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    Summary
    EudraCT Number:2012-005753-23
    Sponsor's Protocol Code Number:NI-501-05
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-03-30
    Trial results View 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 ConcernedSweden - MPA
    A.2EudraCT number2012-005753-23
    A.3Full title of the trial
    A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the long-term safety and efficacy of a new drug, NI-0501, in children with a disease that is called "Haemophagocytic Lymphohistiocytosis".
    A.4.1Sponsor's protocol code numberNI-501-05
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02069899
    A.5.4Other Identifiers
    Name:US INDNumber:111015
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/358/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovimmune SA
    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 supportNovimmune SA
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportSeventh Framework Programme for Research
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovimmune SA
    B.5.2Functional name of contact pointAlessandra Blasiotti
    B.5.3 Address:
    B.5.3.1Street Address14 Chemin des Aulx
    B.5.3.2Town/ cityPlan-les-Ouates
    B.5.3.3Post code1228
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+4122 593 51 62
    B.5.5Fax number+4122 593 82 80
    B.5.6E-mailNI-0501.clinops@novimmune.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/3/10/749
    D.3 Description of the IMP
    D.3.1Product nameemapalumab
    D.3.2Product code NI-0501
    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 INNEMAPALUMAB
    D.3.9.2Current sponsor codeNI-0501
    D.3.9.4EV Substance CodeSUB188645
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    Haemophagocytic lymphohistiocytosis
    E.1.1.1Medical condition in easily understood language
    A rare, life-threatening condition affecting predominantly children. The disease is characterized by uncontrolled inflammation.
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10071583
    E.1.2Term Haemophagocytic lymphohistiocytosis
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To monitor the long-term safety profile of NI-0501.
    E.2.2Secondary objectives of the trial
    - To assess HLH patients' survival after NI-0501 treatment
    - To assess duration of response to NI-0501 treatment (i.e. maintenance of HLH control)
    - To assess post-HSCT outcome measures, if applicable
    - To assess background disease activity, in patients with secondary forms of HLH
    - To study the elimination profile of NI-0501
    - To evaluate the pharmacodynamic (PD) effects (levels of circulating Total IFNγ, CXCL9, CXCL10)
    - To assess the profile of relevant HLH biomarkers, e.g., sCD25
    - To assess the immunogenicity of NI-0501.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Having received at least one dose of NI-0501 during a previous NI-0501 study or under a CU treatment protocol
    2. Informed Consent by the patient or the patient's legal representative(s), as applicable, with the assent of patients who are legally capable of providing it
    3. Having agreed to continue using adequate methods of birth control until 6 months after the last administered dose of NI-0501, whenever relevant. Males with partners(s) of child-bearing potential must agree to take appropriate precautions to avoid fathering a child until 6 months after receiving last dose of NI-0501.
    E.4Principal exclusion criteria
    None
    E.5 End points
    E.5.1Primary end point(s)
    Safety will be assessed as follows:
    • Incidence, seriousness, intensity, relationship to NI-0501 and outcome of adverse events (AEs)
    • Evolution over time of vital signs, physical examination and laboratory values
    E.5.1.1Timepoint(s) of evaluation of this end point
    See E.5.1
    E.5.2Secondary end point(s)
    Efficacy (as relevant, depending on the different patients' characteristics):
    • Duration of Response after completion of NI-0501 treatment (assessed according to the definitions set in the parent study)
    • Survival time up to one year post-HSCT (including survival to HSCT and survival post-HSCT) or one year after last NI-0501 infusion (if transplant is not performed)
    • Post-HSCT outcome indices, e.g. engraftment rate, donor chimerism, incidence of acute and chronic Graft versus Host Disease (GvHD) (it applies to patients who receive HSCT)
    • Monitoring of background disease activity (it applies to patients with secondary forms of HLH)

    Pharmacokinetics: NI-0501 elimination profile

    Pharmacodynamics: IFNγ total

    Immunogenicity: presence of ADAs

    Exploratory PD parameters/endpoints: e.g. additional markers of disease activity

    sCD25, CXCL9, CXCL10
    E.5.2.1Timepoint(s) of evaluation of this end point
    See E.5.2
    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 Yes
    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.8.2.4Number 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 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
    Canada
    France
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    United States
    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 years5
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 55
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 3
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 33
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Newborns, infants, toddlers and children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 55
    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)
    In case of an ongoing serious adverse event, patients will continue to be monitored until resolution or until the outcome of the event is known and stable, beyond the defined study end, as necessary.
    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 Decision2015-05-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-25
    P. End of Trial
    P.End of Trial StatusCompleted
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