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    Summary
    EudraCT Number:2020-003172-41
    Sponsor's Protocol Code Number:CLNP023C12301
    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:2021-08-17
    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 number2020-003172-41
    A.3Full title of the trial
    A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy.
    Studio multicentrico, a singolo braccio, in aperto per valutare l’efficacia e la sicurezza di iptacopan per via orale due volte al giorno in pazienti adulti affetti da EPN mai trattati con inibitori del complemento.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients who are naïve to complement inhibitor therapy.
    Studio di efficacia e sicurezza di iptacopan orale due volte al giorno in pazienti adulti affetti da EPN mai trattati con inibitori del complemento.
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberCLNP023C12301
    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 SponsorNOVARTIS PHARMA AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNOVARTIS FARMA S.p.A
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1
    B.5.3.2Town/ cityORIGGIO (VA)
    B.5.3.3Post code21040
    B.5.3.4CountryItaly
    B.5.4Telephone number0296543289
    B.5.5Fax number029659066
    B.5.6E-mailinfo.studiclinici@novartis.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 Yes
    D.2.1.1.1Trade name HIBERIX - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE PER USO INTRAMUSCOLARE 1 FLACONCINO DI POLVERE + 1 SIRINGA PRERIEMPITA DI SOLVENTE DA 0.5 ML
    D.2.1.1.2Name of the Marketing Authorisation holderGLAXOSMITHKLINE S.P.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO HAEMOPHILUS INFLUENZAE B CONIUGATO CON TOSSOIDETETANICO
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 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.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 Yes
    D.2.1.1.1Trade name MENVEO – Polvere e soluzione per soluzione iniettabile
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxoSmithKline Vaccines s.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135)
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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.IMP: 3
    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 Yes
    D.2.1.1.1Trade name PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml
    D.2.1.1.2Name of the Marketing Authorisation holderMSD
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO PNEUMOCOCCICO
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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.IMP: 4
    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 Yes
    D.2.1.1.1Trade name BEXSERO – Sospensione iniettabile – Siringa preriempita
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxoSmithKline Vaccines s.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO CONTRO IL MEMINGOCOCCO DI GRUPPO B
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 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.IMP: 5
    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 Yes
    D.2.1.1.1Trade name ACT-HIB Polvere e solvente per soluzione iniettabile in siringa preriempita 0,5 ml
    D.2.1.1.2Name of the Marketing Authorisation holderSANOFI PASTEUR EUROPE
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHAEMOPHILUS INFLUENZAE
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.IMP: 6
    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/3/20/2281
    D.3 Description of the IMP
    D.3.1Product nameiptacopan
    D.3.2Product code [LNP023]
    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 INNiptacopan
    D.3.9.2Current sponsor codeLNP023
    D.3.9.3Other descriptive nameLNP023 Hydrochloride Salt
    D.3.9.4EV Substance CodeSUB180361
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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: 7
    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 Yes
    D.2.1.1.1Trade name Nimenrix
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Europe MA EEIG
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 name-
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135)
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit µl microlitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 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.IMP: 8
    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/3/20/2281
    D.3 Description of the IMP
    D.3.1Product nameiptacopan
    D.3.2Product code [LNP023]
    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 INNiptacopan
    D.3.9.2Current sponsor codeLNP023
    D.3.9.3Other descriptive nameLNP023 Hydrochloride Salt
    D.3.9.4EV Substance CodeSUB180361
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    Blood disorder.
    Malattia del sangue.
    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 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
    The primary objective is to assess the effect of iptacopan on proportion of participants treated with iptacopan achieving a sustained increase from baseline in hemoglobin levels of = 2 g/dL in the absence of red blood cell transfusion.
    L’obiettivo primario è valutare l’effetto di iptacopan sulla proporzione di soggetti partecipanti trattati con iptacopan che raggiungono un aumento sostenuto dei livelli di emoglobina rispetto al basale di = 2 g/dL in assenza di trasfusione di globuli rossi.
    E.2.2Secondary objectives of the trial
    - To assess the effect of iptacopan on the proportion of participants achieving sustained hemoglobin levels = 12 g/dL in the absence of red blood cell transfusions.
    - To assess the effect of iptacopan on transfusion avoidance (TA) defined as the proportion of participants who remain free from transfusions.
    - To assess the effect of iptacopan on average change in hemoglobin.
    - To assess the effect of iptacopan on average percent change in LDH.
    - To assess the effect of iptacopan on the rate of breakthrough hemolysis (BTH).
    - To assess the effect of iptacopan on average change in reticulocyte counts.
    - To assess the effect of iptacopan on improving fatigue, using the FACITFatigue questionnaire.
    - To assess the rates of Major Adverse Vascular Events (MAVEs incl. Thrombosis).
    - To assess safety and tolerability of iptacopan.
    - Valutare l’effetto di iptacopan sulla proporzione di soggetti che raggiungono livelli di emoglobina sostenuti = 12 g/dL in assenza di trasfusioni di globuli rossi.
    - Valutare l’effetto di iptacopan sull’evitare trasfusioni definito come la proporzione di partecipanti che non necessita trasfusioni.
    - Valutare l’effetto di iptacopan sul cambiamento medio di emoglobina
    - Valutare l’effetto di iptacopan sulla variazione percentuale media di LDH
    - Valutare l’effetto di iptacopan sul tasso di breakthrough emolisi (BTH)
    - Valutare l’effetto di iptacopan sul miglioramento della fatica, usando il questionario FACIT-Fatigue.
    - Valutare il tasso di Eventi Avversi Vascolari Maggiori (MAVEs compreso trombosi)
    - Valutare la sicurezza e la tollerabilità di iptacopan.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male and female participants = 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and white blood cells (WBCs) (granulocyte/monocyte) clone size = 10%.
    - Mean hemoglobin level <10 g/dL confirmed by central laboratory assessment during Screening and prior to starting study treatment:
    a. By two hemoglobin measurements (mean < 10 g/dL), two to eight weeks apart, for patients not receiving a RBC transfusion during Screening.
    b. By one hemoglobin measurement (<10 g/dL) carried at the first Screening visit for patients receiving a RBC transfusion after which he/she will be eligible.
    - LDH > 1.5 x Upper Limit of Normal (ULN) for at least two central laboratory measurements two to eight weeks apart during the screening period.
    - Vaccination against Neisseria meningitidis infection is required prior to the start of treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local
    regulations, at least 2 weeks prior to first dosing.
    - If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to first dosing.
    - Prima di qualsiasi valutazione si deve acquisire il consenso informato firmato.
    - Partecipanti di sesso maschile e femminile di età = 18 anni con una diagnosi di PNH confermata da citometria a flusso ad alta sensibilità con dimensione del clone di globuli rossi e globuli bianchi (granulociti / monociti) = 10%.
    - livello medio di emoglobina <10 g / dL confermato dalla misurazione del laboratorio centralizzato durante il periodo selezione (screening) e prima di iniziare il trattamento in studio:
    a. Con due misurazioni dell'emoglobina (media <10 g / dL), a distanza di due fino a otto settimane, per i pazienti che non ricevono una trasfusione di RBC durante lo screening.
    b. Mediante una misurazione dell'emoglobina (<10 g / dL) effettuata alla prima visita di selezione (screening) per i pazienti che ricevono una trasfusione di RBC, dopo essere stati considerati eleggibili.
    - LDH >1.5 ULN in almeno due misurazioni effettuate dal laboratorio centralizzato a distanza di due fino a otto settimane durante il periodo di selezione (screening).
    - La vaccinazione contro l'infezione da Neisseria meningitidis è necessaria prima dell'inizio del trattamento con iptacopan. Se il paziente non è stato vaccinato in precedenza, o se è necessario un richiamo, il vaccino deve essere somministrato, se disponibile e secondo le normative locali, almeno 2 settimane prima della prima somministrazione di iptacopan. Se il trattamento con iptacopan deve iniziare prima di 2 settimane dopo la vaccinazione, deve essere iniziato un trattamento antibiotico profilattico.
    - Se non è stato ricevuto in precedenza, deve essere somministrata la vaccinazione contro le infezioni da Streptococcus pneumoniae e Haemophilus influenzae, se disponibile e secondo le normative locali. I vaccini devono essere somministrati almeno 2 settimane prima della prima somministrazione di iptacopan. Se il trattamento con iptacopan deve iniziare prima di 2 settimane dopo la vaccinazione, deve essere iniziato un trattamento antibiotico profilattico.
    - Capace di comunicare bene con lo sperimentatore, di comprendere e rispettare i requisiti dello studio.
    E.4Principal exclusion criteria
    - Prior treatment with a complement inhibitor, including anti-C5 antibody.
    - Known or suspected hereditary complement deficiency at screening.
    - History of hematopoietic stem cell transplantation.
    - Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <500x106/L).
    - Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration.
    - A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
    - Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., New York Heart Association (NYHA) class IV), severe pulmonary disease (e.g., severe
    pulmonary hypertension (World Health Organization (WHO) class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.
    - Trattamento precedente con inibitori del complemento, compresi anticorpi anti-C5.
    - Deficit ereditario del complemento noto o sospetto
    - Storia di trapianto di cellule staminali ematopoietiche
    - Pazienti con evidenza testata dal laboratorio di insufficienza midollare (reticolociti <100x109/L, piastrine <30x109/L, neutrofili <0,5x109/L)
    - Infezione batterica, virale (incluso COVID-19) o fungina sistemica attiva entro 14 giorni prima della somministrazione del farmaco in studio.
    - Storia di infezioni invasive ricorrenti causate da organismi incapsulati, ad es. meningococco o pneumococco
    - Principali comorbidità concomitanti incluse, ma non limitate a, grave malattia renale (p. es., Dialisi), malattia cardiaca avanzata (p. es., insufficienza cardiaca Classe NYHA IV), malattia polmonare grave (p.es., Ipertensione polmonare grave (classe IV OMS)) o malattia epatica (p. es., epatite attiva) che a giudizio dello sperimentatore preclude la partecipazione del paziente allo studio.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of = 2 g/dL assessed, in the absence of packed red blood cell (pRBC) transfusions between Day 14 and Day 168.
    Proporzione di partecipanti che raggiungono un aumento sostenuto dal basale nei livelli di emoglobina di = 2 g/dl valutati, in assenza di trasfusione tra il giorno 14 e il giorno 168.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between Day 126 and Day 168.
    Tra il giorno 126 e il giorno 168.
    E.5.2Secondary end point(s)
    1. Proportion of participants achieving sustained hemoglobin levels = 12 g/dL in absence of red blood cell transfusion between Day 14 and Day 168
    2. Transfusion avoidance (TA) defined as the proportion of participants who remain free from transfusions
    3. Change from baseline in hemoglobin (g/dL) as mean of visits
    4. Percent change from baseline in LDH levels (U/L) as mean of visits
    5. Occurrences of breakthrough hemolysis
    6. Change from baseline in reticulocyte counts as mean of visits
    7. Change from baseline in FACIT-Fatigue scores as mean of visits
    8. Occurrences of MAVEs occurring
    1. Proporzioni di partecipanti che raggiungono livelli sostenuti di emoglobina = 12 g/dl in assenza di trasfusione di globuli rossi trasfusione di globuli rossi tra il giorno 14 e il giorno 168
    2. Proporzione di partecipanti che rimangono liberi da trasfusioni
    3. Cambiamento dal basale dell'emoglobina (g/dl) come media delle visite
    4. Variazione percentuale dal basale dei livelli di LDH (U/L) come media delle visite
    5. Eventi di emolisi da rottura
    6. Cambiamento dal basale nella conta dei reticolociti come media delle visite
    7. Cambiamento dal basale nei punteggi FACIT-Fatigue come media delle visite
    8. Occorrenze di MAVE che si verificano tra il giorno 1 e il giorno 168
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Between Day 126 and Day 168
    2. Between Day 14 and Day 168
    3. Between Day 126 and Day 168
    4. Between Day 126 and Day 168
    5. Between Day 1 and Day 168
    6. Between Day 126 and Day 168
    7. Between Day 126 and Day 168
    8. Between Day 1 and Day 168
    1. Tra il giorno 126 e il giorno 168
    2. Tra il giorno 14 e il giorno 168
    3. Tra il giorno 126 e il giorno 168
    4. Tra il giorno 126 e il giorno 168
    5. Tra il giorno 1 e il giorno 168
    6. Tra il giorno 126 e il giorno 168
    7. Tra il giorno 126 e il giorno 168
    8. Tra il giorno 1 e il giorno 168
    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
    Tollerabilità
    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 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 concerned3
    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
    China
    Japan
    Korea, Republic of
    Malaysia
    Singapore
    United States
    France
    Germany
    Italy
    United Kingdom
    Czechia
    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 years0
    E.8.9.1In the Member State concerned months24
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months27
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 40
    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 can receive post-trial access by joining the roll-over extension program to allow participants' access to LNP023 and to enable long-term safety monitoring.
    Participants can receive post-trial access by joining the roll-over extension program to allow participants' access to LNP023 and to enable long-term safety monitoring.
    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 Decision2021-07-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-19
    P. End of Trial
    P.End of Trial StatusOngoing
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