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    EudraCT Number:2021-002039-40
    Sponsor's Protocol Code Number:CLNP023L12201
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-09-14
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2021-002039-40
    A.3Full title of the trial
    An open-label, multi-center, phase 2 basket study to assess efficacy, safety and pharmacokinetics of iptacopan (LNP023) in participants with autoimmune benign hematological disorders
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Basket study to assess efficacy, safety and PK of iptacopan (LNP023) in autoimmune benign hematological disorders
    A.4.1Sponsor's protocol code numberCLNP023L12201
    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.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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma GmbH
    B.5.2Functional name of contact pointMedizinischer Infoservice (MCC)
    B.5.3 Address:
    B.5.3.1Street AddressRoonstrasse 25
    B.5.3.2Town/ cityNurnberg
    B.5.3.3Post code90429
    B.5.4Telephone number+49 911 27312100
    B.5.5Fax number+49 911 27312160
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product 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 nameIptacopan hydrochloride
    D.3.9.4EV Substance CodeSUB216376
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Immune thrombocytopenia (ITP)
    Cold agglutinin disease (CAD)
    E.1.1.1Medical condition in easily understood language
    Immune thrombocytopenia (ITP)
    Cold agglutinin disease (CAD)
    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 23.0
    E.1.2Level PT
    E.1.2Classification code 10083842
    E.1.2Term Immune thrombocytopenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10068863
    E.1.2Term Cold agglutinin disease
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Cohort 1: To assess the ability of iptacopan to induce a clinically meaningful increase in platelet count in participants with primary ITP

    Cohort 2: To assess the ability of iptacopan to induce a clinically meaningful increase in hemoglobin levels in participants with primary CAD
    E.2.2Secondary objectives of the trial
    • To assess the time to first response
    • To assess the duration of response during Part A
    • To assess the magnitude of response during Part A
    • To assess the need for rescue therapy during Part A
    • Cohort 2: To assess the effect of iptacopan on relevant disease biomarkers (BM) not covered in the primary objective during Part A
    • To assess the safety and tolerability of iptacopan in participants with benign hematological disorders
    • To assess the pharmacokinetics of iptacopan in the respective target
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All Cohorts: • Written informed consent • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required and vaccination against Haemophilus influenzae infection is recommended prior to the start of treatment. • weight of at least 35kg

    Cohort 1 specific inclusion criteria: •Male and female participants aged ≥18 years at baseline with a diagnosis of persistent or chronic primary ITP (diagnosed at least 3 months prior to baseline). • Participants must have received at least 1 unique prior therapy administered with the intention to treat ITP. • Sustained thrombocytopenia

    Cohort 2 specific inclusion criteria: • Male and female participants aged ≥18 years at baseline with a diagnosis of primary CAD, including CAD arising in the setting of a low-grade lymphoproliferative disorder a) not requiring any therapy and b) without evidence of significant
    splenomegaly, hepatomegaly, or diffuse lymphadenopathy. • Positive
    direct antiglobulin test for C3d only (or predominantly) and cold
    agglutinin titer of ≥64 at 4°C. • Laboratory evidence of ongoing
    hemolysis. • Sustained anemia • Participants must have received at least 1 unique prior therapy administered with the intention to treat CAD
    E.4Principal exclusion criteria
    All cohorts: • Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations • Past or concomitant use of medications prohibited by the protocol • Known or suspected hereditary or acquired complement deficiency • History of primary or secondary immunodeficiency, including a positive HIV test result • Chronic infection with Hepatitis B or C virus • History of recurrent invasive infections caused by encapsulated organisms, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae • Presence or suspicion of any active infection within 14 days prior to first study drug administration. • Any medical condition deemed likely to interfere with the participant’s participation in the study • Any malignant disease diagnosed within the past 5 years, with the exception of localized non-melanoma skin cancer, in situ cervical cancer, or, for CAD, a low-grade lymphoproliferative disorder. • History of bone marrow/hematopoietic stem cell or solid organ transplantation. • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after last iptacopan dose. Active severe bleeding or history of intracranial hemorrhage. • Liver disease, or liver injury as indicated by abnormal liver function tests. • Severe concurrent co-morbidities of unstable medical conditions.

    Cohort 1 specific exclusion criteria: • Secondary ITP, as may arise in the setting of certain autoimmune disorders, immunodeficiency syndromes, infections, malignancies, and drug treatments • No ITP-directed
    background therapy permitted, with the exception of either a thrombopoietin receptor agonist (TPO-RA) or low-dose corticosteroid, as long as stable dosage for at least 4 weeks prior to first iptacopan dose. • Abnormal coagulation screening labs.

    Cohort 2 specific exclusion criteria: • Secondary cold agglutinin syndrome, as may arise in the setting of certain infections, autoimmune disorders, and malignancies (with the exception of a low-grade lymphoproliferative disorder) • No CAD-directed background therapy permitted
    E.5 End points
    E.5.1Primary end point(s)
    • Cohort 1 (ITP): A clinically meaningful response, defined by a platelet count of ≥50 k/µL sustained for at least 2 consecutive weeks during the main, 12-week treatment part without the use of rescue therapy
    • Cohort 2 (CAD): A clinically meaningful response, defined by a hemoglobin level increase of ≥1.5 g/dL above baseline sustained for at least 2 consecutive weeks during the main, 12-week treatment part without the use of rescue therapy
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1 to Day 85
    E.5.2Secondary end point(s)
    • Cohort 1: Time to first platelet count ≥50 k/μL
    • Cohort 2: Time to first hemoglobin level ≥1.5 g/dL above baseline
    • Cohort 1: Duration during which platelet count remains ≥50k/μL without the use of rescue therapy
    • Cohort 2: Duration during which hemoglobin level remains ≥1.5 g/dL above baseline without the use of rescue therapy
    • Cohort 1: Magnitude of platelet count increase from baseline
    • Cohort 2: Magnitude of hemoglobin increase from baseline
    • Use of rescue therapy
    • Lactate dehydrogenase (LDH), total bilirubin, reticulocyte count and haptoglobin
    • Safety parameters include vital signs, adverse events, hematology, blood chemistry, reproductive and thyroid hormones, coagulation, urinalysis and ECG evaluation.
    • Iptacopan PK parameters including but not limited to Cmax, AUCtau, AUClast, Ctrough and Tmax.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Platelet count, Hemoglobin levels, Number of patients who use rescue therapy: Day 1 to Day 85

    • Lactate dehydrogenase (LDH): Screening, Day 15, Day 29, Day 85, Day 113

    • Total billirubin: Screening, Day 15, Day 29, Day 85, Day 113

    • Reticulocytes count: Screening, Baseline, Day1, Day 15, Day 29, Day 85, Day 99, Day 113

    • Haptoglobin: Screening, Day 15, Day 29, Day 85, Day 113

    • Pharmacokinetic parameter: Day 15, Day 29, Day 57
    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
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    Korea, Republic of
    United Kingdom
    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
    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 months10
    E.8.9.1In the Member State concerned days13
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    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: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    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 20
    F.4.2.2In the whole clinical trial 30
    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)
    Continuing care should be provided by the investigator and/or referring physician after the EOS visit as per local standard of care
    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 Decision2021-10-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-29
    P. End of Trial
    P.End of Trial StatusOngoing
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