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    Summary
    EudraCT Number:2014-005338-74
    Sponsor's Protocol Code Number:CLFG316X2201
    National Competent Authority:Lithuania - SMCA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-06-09
    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 ConcernedLithuania - SMCA
    A.2EudraCT number2014-005338-74
    A.3Full title of the trial
    An open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH)
    Atviras koncepcijos patvirtinimo tyrimas prieš C5 veikiančio monokloninio antikūno LFG316 veiksmingumui, saugumui ir farmakokinetikai įvertinti, skiriant pacientams, kuriems yra paroksizminė naktinė hemoglobinurija (PNH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNH
    A.3.2Name or abbreviated title of the trial where available
    CLFG316X2201
    A.4.1Sponsor's protocol code numberCLFG316X2201
    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 Services 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 Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSIA Novartis Baltics Lithuanian Branch
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressKonstitucijos pr. 7
    B.5.3.2Town/ cityVilnius
    B.5.3.3Post codeLT-09308
    B.5.3.4CountryLithuania
    B.5.4Telephone number+370 5 269 1650
    B.5.5Fax number-
    B.5.6E-mailDRA.Lithuania@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.3.2Product code LFG316
    D.3.4Pharmaceutical form Lyophilisate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNo
    D.3.9.2Current sponsor codeLFG316
    D.3.9.3Other descriptive namehuman monoclonal antibody directed against complement 5 protein
    D.3.9.4EV Substance CodeSUB32390
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 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 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 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.3.2Product code LFG316
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNo
    D.3.9.2Current sponsor codeLFG316
    D.3.9.3Other descriptive namehuman monoclonal antibody directed against complement 5 protein
    D.3.9.4EV Substance CodeSUB32390
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 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 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.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
    E.1.1.1Medical condition in easily understood language
    Rare and serious blood disease that causes the destruction of red blood cells
    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 LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To assess the effect of LFG316 on the reduction of intravascular hemolysis in PNH patients
    E.2.2Secondary objectives of the trial
    •To assess the tolerability and pharmacokinetics of LFG316 in patients with PNH
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male and female patients >= 18 years old with a diagnosis of PNH prior to screening. Based on local requirements (applicable in Czech Republic) only patients between the age of 18-65
    (inclusive) with a diagnosis of PNH prior to screening may be eligible for inclusion in this study.
    •A documented PNH clone size of ≥10% by RBCs and/or granulocytes, measured by GPI-deficiency on flow cytometry.
    •Serum LDH levels at least 1.5-fold above the upper limit of normal (ULN) at screening.
    •Negative pregnancy test for women of child bearing potential at screening.
    •Previous vaccination against Neisseria meningitidis types A, C, Y and W-135 is required at least 2 weeks prior to first dosing. Vaccination against meningitidis type B should be conducted if available and acceptable by local regulations, at least 2 weeks prior to first dosing.
    •Subjects to be included in this study after protocol amendment 6 also have to fulfill criterion: PNH patients that are carriers of the C5 gene minor variants as defined by nucleic acid changes that lead to amino acid
    exchanges in position p.Arg885.
    E.4Principal exclusion criteria
    •Known or suspected hereditary complement deficiency.
    •History of recurrent meningitis, history of meningococcal meningitis despite vaccination
    •Presence or suspicion (based on judgment of the investigator) of severe active bacterial infection within 2 weeks prior to first dose of LFG316, or severe recurrent bacterial infections .
    •Under active therapy with other agents interfering with the complement system
    •Co-morbidities that are a likely caused by underlying autoimmune diseases other than PNH
    •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 50 days after the last dose of LFG316.
    •Severe concurrent co-morbidites,eg: patients with severe kidney disease (dialysis),advanced cardiac disease (NYHA class IV) severe pulmonary artierial hypertension (WHO classIV), unstable thrombotic event not amenable to active treatment as judged by the investigator.
    •Either on of the following laboratory abnormalities at screening:
    a. Neutrophils <0.5 x 1000000000/L
    b.Platelets<30x1000000000/L
    E.5 End points
    E.5.1Primary end point(s)
    •Reduction in serum LDH levels within the first 4 weeks of treatment
    •Reduction in serum LDH levels over the entire treatment period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weekly in Period 1 and every 14 days in Period 2.
    Patients participating in extension Period 3 will roll over from the last dosing visit of period 2 to the first visit (which includes drug administration) of period 3. The same assessment schedule applied in Period 1 and 2 will be applied between the last dosing visit of Period 2 and the first visit of Period 3.
    E.5.2Secondary end point(s)
    •Standard safety monitoring with increased vigilance for infections
    •Measurement of serum concentrations of LFG316
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety
    Weekly in Period 1 and every 2 weeks in Period 2
    Serum concentration
    Weekly in Period 1
    Every 4 weeks in Period 2
    Patients participating in extension Period 3 will roll over from the last dosing visit of period 2 to the first visit (which includes drug administration) of period 3. The same assessment schedule applied in Period 1 and 2 will be applied between the last dosing visit of Period 2 and the first visit of Period 3.
    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 No
    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 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) 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.8.3 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 EEA3
    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
    Czech Republic
    Japan
    Lithuania
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days27
    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: 9
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 3
    F.4.2.2In the whole clinical trial 10
    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)
    The investigator must provide follow-up medical care for all subjects who are prematurely withdrawn from the study, or must refer them for appropriate ongoing care.This care may include enrollment in an extension study.
    Each subject will be asked to complete this study in its entirety (period 1 and period 2) and they may be offered to enter an additional extension trial, provided LFG316 shows benefit as defined by the response criteria.
    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 Decision2015-07-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-25
    P. End of Trial
    P.End of Trial StatusOngoing
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