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    The EU Clinical Trials Register currently displays   43227   clinical trials with a EudraCT protocol, of which   7153   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2020-004385-19
    Sponsor's Protocol Code Number:CLNP023C12001B
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-15
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-004385-19
    A.3Full title of the trial
    An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.
    Etude d’extension en ouvert, multicentrique, ayant pour but de caractériser la tolérance et la sécurité d’emploi à long terme de l’iptacopan (LNP023) chez des patients atteints d’hémoglobinurie paroxystique nocturne (HPN) ayant terminé des études de phase II et III évaluant l’iptacopan
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal Hemoglobinuria
    Sécurité et tolérance à long terme de l'iptacopan chez les patients atteints d’hémoglobinurie paroxystique nocturne (HPN)
    A.4.1Sponsor's protocol code numberCLNP023C12001B
    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 Pharma S.A.S
    B.5.2Functional name of contact pointInformation&Communication Médicales
    B.5.3 Address:
    B.5.3.1Street Address8/10 rue Henri Sainte Claire Deville, CS 40150
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92563
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 1 5547 6600
    B.5.5Fax number+33 1 5547 6100
    B.5.6E-mailicm.phfr@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 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 name LNP023 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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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/3/20/2281
    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 name LNP023 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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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)
    Hémoglobinurie paroxystique nocturne (HPN)
    E.1.1.1Medical condition in easily understood language
    Blood disorder
    Maladie du sang
    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
    Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
    Évaluations de la sécurité, y compris, mais sans s'y limiter, les événements indésirables/événements indésirables graves, les paramètres de laboratoire de sécurité, les signes vitaux, etc. jusqu'à la visite de fin d'étude
    E.2.2Secondary objectives of the trial
    1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
    2. Proportion of participants who remain free from transfusions evaluated over yearly intervals
    3. Rate of breakthrough hemolysis (BTH)
    4. Proportion of participants with Major Adverse Vascular Events MAVEs (incl. thrombosis) evaluated over yearly intervals
    1. Proportion de participants ayant un maintien durable de l’hémoglobine à un taux ≥ 12 g/dl sans recours à des transfusions de globules rouges
    2. Proportion de patients ne nécessitant pas de transfusions (évaluation annuelle)
    3. Taux de poussées d'hémolyse
    4. Proportion de participants présentant des événements vasculaires indésirables majeurs (y compris thrombose) évalués à des intervalles annuels
    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 who have completed Phase 2 or Phase 3 clinical studies
    - Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
    - Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months
    - Patients de sexe masculin ou féminin âgés de ≥ 18 ans, diagnostiqués avec une HPN, ayant terminé les études cliniques de phase 2 ou de phase 3
    - Les vaccinations antérieures contre Neisseria meningitidis, Streptococcus pneumoniae et Haemophilus influenzae doivent être à jour
    - Patients cliniquement stables sous traitement par LNP023 en monothérapie depuis au moins 3 mois et pour lesquels la poursuite de ce traitement pourrait être bénéfique selon l’avis du médecin-investigateur
    E.4Principal exclusion criteria
    - Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason
    - Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
    - History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
    - History of hematopoietic stem cell transplantation
    Other protocol-defined inclusion/exclusion criteria may apply.
    - Patients n’ayant pas rempli les critères de sélection ou de la baseline dans les études parentes dans l’HPN ou ayant prématurément arrêté l’étude quelle qu’en ait été la raison
    - Toute comorbidité ou pathologie (incluant entre autres : infection bactérienne, virale ou fongique systémique active ou cancer) qui pourrait accroître le risque pour le patient ou potentiellement influencer les données de l’étude selon le médecin-investigateur
    - Antécédents d’infections invasives récurrentes dues à des organismes encapsulés tels que Neisseria meningitidis, Streptococcus pneumoniae ou Haemophilus influenzae
    - Antécédents de greffe de cellules souches hématopoïétiques
    D'autres critères d'inclusion/exclusion définis par le protocole peuvent s'appliquer.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants with adverse events
    Proportion de participants présentant des événements indésirables






    E.5.1.1Timepoint(s) of evaluation of this end point
    Time Frame: 60 months
    Délai : 60 mois
    E.5.2Secondary end point(s)
    1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
    2. Proportion of participants who remain free from transfusions
    3. Rate of breakthrough hemolysis (BTH)
    4. Proportion of participants with Major Adverse Vascular Events MAVEs
    1. Proportion de participants ayant un maintien durable de l’hémoglobine à un taux ≥ 12 g/dl sans recours à des transfusions de globules rouges
    2. Proportion de patients ne nécessitant pas de transfusions
    3. Taux de poussées d'hémolyse
    4. Proportion de participants présentant des événements vasculaires indésirables majeurs
    E.5.2.1Timepoint(s) of evaluation of this end point
    Time Frame: 60 months
    Délai : 60 mois
    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 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) 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 Yes
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA41
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Brazil
    China
    Japan
    Korea, Republic of
    Malaysia
    Singapore
    Taiwan
    United States
    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
    Dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    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: 157
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 76
    F.4.2.2In the whole clinical trial 165
    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 will switch to commercial drug as study will end when the drug is commercially available
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-31
    P. End of Trial
    P.End of Trial StatusOngoing
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