineranno lo studio o fino a quando lo Sponsor stabilirà che lo studio deve essere interrotto anticipatamente per motivi di sicurezza o i partecipanti potranno ricevere il farmaco dello studio se continueranno a ricevere un beneficio clinico superiore al rischio, come stabilito dallo sperimentatore, o fino a quando il partecipante avrà accesso a inclacumab da una fonte alternativa (es. commercializzazione o programma di accesso gestito).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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 Yes
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 482
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Adolescents
    Adolescenti
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 520
    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)
    Participants may receive study drug if they continue to receive clinical benefit that outweighs risk, as determined by the Investigator, until the participant has access to inclacumab from an alternative source (eg, through commercialization or a managed-access program).
    I partecipanti potranno ricevere il farmaco dello studio se continuano a trarre un beneficio clinico superiore al rischio, come stabilito dallo sperimentatore, fino a quando il partecipante avrà accesso a inclacumab da una fonte alternativa (per es. tramite commercializzazione o un programma di accesso gestito).
    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 Decision2021-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-09
    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-Mon May 13 20:59:46 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA