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    Summary
    EudraCT Number:2021-004253-22
    Sponsor's Protocol Code Number:ALXN2040-PNH-303
    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:2022-06-09
    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 number2021-004253-22
    A.3Full title of the trial
    A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study
    Studio di estensione a lungo termine (LTE) per caratterizzare la sicurezza e l’efficacia di danicopan come terapia aggiuntiva a un inibitore del componente 5 del complemento (C5i) in pazienti affetti da emoglobinuria parossistica notturna (EPN) precedentemente trattati con danicopan in uno studio clinico sponsorizzato da Alexion
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to C5i in Patients with PNH
    Uno studio di sicurezza ed efficacia a lungo termine di Danicopan come terapia aggiuntiva a C5i in pazienti con EPN.
    A.3.2Name or abbreviated title of the trial where available
    .
    .
    A.4.1Sponsor's protocol code numberALXN2040-PNH-303
    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 SponsorALEXION PHARMACEUTICALS INCORPORATED
    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 supportAlexion Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 Rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code2300
    B.5.3.4CountryFrance
    B.5.4Telephone number0033787148158
    B.5.5Fax number0033147100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1946
    D.3 Description of the IMP
    D.3.1Product nameDanicopan
    D.3.2Product code [ALXN2040]
    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 INNDANICOPAN
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeALXN2040
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 2
    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/3/17/1946
    D.3 Description of the IMP
    D.3.1Product nameDanicopan
    D.3.2Product code [ALXN2040]
    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 INNDANICOPAN
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeALXN2040
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Paroxysmal Nocturnal Hemoglobinuria
    Pazienti con emoglobinuria parossistica notturna
    E.1.1.1Medical condition in easily understood language
    Patients with Paroxysmal Nocturnal Hemoglobinuria
    Pazienti con emoglobinuria parossistica notturna
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10034042
    E.1.2Term Paroxysmal nocturnal haemoglobinuria
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To characterize the long-term safety of treatment with danicopan as an
    add-on therapy to a complement component 5 inhibitor (C5i).
    Caratterizzare la sicurezza a lungo termine del trattamento con danicopan come terapia aggiuntiva a un inibitore del componente 5 del complemento (C5i).
    E.2.2Secondary objectives of the trial
    -To characterize the long-term efficacy of danicopan as an add-on therapy to a C5i
    -To characterize the long-term effect of treatment with danicopan as an add-on therapy
    to a C5i on Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue scores
    and on European Organisation for Research and Treatment of Cancer Quality-of-life
    Questionnaire (EORTCQLQ- C30) scores
    -To further characterize the safety of danicopan as an add-on therapy to
    a C5i
    Caratterizzare l’efficacia a lungo termine di danicopan come terapia aggiuntiva a un C5i - Caratterizzare l’effetto a lungo termine del trattamento con danicopan come terapia aggiuntiva a un C5i, in base ai punteggi dell’affaticamento secondo la valutazione funzionale della terapia di una malattia cronica (FACIT) e in base ai punteggi del Questionario sulla qualità della vita composto da 30 domande ideato dall’Organizzazione europea per la ricerca e il trattamento del cancro (EORTCQLQ-C30)
    -Caratterizzare ulteriormente la sicurezza di danicopan come terapia aggiuntiva a un C5i
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment. - Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.
    - Tutti i partecipanti che hanno completato la loro partecipazione a uno studio clinico sponsorizzato da Alexion con danicopan come aggiunta a un trattamento C5i. - Documentazione di vaccinazione contro la Neisseria meningitidis: tutti i partecipanti devono essere rivaccinati in base alle linee guida nazionali sulle vaccinazioni o alla prassi locale sull’uso della vaccinazione con inibitori del complemento.
    E.4Principal exclusion criteria
    - Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant. - Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
    - Qualsiasi condizione medica (ad es. cardiaca, polmonare, renale, oncologica o psichiatrica) che, a giudizio dello sperimentatore, potrebbe interferire con la partecipazione allo studio, comportare un qualsiasi rischio aggiuntivo per il partecipante o confondere la valutazione del partecipante. - Partecipanti di sesso femminile in gravidanza, in allattamento o che intendono concepire nel corso dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
    • Incidenza degli eventi avversi emergenti dal trattamento (TEAE) e dei TEAE seri
    E.5.1.1Timepoint(s) of evaluation of this end point
    Over time throughout the study. Planned time points for all safety assessments are provided in the SoA (Section 1.3 of the protocol)
    Nel tempo, per tutta la durata dello studio. I punti temporali programmati per tutte le valutazioni di sicurezza sono forniti nel Programma delle attività (SoA) (Sezione 1.3 del protocollo)
    E.5.2Secondary end point(s)
    • Change in hemoglobin (Hgb) values over time
    • Change in absolute reticulocyte count over time
    • Change in lactate dehydrogenase (LDH) over time
    • Proportion of patients with LDH minor/equal 1.5 × upper limit of normal (ULN) over
    time
    • Proportion of patients with transfusion avoidance (TA), defined as
    patients who remain transfusion free and do not require transfusion as
    per protocol specified guidelines
    • Change in FACIT Fatigue scores over time
    • Change in EORTC-QLQ-C30 scores over time
    • Change in safety laboratory parameters over time
    • TEAEs leading to discontinuation; • Change in hemoglobin (Hgb) values over time
    • Change in absolute reticulocyte count over time
    • Change in lactate dehydrogenase (LDH) over time
    • Proportion of patients with LDH = 1.5 × upper limit of normal (ULN)
    over time
    • Proportion of patients with transfusion avoidance (TA), defined as
    patients who remain transfusion free and do not require transfusion as
    per protocol specified guidelines
    • Change in FACIT Fatigue scores over time
    • Change in EORTC-QLQ-C30 scores over time
    • Change in safety laboratory parameters over time
    • TEAEs leading to discontinuation
    • Variazione nei valori dell’emoglobina (Hgb) nel tempo
    • Variazione nella conta assoluta dei reticolociti nel tempo
    • Variazione della lattato deidrogenasi (LDH) nel tempo
    • Percentuale di pazienti con LDH minor/equal 1,5 × limite superiore della norma (ULN) nel tempo
    • Percentuale di pazienti con non-ricorso a trasfusioni (transfusion avoidance, TA), definiti come pazienti che non ricevono alcuna trasfusione e non necessitano di trasfusioni secondo le linee guida specificate nel protocollo
    • Variazione nei punteggi FACIT per l’affaticamento nel tempo
    • Variazione nei punteggi EORTC-QLQ-C30 nel tempo
    • Variazione nei parametri di laboratorio di sicurezza nel tempo
    • TEAE che determinano l’interruzione; • Variazione nei valori dell’emoglobina (Hgb) nel tempo
    • Variazione nella conta assoluta dei reticolociti nel tempo
    • Variazione della lattato deidrogenasi (LDH) nel tempo
    • Percentuale di pazienti con LDH =1,5 × limite superiore della norma (ULN) nel tempo
    • Percentuale di pazienti con non-ricorso a trasfusioni (transfusion avoidance, TA), definiti come pazienti che non ricevono alcuna trasfusione e non necessitano di trasfusioni secondo le linee guida specificate nel protocollo
    • Variazione nei punteggi FACIT per l’affaticamento nel tempo
    • Variazione nei punteggi EORTC-QLQ-C30 nel tempo
    • Variazione nei parametri di laboratorio di sicurezza nel tempo
    • TEAE che determinano l’interruzione
    E.5.2.1Timepoint(s) of evaluation of this end point
    Over time throughout the study. Planned time points for all safety assessments are provided in the SoA (Section 1.3 of the protocol); Over time throughout the study. Planned time points for all safety assessments are provided in the SoA (Section 1.3 of the protocol)
    Nel tempo, per tutta la durata dello studio. I punti temporali programmati per tutte le valutazioni di sicurezza sono forniti nel Programma delle attività (SoA) (Sezione 1.3 del protocollo); Nel tempo, per tutta la durata dello studio. I punti temporali programmati per tutte le valutazioni di sicurezza sono forniti nel Programma delle attività (SoA) (Sezione 1.3 del protocollo)
    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 Yes
    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) 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; Placebo. - Alcuni pazienti che effettuano il roll-over potrebbero ricevere il placebo in fa
    Open; Placebo. - Some patients rolling over may receive placebo at the beginning to maintain the do
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    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
    Argentina
    Brazil
    Canada
    Israel
    Japan
    Korea, Republic of
    Malaysia
    Taiwan
    Thailand
    United States
    France
    Poland
    Netherlands
    Spain
    Czechia
    Germany
    Greece
    Italy
    Turkey
    United Kingdom
    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 years3
    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 years3
    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: 93
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 60
    F.4.2.2In the whole clinical trial 95
    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)
    The protocol does not define any post-study treatment plan. Patients can continue their C5 inhibitor-based therapy based on investigator's decision. Patients completing the study may receive commercial danicopan once available in their country.
    Il protocollo non definisce alcun piano di trattamento post-studio. I pazienti possono continuare la loro terapia a base di inibitori C5 in base alla decisione dello sperimentatore. I pazienti che completano lo studio potranno ricevere la versione commerciale di danicopan quando sarà disponibile nel loro Paese.
    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 Decision2022-08-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-13
    P. End of Trial
    P.End of Trial StatusOngoing
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    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
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