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    Summary
    EudraCT Number:2020-003172-41
    Sponsor's Protocol Code Number:CLNP023C12301
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-04-15
    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 number2020-003172-41
    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
    Etude multicentrique, à un seul bras, en ouvert, évaluant l'efficacité et la sécurité d'emploi de l'iptacopan administré par voie orale deux fois par jour chez des patients adultes atteints d’hémoglobinurie paroxystique nocturne (HPN) naïfs de traitement par inhibiteur du complément
    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 iptacopan in Adult PNH Patients who are naïve to complement inhibitor therapy
    Etude de l'efficacité et de la sécurité d'emploi de l'iptacopan administré par voie orale deux fois par jour chez des patients adultes atteints d’hémoglobinurie paroxystique nocturne (HPN) naïfs de traitement par inhibiteur du complément
    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 Pharma SAS
    B.5.2Functional name of contact pointInformation&Communication Médicales
    B.5.3 Address:
    B.5.3.1Street Address8/10 rue Henry 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.3.9.3Other 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.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 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.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
    Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 2 g/dL in the absence of red blood cell transfusion
    Evaluer l'effet de l'iptacopan (LNP023) sur la proportion de patients traités par l’iptacopan ayant atteint une augmentation durable par rapport à la baseline des taux d'hémoglobine ≥ 2 g/dl en l'absence de transfusion de globules rouges
    E.2.2Secondary objectives of the trial
    - Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
    - Transfusion avoidance (TA) defined as the proportion of participants who remain free from transfusions
    - Change from baseline in hemoglobin
    - Percent change from baseline in LDH
    - Rate of breakthrough hemolysis (BTH)
    - Change from baseline in reticulocyte counts
    - Change in fatigue score, using the FACIT-Fatigue questionnaire
    - Rates of Major Adverse Vascular Events (MAVEs incl. thrombosis)
    - Evaluer l'effet de l'iptacopan sur la proportion de patients atteignant des taux d'hémoglobine durables ≥ 12 g/dl en absence de transfusions de globules rouges.
    - Evaluer l'effet de l'iptacopan sur l'évitement des transfusions défini comme la proportion de patients exempts de transfusions.
    - Evaluer l'effet de l'iptacopan sur la variation moyenne de l’hémoglobine
    - Evaluer l'effet de l'iptacopan sur le pourcentage moyen de variation du LDH
    - Evaluer l'effet de l'iptacopan sur le taux d’épisodes hémolytiques intercurrents
    - Evaluer l'effet de l'iptacopan sur la variation moyenne du nombre de réticulocytes
    - Evaluer l'effet de l'iptacopan sur l'amélioration de la fatigue, à l'aide du questionnaire FACIT-Fatigue
    - Evaluer les taux d'événements vasculaires majeurs (y compris thromboses)
    - Evaluer la sécurité d'emploi et la tolérance de l’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 WBCs clone size ≥ 10%
    - Mean hemoglobin level <10 g/dL • LDH > 1.5 x Upper Limit of Normal (ULN)
    - Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment
    - If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given

    Other protocol defined inclusion/exclusion criteria may apply.
    -Patients masculins et féminins ≥ 18 ans avec un diagnostic d’HPN confirmé par cytométrie de flux à haute sensibilité avec quantification de la taille du clone monocytaire ou granulocytaire des globules rouges et blancs ≥ 10%.
    -Taux d'hémoglobine moyen <10 g/dL confirmé par l'évaluation du laboratoire central pendant la sélection et avant de commencer le traitement
    -LDH > 1,5 x limite supérieure de la normale (LSN)
    -La vaccination contre l'infection par Neisseria meningitidis est requise avant le début du traitement par iptacopan.
    -Si elle n‘a pas été réalisée auparavant, la vaccination contre les infections à Streptococcus pneumoniae et Haemophilus influenzae doit être administrée,
    E.4Principal exclusion criteria
    - Prior treatment with a complement inhibitor, including anti-C5 antibody
    - Known or suspected hereditary complement deficiency
    - History of hematopoietic stem cell transplantation
    - Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L).
    - Active systemic bacterial, viral (incl. COVID-19)or fungal infection within 14 days prior to study drug administration
    - 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., NYHA class IV heart failure), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.

    Other protocol defined inclusion/exclusion criteria may apply.
    -Traitement antérieur par un inhibiteur du complément, y compris par anticorps anti-C5
    -Déficit héréditaire en complément connu ou suspecté
    -Antécédents de greffe de cellules souches hématopoïétiques
    -Signes biologiques d'aplasie médullaire (réticulocytes < 100x109/L, plaquettes < 30x109/L, neutrophiles < 0,5x109/L)
    -Infection systémique bactérienne, virale (incluant COVID-19) ou fongique active dans les 14 jours précédant l'administration du médicament à l'étude
    -Antécédents d'infections invasives récurrentes provoquées par des organismes encapsulés, par ex. méningocoque ou pneumocoque
    -Comorbidités concomitantes majeures, y compris, mais sans s'y limiter, une maladie rénale sévère (par exemple, dialyse), une maladie cardiaque sévère (par ex Insuffisance cardiaque, New York Heart Association-NYHA classe IV), une maladie pulmonaire sévère (par ex., hypertension pulmonaire sévère - classe IV de l'Organisation Mondiale de la Santé-OMS) ou une maladie hépatique (par ex., hépatite active) qui, de l'avis de l'investigateur, exclut la participation du patient à l'étude
    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 between Day 126 and Day 168, in the absence of packed red blood cell (pRBC) transfusions between Day 14 and Day 168
    Proportion de patients ayant obtenu une augmentation durable des taux d'hémoglobine par rapport à la baseline ≥ 2 g/dL entre le jour 126 et le jour 168 en l'absence de transfusion entre le jour 14 et le jour 168
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between Day 126 and Day 168
    Entre le jour 126 et le jour 168
    E.5.2Secondary end point(s)
    1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL between Day 126 and Day 168 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 between Day 126 and Day 168
    4. Percent change from baseline in LDH levels (U/L) as mean of visits between Day 126 and Day 168
    5. Occurrences of breakthrough hemolysis reported between Day 1 and Day 168
    6. Change from baseline in reticulocyte counts as mean of visits between Day 126 and Day 168
    7. Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168
    8. Occurrences of MAVEs occurring between Day 1 and Day 168
    1. Réponse définie par un taux d'hémoglobine ≥ 12 g/dL entre le jour 126 et le jour 168, en l'absence de transfusions de globules rouges entre le jour 14 et le jour 168.
    2. Absence d’administration de transfusions de culot globulaire (pRBC) entre le jour 14 et le jour 168.
    3. Variation par rapport à la baseline de l'hémoglobine (g/dL) moyenne aux visites entre le jour 126 et le jour 168.
    4. Pourcentage de variation par rapport à la baseline des taux de LDH (U/L) moyenne aux visites entre le jour 126 et le jour 168.
    5. Occurrence d’épisodes hémolytiques intercurrents entre le jour 1 et le jour 168.
    6. Variation par rapport à la baseline du nombre de réticulocytes (109/L) moyen aux visites entre le jour 126 et le jour 168.
    7. Variation par rapport à la baseline des scores FACIT-Fatigue moyens aux visites entre le jour 126 et le jour 168.
    8. Occurrence d’événements indésirables vasculaires majeurs (MAVE) survenant entre le jour 1 et le jour 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. Entre le jour 126 et le jour 168
    2. Entre le jour 14 et le jour 168
    3. Entre le jour 126 et le jour 168
    4. Entre le jour 126 et le jour 168
    5. Entre le premier jour et le jour 168
    6. Entre le jour 126 et le jour 168
    7. Entre le jour 126 et le jour 168
    8. Entre le premier jour and le jour 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 Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    tolerability
    tolérabilité
    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 concerned4
    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 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
    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
    La Dernière visite du dernièr patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial 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 state5
    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.
    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 Decision2021-06-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-06
    P. End of Trial
    P.End of Trial StatusCompleted
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