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    Summary
    EudraCT Number:2020-005186-13
    Sponsor's Protocol Code Number:CLNP023F12301
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-05-27
    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 ConcernedAustria - BASG
    A.2EudraCT number2020-005186-13
    A.3Full title of the trial
    A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of iptacopan (LNP023) in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor therapy
    A.4.1Sponsor's protocol code numberCLNP023F12301
    A.5.4Other Identifiers
    Name:EU CT NumberNumber:2023-508840-22
    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 GmbH
    B.5.2Functional name of contact pointDrug Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressJakov-Lind-Straße 5 / Top 3.05
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1020
    B.5.3.4CountryAustria
    B.5.4Telephone number+43 1 86657 0
    B.5.5Fax number +43 1 86657 6458
    B.5.6E-mailaustria.dra@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 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.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
    atypical hemolytic uremic syndrome
    E.1.1.1Medical condition in easily understood language
    atypical hemolytic uremic syndrome
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10079841
    E.1.2Term Atypical hemolytic uremic syndrome
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - Percentage of participants with complete TMA response without the use of PE/PI and anti-C5 antibody
    - Long term safety and efficacy evaluations
    E.2.2Secondary objectives of the trial
    - Time to achieve complete TMA response
    - Percentage of participants with increase from baseline in hemoglobin levels ≥ 2 g/dL
    - Change from baseline in hematologic parameters
    - Percentage of participants on dialysis
    - Change from baseline on estimated glomerular filtration rate
    - Change from baseline in chronic kidney disease (CKD) stage
    - Change from baseline in patient-reported outcomes score
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    - Biomarker substudy (to measure biomarkers or proteins related to complement pathways, aHUS, or iptacopan mechanism of action)

    - Pharmacokinetic substudy (to characterize iptacopan exposure in aHUS patients)
    E.3Principal inclusion criteria
    • Male and female patients ≥ 18 years of age with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and kidney injury, based on the following
    laboratory findings:
    - Platelet count <150x109/L during the Screening Period, and
    - LDH =1.5 x upper limit of normal (ULN) during the Screening Period and hemoglobin = lower limit of normal (LLN) for age and gender during the Screening Period, and
    - Serum creatinine =ULN during the Screening Period with acute worsening of kidney function. (Patients requiring dialysis for acute kidney injury are eligible)
    • Vaccination against Neisseria meningitidis and Streptococcus
    pneumoniae infections is required prior to the start of study 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 study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination
    • If not received previously, vaccination against Haemophilus influenzae infection should be given if available and according to local regulations. The vaccine should be given at least 2 weeks prior to first study drug administration.
    • Among patients with a kidney transplant, (a) known history of aHUS prior to current kidney transplantation, or (b) If no history of aHUS prior to the current transplantation is available, patients may be eligible
    without changes of immunosuppressive regimen if investigator excludes other causes of TMA not attributable to aHUS, especially transplant rejection. If immunosuppressive regimens (e.g., calcineurin inhibitor
    [CNI] or mammalian target of rapamycin inhibitor [mTORi] need to be modified after transplantation, evidence that TMA has persisted for at least 4 days after modification needs to be available.
    E.4Principal exclusion criteria
    • Previous or ongoing treatment with complement inhibitors, including anti-C5 antibody
    • A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or Positive direct Coombs test
    • Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS
    • Started receiving PE/PI 14 days or longer before the start of screening visit for the current TMA
    • Bone marrow transplantation (BMT)/hematopoietic stem cell
    transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
    • In patients with a kidney transplant, acute kidney dysfunction
    consistent with the diagnosis of transplantation failure due to
    acute/chronic active T-Cell mediated rejection (TCMR) and/or
    active/chronic active antibody-mediated rejection (ABMR) according to Banff 2017 criteria
    • Among patients with native kidney, history or presence of any kidney disease other than aHUS, such as:
    - Known kidney biopsy finding suggestive of underlying disease other than aHUS
    - Kidney ultrasound finding demonstrating small kidneys suggestive of chronic kidney failure
    - Known family history and/or genetic diagnosis of non-complement mediated genetic kidney disease (eg, focal segmental glomerulosclerosis)
    - Laboratory tests indicative of a kidney disease other than aHUS (eg, Anti-glomerular Basement Membrane (anti-GBM) antibodies, Anti- Neutrophil Cytoplasmic Antibodies (ANCA), Anti-Phospholipase A2 Receptor (anti-PLA2R) antibodies, anti-double stranded DNA (antidsDNA) antibodies)
    - Liver disease or liver injury at screening
    - - Patients with sepsis or active severe systemic bacterial, viral (including COVID-19) or fungal infection
    -Presence of systemic infections (bacterial, viral, fungal or parasitic) that, in the opinion of the Investigator, confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease
    • Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e. meningococcus, pneumococcus), or H. influenzae
    • Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
    • Chronic hemo- or peritoneal dialysis
    • Any adenoviral COVID-19 vaccine within 28 days prior to screening visit
    E.5 End points
    E.5.1Primary end point(s)
    - The number/percentage of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment. Complete TMA Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 10^9/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline), maintained for two measurements obtained at least four weeks apart, and any measurement in between
    - Long term (one year) safety, tolerability and efficacy of iptacopan via 1) safety evaluations including adverse events/serious adverse events, safety laboratory parameters, vital signs etc. after 52 weeks of study treatment, and 2) efficacy evaluations including complete TMA response, hematological parameters (platelets, LDH, hemoglobin), eGFR, PROs after 52 weeks of study treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    - 26 weeks of study treatment
    - 52 weeks of study treatment
    E.5.2Secondary end point(s)
    - Effect of study treatment iptacopan on time to complete TMA response during the first 26 weeks of study treatment
    - Response is defined as the percentage of participants with an increase in hemoglobin of ≥ 2g/dL from baseline, observed at two measurements obtained at least 4 weeks apart and any measurement in between during 26 weeks of study treatment
    - Change from baseline in hematologic parameters (platelets, LDH, hemoglobin) at week 26
    - For participants requiring dialysis withing 5 days prior to iptacopan treatment initiation, the number of participants who no longer require dialysis through 26 weeks of study treatment will be evaluated by means of proportion and corresponding confidence interval
    - Change from baseline in eGFR after 26 weeks of study treatment
    - Change from baseline in CDK stage (1-5) based on eGFR categories at week 26
    - Change from baseline in patient-reported outcomes scores for FACIT-Fatigue, Patient Global Impression of Severity (PGIS), EuroQol 5-level EQ-5D version (EQ-5D-5L) and Short-form 36 health survey questionnaire version 2 (SF-36 v2) at week 26
    E.5.2.1Timepoint(s) of evaluation of this end point
    - 26 weeks of study treatment
    - 26 weeks of study treatment
    - At week 26
    - 26 weeks of study treatment
    - At week 26
    - At week 26
    - At week 26
    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 No
    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
    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 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 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
    Taiwan
    Brazil
    China
    India
    Japan
    Korea, Republic of
    Russian Federation
    United Kingdom
    United States
    Austria
    Czechia
    Greece
    Hungary
    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
    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 months1
    E.8.9.1In the Member State concerned days6
    E.8.9.2In all countries concerned by the trial years3
    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: 45
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 50
    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 who complete the study may be eligible to enroll in a single-arm open-label iptacopan rollover extension program (REP). Participants who prematurely withdraw from the study for any reason are not eligible to enroll in the REP.
    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 Decision2021-07-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-26
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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