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    Summary
    EudraCT Number:2016-002596-10
    Sponsor's Protocol Code Number:INCB54828-203
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-01-18
    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 number2016-002596-10
    A.3Full title of the trial
    A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the
    Efficacy and Safety of INCB054828 in Subjects With Myeloid/Lymphoid
    Neoplasms With FGFR1 Rearrangement
    Studio di fase 2, in aperto, multicentrico teso a valutare l'efficacia e la sicurezza di INCB054828 in monoterapia in soggetti affetti da neoplasie mieloidi/linfoidi con riarrangiamento di FGFR1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of INCB054828 in Subjects With Myeloid/Lymphoid
    Neoplasms With FGFR1 Rearrangement
    Studio di fase 2, in aperto, multicentrico teso a valutare l'efficacia e la sicurezza di INCB054828 in monoterapia in soggetti con tumori mieloidi/linfoidi con riarrangiamento di FGFR1
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberINCB54828-203
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    A.5.4Other Identifiers
    Name:NANumber:NA
    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 SponsorINCYTE CORPORATION
    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 supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointinformazioni sulle sperimentazioni
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.4Telephone number13024252734
    B.5.5Fax number13024252734
    B.5.6E-mailRA@incyte.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 namePemigatinib
    D.3.2Product code INCB054828
    D.3.4Pharmaceutical form 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 INNPemigatinib
    D.3.9.2Current sponsor codeINCB054828
    D.3.9.4EV Substance CodeSUB183791
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2 to 4
    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
    Lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead
    to FGFR1 activation
    Neoplasia linfoide o mieloide con riarrangiamento 8p11 noto per portare all¿attivazione di FGFR1
    E.1.1.1Medical condition in easily understood language
    Lymphoid or myeloid neoplasm with 8p11 rearrangement
    Tumore linfoide o mieloide con riarrangiamento 8p11
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10072680
    E.1.2Term Fibroblast growth factor 23 increased
    E.1.2System Organ Class 10022891 - Investigations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10009714
    E.1.2Term CNS neoplasia
    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
    The primary objective of this study is to evaluate the efficacy of
    INCB054828 in subjects with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1 rearrangement.
    L¿obiettivo principale di questo studio ¿ valutare l¿efficacia di INCB054828 in soggetti affetti da neoplasie mieloidi/linfoidi con riarrangiamento del recettore del fattore di crescita dei fibroblasti (FGFR) 1.
    E.2.2Secondary objectives of the trial
    The secondary objective is to evaluate the safety of INCB054828 in subjects with myeloid/lymphoid neoplasms with FGFR1 rearrangement.
    L¿obiettivo secondario ¿ valutare la sicurezza di INCB054828 in soggetti affetti da neoplasie mieloidi/linfoidi con riarrangiamento FGFR1.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Men and women, aged 18 or older.
    • Documented lymphoid or myeloid neoplasm with 8p11 rearrangement
    known to lead to FGFR1 activation, based on standard diagnostic
    cytogenetic evaluation performed locally, before signing informed
    consent for this study.
    • Only subjects who are not candidates for stem cell transplantation, or
    have relapsed after stem cell transplantation and delayed lymphocyte
    infusion and who have progressed and are not candidates for other
    disease-modifying therapies are eligible for the study. All
    relapsed/refractory subjects must have evidence of either cytogenetic or
    hematological disease and have no evidence of residual toxicity (eg,
    graft-versus-host disease requiring treatment).
    • Life expectancy = 12 weeks.
    • ECOG performance status 0 to 2.
    • Uomini o donne di età =18 anni.
    • Neoplasia mieloide o linfoide con riarrangiamento 8p11, nota per portare all’attivazione di FGFR1, documentata prima di firmare il modulo di consenso informato per questo studio.

    • Aspettativa di vita =12 settimane.
    • Stato di validità ECOG da 0 a 2.
    E.4Principal exclusion criteria
    • Prior receipt of a selective FGFR inhibitor.
    • History and/or current evidence of ectopic mineralization/calcification,
    including but not limited to soft tissue, kidneys, intestine, myocardia, or
    lung, except calcified lymph nodes and asymptomatic arterial or
    cartilage/tendon calcifications. • Current evidence of corneal disorder/keratopathy, including but not
    limited to bullous/band keratopathy, corneal abrasion,
    inflammation/ulceration, and keratoconjunctivitis, as confirmed by
    ophthalmologic examination.
    • Use of any potent cytochrome P450 3A4 inhibitors or inducers within
    14 days or 5 half-lives (whichever is shorter) before the first dose of
    study drug.
    • Trattamento pregresso con un inibitore selettivo di FGFR.
    • Anamnesi e/o evidenza corrente di mineralizzazione/calcificazione ectopica, comprese a titolo esemplificativo ma non esaustivo quelle a livello di tessuto molle, reni, intestino, miocardio o polmoni, eccetto linfonodi calcificati e calcificazioni asintomatiche a livello di arterie o di cartilagine/tendine.
    • Evidenza corrente di disturbo corneale/cheratopatia, comprese a titolo esemplificativo ma non esaustivo cheratopatia bollosa/a banda, abrasione corneale, infiammazione/ulcera e cheratocongiuntivite, come confermato da esame oftalmologico.
    • Uso di qualsiasi inibitore o induttore potente del citocromo P450 3A4 nei 14 giorni o nelle 5 emivite (qualsiasi sia il periodo più breve) che precedono la prima dose del farmaco dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is the proportion of subjects who
    achieve CR as determined by investigator assessment according to the
    response criteria listed in Appendix D of the protocol
    L’endpoint primario di questo studio è la percentuale di soggetti che raggiungono una risposta completa (CR), determinata dalla valutazione dello sperimentatore secondo i criteri relativi alla risposta elencati nell'Appendice D del protocollo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Bone marrow biopsies or aspirates will be obtained before
    randomization and then every 3 cycles starting from Cycle 2 Day 1. After the first 12 months of treatment, a bone marrow biopsy or aspirate will be obtained every 6 cycles for subjects who have not yet achieved
    complete cytogenic response (CCyR) or every 12 cycles if the subject has
    a confirmed CCyR until Month 24. Following Month 24, a bone marrow biopsy or aspirate will be obtained every 12 months until disease progression or end of treatment.
    Prima della randomizzazione e poi ogni 3 cicli a partire dal giorno 1 del ciclo 2 saranno prelevati campioni bioptici o di agoaspirato di midollo osseo. Dopo i primi 12 mesi di trattamento sarà eseguita la biopsia o l’agoaspirato del midollo osseo ogni 6 cicli per i soggetti che non hanno ancora raggiunto una risposta citogenica completa (CCyR) o ogni 12 cicli se i soggetti presentano una CCyR confermata, fino al mese 24. Dopo il mese 24 sarà eseguito il prelievo di un campione bioptico o di agoaspirato di midollo osseo ogni 12 mesi fino a progressione della malattia o conclusione del trattamento.
    E.5.2Secondary end point(s)
    • The proportion of subjects who achieve response, defined as a best
    response of CR or PR, as determined by investigator assessment
    according to the response criteria listed in Appendix D of the protocol.
    • The proportion of subjects who achieve a complete cytogenetic
    response (CCyR) as assessed by local analysis and investigator
    evaluation.
    • The proportion of subjects who achieve a partial cytogenetic response
    (PCyR) as assessed by local analysis and investigator evaluation.
    • Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause.
    • Duration of response, defined as the time from first assessment of CR or PR to the earlier of disease progression or death due to any cause.
    - Progression-free survival.
    - Overall survival.
    - Safety and tolerability, as assessed by evaluating the frequency, duration, and severity of adverse events; through review of findings of physical examinations, changes in vital signs, and electrocardiograms (ECGs); and through clinical laboratory blood and urine sample
    evaluations.
    - percentuale di pazienti che raggiungono una risposta, definita come migliore risposta di CR o risposta parziale (PR), determinata dalla valutazione dello sperimentatore secondo i criteri relativi alla risposta dell'Appendice D del protocollo;
    • percentuale di pazienti che raggiungono una risposta citogenetica completa (CCyR) determinata dall'analisi locale e dalla valutazione dello sperimentatore;
    • percentuale di pazienti che raggiungono una risposta citogenetica parziale (PCyR) determinata dall'analisi locale e dalla valutazione dello sperimentatore;
    • durata della CR, definita come il tempo dalla prima valutazione della CR alla primissima progressione della malattia o morte per ogni causa;
    • durata della risposta, definita come il tempo dalla prima valutazione della CR o PR alla primissima progressione della malattia o morte per ogni causa
    - Sopravvivenza libera da progressione.
    - Sopravvivenza generale.
    - Sicurezza e tollerabilit¿ valutate in base a frequenza, durata e gravit¿ degli eventi avversi, mediante esame dei riscontri degli esami obiettivi, variazione dei parametri vitali ed elettrocardiogrammi (ECG) e analisi cliniche di laboratorio di campioni ematici e di urina.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Bone marrow biopsies or aspirates (or peripheral blood if the marrow
    aspiration is a dry tap) will be obtained within 6 weeks before
    enrollment (Cycle 1 Day 1), after 6 (± 1) weeks on treatment, and then
    every 9 (± 1) weeks for the first 12 months of treatment. After the first
    12 months of treatment, a bone marrow biopsy or aspirate will be
    obtained every 18 (± 1) weeks for subjects who have not yet achieved
    complete cytogenetic response (CCyR) or every 36 (± 1) weeks if the subject has a confirmed CCyR until Month 24. Following Month 24, a
    bone marrow biopsy or aspirate will be obtained every 52 (± 2) weeks
    until disease progression or EOT.
    I campioni bioptici di midollo osseo o agoaspirato (or sangue periferico se il midollo raccolto è insufficiente) saranno ottenuti entro 6 settimane dall'arruolamento (Ciclo 1 giorno 1), dopo 6 (± 1) settimane dal trattamento, e poi dopo ogni 9 (± 1) settimane dai primi 12 mesi di trattamento. Dopo i primi 12 mesi di trattamento sarà eseguita la biopsia o l'agoaspirato del midollo osseo ogni 18 (± 1) settimane per i soggetti che non hanno ancora raggiunto la risposta citogenica completa (CCyR) o ogni 36 (± 1) settimane se i soggetti presentano una CCyR confermata, fino al mese 24. Dopo il mese 24 sarà eseguito il prelievo di un campione bioptico o di agoaspirato di midollo osseo ogni 52 (± 2) settimane fino a progressione della malattia o conclusione del trattamento.
    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 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 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 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 EEA15
    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 Information not present in EudraCT
    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 months1
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days1
    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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 26
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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
    nessuno
    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 Decision
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-09-19
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