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    Summary
    EudraCT Number:2020-000501-93
    Sponsor's Protocol Code Number:BCX9930-201
    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:2021-01-25
    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 number2020-000501-93
    A.3Full title of the trial
    A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Uno studio di fase 2, in aperto, per valutare la sicurezza a lungo termine di BCX9930 per via orale in soggetti affetti da emoglobinuria parossistica notturna (EPN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long term study of BCX9930 in patients with HPN
    Uno studio a lungo termine su BCX9930 in pazienti affetti da EPN
    A.3.2Name or abbreviated title of the trial where available
    A long term study of BCX9930 in patients with HPN
    Uno studio a lungo termine su BCX9930 in pazienti affetti da EPN
    A.4.1Sponsor's protocol code numberBCX9930-201
    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 SponsorBIOCRYST PHARMACEUTICALS 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 supportBioCryst Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointClinical Operations UK
    B.5.3 Address:
    B.5.3.1Street Address26-28 Hammersmith Grove
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW6 7BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442088341144
    B.5.5Fax number+442088341156
    B.5.6E-mailoperations@ams-europe.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 nameBCX9930
    D.3.2Product code [BCX9930]
    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 INNBCX9930
    D.3.9.2Current sponsor codeBCX9930
    D.3.9.3Other descriptive nameBCX9930 hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 200
    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
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Emoglobinuria Parossistica Notturna (EPN)
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Emoglobinuria Parossistica Notturna (EPN)
    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 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess long-term safety and tolerability data in eligible subjects with paroxysmal nocturnal hemoglobinuria (PNH) who previously received BCX9930 in a BioCryst-sponsored study and derived benefit from BCX9930 treatment
    Valutare la sicurezza e la tollerabilità a lungo termine in soggetti idonei con emoglobinuria parossistica notturna (EPN) che in precedenza avevano assunto il BCX9930 in uno studio sponsorizzato da BioCryst e che hanno tratto beneficio dal trattamento con il BCX9930.
    E.2.2Secondary objectives of the trial
    • To assess the continued effectiveness of BCX9930 in treatment of PNH during long-term administration
    • To evaluate patient-reported outcomes (PRO) of long-term BCX9930 treatment
    • To collect PK, PD, and complement biomarker data in subjects with PNH for incorporation into meta-study models of population PK and PK/PD.
    • To characterize the effects of BCX9930 in subjects with PNH by
    clinical measurements and PD and complement biomarkers.
    • To evaluate biomarkers of complement activation during episodes of breakthrough hemolysis (BTH)
    • Valutare l'efficacia continua del BCX9930 nel trattamento della EPN durante la somministrazione a lungo termine
    • Valutare i risultati riportati dal paziente (PRO) del trattamento a lungo termine con il BCX9930
    • Raccogliere dati su PK, PD e biomarcatori del complemento in soggetti con EPN da incorporare in modelli di meta-studio della popolazione PK e PK/PD.
    • Caratterizzare gli effetti del BCX9930 in soggetti affetti da EPN mediante misurazioni cliniche, PD e biomarcatori del complemento.
    • Valutare i biomarcatori dell'attivazione del complemento durante gli episodi di emolisi intercorrente (BTH)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able to provide written informed consent
    2. Male or non-pregnant, non-lactating female subjects
    3. Subjects who have successfully participated in a previous BCX9930 study of PNH and who experienced a clinical benefit, as confirmed by the Investigator. Successful participation is defined as completion of planned duration of dosing with BCX9930 in the prior study, not inclusive of any extension period.
    4. Female participants must meet at least 1 of the following
    contraception requirements:
    a. Be a woman of childbearing potential who agrees to use a highly
    effective contraceptive method throughout the study and for a duration of 30 days after the last dose of study drug.
    b. Alternatively, true abstinence is acceptable for women of
    childbearing potential when it is in line with the subject's preferred and usual lifestyle.
    c. Be a woman of nonchildbearing potential.
    5. Male participants must meet the following contraception
    requirements:
    a. Subjects with female partners of childbearing potential must agree to utilize a highly effective contraceptive method.
    b. Alternatively, true abstinence is acceptable when it is in line with the subject's preferred and usual lifestyle.
    6. In the opinion of the Investigator, the subject is expected to
    adequately comply with all required study procedures and restrictions for the duration of the study.
    1. Capacità di fornire un consenso informato scritto
    2. Soggetti di sesso maschile o femminile non in gravidanza e non in allattamento
    3. Soggetti che hanno partecipato con successo a un precedente studio sul BCX9930 per la EPN e che hanno riscontrato un beneficio clinico, come confermato dallo Sperimentatore. Il successo della partecipazione viene definito al completamento della durata pianificata del dosaggio di BCX9930 nello studio precedente, senza includere alcun periodo di proroga.
    4. I partecipanti di sesso femminile devono soddisfare almeno 1 dei seguenti requisiti contraccettivi:
    a. Essere una donna in età fertile che accetta di utilizzare un metodo contraccettivo altamente efficace durante lo studio e per una durata di 30 giorni dopo l'ultimo dosaggio somministrato del farmaco di studio.
    b. In alternativa, è accettabile la vera astinenza per le donne in età fertile quando in linea con lo stile di vita preferito e abituale del soggetto.
    c. Essere una donna potenzialmente non fertile.
    5. I partecipanti di sesso maschile devono soddisfare i seguenti requisiti contraccettivi:
    a. I soggetti con partner femminili in età fertile devono concordare di utilizzare un metodo contraccettivo altamente efficace.
    b. In alternativa, è accettabile la vera astinenza quando in linea con lo stile di vita preferito e abituale del soggetto.
    6. Secondo il parere dello Sperimentatore, è previsto che il soggetto adempia in modo adeguato a tutte le procedure e restrizioni di studio richieste, per tutta la durata dello studio
    E.4Principal exclusion criteria
    1. Any clinically significant medical or psychiatric condition that, in the opinion of the Investigator or Sponsor, would interfere with the
    subject's ability to participate in the study or participation would
    increase the risk for that subject
    2. Any clinically significant history of angina, known coronary artery
    disease, myocardial infarction, syncope, clinically significant cardiac arrhythmias, left ventricular hypertrophy, cardiomyopathy, aortic stenosis, or any other cardiovascular abnormality.
    3. Chronic systemic corticosteroid use. Note: Topical, inhaled, ocular, or nasal sprays containing corticosteroids are allowed.
    4. Investigational drug exposure, other than BCX9930, within 30 days of the baseline visit, or 5.5 half-lives of the investigational drug (whichever is longer)
    5. For subjects requiring a screening visit:
    a. Clinically significant abnormal ECG at the screening visit. This
    includes, but is not limited to, a QT interval corrected using Fridericia's method (QTcF) > 450 msec in males and > 470 msec in females, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping.
    b. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) (Exception: Subjects may be enrolled with ALT or AST > 3 × ULN if explained by a documented chronic elevation due to hemolysis. In these cases, ALT and AST must be < 5 × ULN.)
    6. Daily use of medications listed in the currently applicable prohibited medications list
    7. Pregnant, planning to become pregnant, or having been pregnant within 90 days of baseline, or lactating
    1. Qualsiasi condizione medica o psichiatrica clinicamente significativa che, secondo l'opinione dello Sperimentatore o dello Sponsor, potrebbe interferire con la possibilità del soggetto di partecipare allo studio o la cui partecipazione aumenterebbe il rischio per quel soggetto
    2. Qualsiasi storia clinicamente significativa di angina, coronaropatia, infarto miocardico, sincope, aritmie cardiache clinicamente significative, ipertrofia ventricolare sinistra, cardiomiopatia, stenosi aortica o qualsiasi altra anomalia cardiovascolare.
    3. Uso di corticosteroidi sistemici cronici. Nota: sono ammessi spray topici, per inalazione, oculari o nasali contenenti corticosteroidi.
    4. Esposizione a farmaci sperimentali, diversi dal BCX9930, entro 30 giorni dalla visita basale o 5,5 emivite del farmaco sperimentale (a seconda di quale sia il periodo più lungo)
    5. Per i soggetti che richiedono una visita di screening:
    a. Un ECG significativamente anomalo da un punto di vista clinico durante la visita di screening. Ciò include, ma non è limitato a, un intervallo di QT corretto usando il metodo di Fridericia (QTcF) > 450 msec nei maschi e> 470 msec nelle femmine, o contrazioni ventricolari e/o atriali premature più frequenti di quelle occasionali e/o come distici o superiore nel raggruppamento.
    b. Alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) > 3 × limite superiore alla norma (ULN) (eccezione: i soggetti possono essere arruolati con ALT o AST> 3 × ULN se spiegato da un aumento cronico documentato dovuto all'emolisi. In questi casi, ALT e AST devono essere <5 x ULN.)
    6. Uso quotidiano dei farmaci elencati nell'elenco dei farmaci vietati attualmente applicabile
    7. In gravidanza, in pianificazione di gravidanza, o in stato di gravidanza nei 90 giorni precedenti il baseline, o in allattamento
    E.5 End points
    E.5.1Primary end point(s)
    Measurement of safety and tolerability by subject incidence of graded
    treatment-emergent adverse events (TEAEs), laboratory abnormalities, changes to vital signs, electrocardiogram (ECG) results, and physical examination findings
    Misurazione della sicurezza e della tollerabilità per incidenza soggettiva di eventi avversi emergenti da trattamento (TEAE) valutati, anomalie di laboratorio, modifiche ai parametri vitali, risultati degli elettrocardiogrammi (ECG) e risultati degli esami fisici.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 48
    Settimana 48
    E.5.2Secondary end point(s)
    • Clinical PNH symptom assessments including fatigue, dyspnea, chest pain/discomfort, difficulty swallowing (esophageal pain), abdominal pain, headache, erectile dysfunction, hemoglobinuria, jaundice, incidence of BTH, and incidence of thromboembolic events
    • Clinical measurements of PNH (LDH, hemoglobin, haptoglobin, reticulocytes, transfusion requirements)
    • PRO endpoints will include scores from the Quality of Life Questionnaire for Patients with Aplastic Anemia/PNH (QLQ-AA/PNH), Functional Assessment of Chronic Illness Therapy (FACIT) fatigue scale, and Treatment Satisfaction Questionnaire for Medication (TSQM).
    • Concentration of BCX9930 in plasma at steady state in subjects with PNH.
    • PD and complement biomarker measurements will include PNH red blood cell (RBC) and white blood cell (WBC) clone size, plasma Factor Bb levels and AP activity (as assessed via AP Hemolysis and AP Weislab)
    • Valutazioni cliniche dei sintomi della EPN, inclusi affaticamento, dispnea, dolore/disagio al torace, difficoltà a deglutire (dolore esofageo), dolore addominale, mal di testa, disfunzione erettile, emoglobinuria, ittero, incidenza della BTH ed incidenza di eventi tromboembolici
    • Misurazioni cliniche della EPN (LDH, emoglobina, aptoglobina, reticolociti, requisiti di trasfusione)
    • Gli endpoint dei PRO includeranno i punteggi del questionario sulla qualità della vita per i pazienti con anemia aplastica/EPN (QLQ-AA/EPN), della scala di affaticamento per la valutazione funzionale relativa al trattamento della patologia cronica (FACIT) e del Questionario di soddisfazione per il trattamento in materia di medicamenti (TSQM).
    • Concentrazione di BCX9930 nel plasma allo stato stazionario in soggetti con EPN.
    • Le misurazioni del PD e del biomarcatore del complemento includeranno le dimensioni dei cloni dei globuli rossi (RBC) e dei globuli bianchi (WBC), i livelli del fattore Bb nel plasma e l'attività AP (valutata tramite emolisi AP e AP Weislab).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 48
    Settimana 48
    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 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 Yes
    E.8.1.7.1Other trial design description
    In 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    European Union
    South Africa
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 180
    F.4.2.2In the whole clinical trial 200
    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 Decision2020-11-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-23
    P. End of Trial
    P.End of Trial StatusOngoing
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