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    Summary
    EudraCT Number:2015-005347-14
    Sponsor's Protocol Code Number:H2020/PHRC-N/2014/GB-01
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2016-05-09
    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 ConcernedUK - MHRA
    A.2EudraCT number2015-005347-14
    A.3Full title of the trial
    Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for controlling neuro-inflammation in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients:
    A randomized, double-blind, placebo- controlled, phase-II Proof of Concept/ Proof of Mechanism Clinical Trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for controlling neuro-inflammation in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients
    A.3.2Name or abbreviated title of the trial where available
    MIROCALS
    A.4.1Sponsor's protocol code numberH2020/PHRC-N/2014/GB-01
    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 SponsorCHU DE NIMES
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMinistry of health
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportThe Motor Neurone Disease Association
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportEuropean commission
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCHU Nîmes
    B.5.2Functional name of contact pointClinical trial information
    B.5.3 Address:
    B.5.3.1Street Addressplace du Pr Debré
    B.5.3.2Town/ cityNîmes
    B.5.3.3Post code30029
    B.5.3.4CountryFrance
    B.5.4Telephone number+334 66 68 40 25
    B.5.5Fax number+ 334 66 68 34 00
    B.5.6E-maildrc@chu-nimes.fr
    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 Yes
    D.2.1.1.1Trade name Proleukin 18 MUI
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Pharmaceuticals UK Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameproleukin
    D.3.4Pharmaceutical form Solution for infusion in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis (ALS)
    E.1.1.1Medical condition in easily understood language
    MOTOR NEURONE DISEASE (MND)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous 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
    The main Objective is to evaluate the clinical efficacy and safety of the experimental drug (ld IL-2) over an 18 months period, in order to establish the proof of concept (PoC) that modifying immune responses through the enhancement of regulatory T cells, modifies rate of ALS disease progression.
    E.2.2Secondary objectives of the trial
    The secondary Objectives are:
    To validate a new phase-II study design to improve the efficiency of drug development in ALS with early determination of drug response using established biomarkers (BMs).
    The aims of this new trial design are:
    (i) To shorten future trials duration in ALS using an early drug responding surrogate marker of disease activity;
    (ii) To establish the proof of mechanism (PoM) of the tested drugs;
    (iii) To identify drug responder status.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    An ancillary study will be performed on a subgroup of the subjects included in the clinical trial. This will be optional to the patients and will be conducted in the UK only.
    The new exploratory objective addresses this ancillary study;
    (iv) Neuroimaging study
    E.3Principal inclusion criteria
    Inclusion criteria for the persons taking part in the trial
    1 / Run-in period
    •The patient has been correctly informed
    •The patient must have given his/her informed and signed consent.
    •The patient must be 18 years old and less than 76 years old
    •Possible, Probable, Probable laboratory-supported or Definite ALS as defined by El Escorial Revised ALS diagnostic criteria
    •Disease duration ≤ 24 months
    •Slow vital capacity ≥ 70% of normal
    •No prior (ie. less than 4 weeks/ 28 days) or current riluzole treatment; when riluzole treatment is present and the prior duration of treatment is less than or equal to 4 weeks (28 days), a riluzole wash-out period of at least 4 weeks (28 days) must be performed prior to entry.
    •Lumbar punctures accepted by patient and feasible

    2/ Randomised controlled trial period
    •Run-in period completed, including lumbar puncture and sampling for blood biomarkers at inclusion and at 3 months
    •The patient is willing to continue participation
    •Disease duration ≤ 28 months
    •Stable dose of riluzole treatment for at least 3 months
    E.4Principal exclusion criteria
    Exclusion criteria for persons taking part in the trial (run-in and Randomised Control Trial periods)
    •The patient is participating in another research study that may interfere with the results or conclusions of this study
    •Within the past three months, the patient has participated in another study that may interfere with the results or conclusions of this study
    •The patient is in an exclusion period determined by a previous study
    •The patient does not have capacity to give fully informed consent and/or to comply with study requirements and procedures
    •Significant cognitive impairment as judged by investigator on the basis of his/her assessment of the patient, taking into account information from caregivers
    •Other neurodegenerative disease that could explain signs or symptoms
    •Contra indication for lumbar puncture (presence of contra-indicated treatment, –see section 11.3.2, or coagulation test abnormality, clinically significant coagulopathy or thrombocytopenia)
    •Non authorised treatment (see section 11.3 of the protocol)
    •Other disease or disorders that could preclude functional assessments or life-threatening disorders
    •Any Cancer within the past 5 years (except stable non-metastatic basal cell skin carcinoma or in situ carcinoma of the cervix)
    •Severe cardiac or pulmonary disease
    •Any documented, active, past or present ,auto-immune disorders except asymptomatic Hashimoto thyroiditis
    •Using assisted ventilation
    •Gastrostomy
    •Women of child-bearing age and sexually active men without effective contraception or woman who are pregnant or breast feeding
    •Any clinically significant laboratory abnormality (excepting cholesterol, triglyceride and glucose).
    •Clinically significant evidence of active viral infection at randomisation: CMV, EBV, HIV-1, HBV or HCV
    •History of documented symptomatic and treated asthma within the past 5 years
    E.5 End points
    E.5.1Primary end point(s)
    Survival from randomisation to date of death from any cause or date of end of study
    E.5.1.1Timepoint(s) of evaluation of this end point
    548 days
    E.5.2Secondary end point(s)
    A.Secondary clinical efficacy :
    A.1 ALSFRSr;
    A.2 Slow Vital Capacity;
    A.3 QoL EuroQL5D,
    A.4 ECAS
    B.Secondary laboratory efficacy
    B.1 Immuno-cytometry: Tregs (CD4+CD25+CD127-FOXP3+), Total Lymphocytes, CD3+, CD4+, CD8+ , NK (CD3-CD56+), B(CD19
    B.2 pNFH and CCL2 levels (CSF and Blood)
    E.5.2.1Timepoint(s) of evaluation of this end point
    A.Secondary clinical efficacy :
    A.1 monthly
    A.2 6-monthly
    A.3 6-monthly
    A.4 at inclusion and at month 4
    B.Secondary laboratory efficacy
    B.1 at Inclusion, Randomisation, D8, D113 and D120
    B.2 pNFH and CCL2 levels (Blood) at Inclusion,Randomisation, D8, D113 and D120, pNFH and CCL2 levels (CSF)at Inclusion, Randomisation and D113
    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 No
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned5
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    The end of the Trial corresponds to the date on wich the database is locked
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months36
    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: 54
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 162
    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 state72
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 216
    F.4.2.2In the whole clinical trial 216
    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)
    After the completion of the study, treatment with ld IL-2 will not be provided by the study sponsor as sufficient efficacy and safety data will not yet be available.
    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 Decision2016-07-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-25
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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