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    Summary
    EudraCT Number:2017-003916-37
    Sponsor's Protocol Code Number:08448
    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:2018-02-19
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-003916-37
    A.3Full title of the trial
    Multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Stopping Eculizumab Treatment Safely in atypical Haemolytic Uraemic Syndrome (SETS aHUS)
    A.3.2Name or abbreviated title of the trial where available
    SETS aHUS: Stopping Eculizumab Treatment Safely in aHUS
    A.4.1Sponsor's protocol code number08448
    A.5.4Other Identifiers
    Name:EUDRACTNumber:2017-003916-37
    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 SponsorNewcastle Upon Tyne Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    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 supportNational Institute for Health Research
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNewcastle Clinical Trial Unit
    B.5.2Functional name of contact pointSarah Dunn
    B.5.3 Address:
    B.5.3.1Street Address1-2 Claremont Terrace
    B.5.3.2Town/ cityNewcastle Upon Tyne
    B.5.3.3Post codeNE2 4AE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01912082521
    B.5.6E-mailsarah.dunn2@ncl.ac.uk
    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 Solaris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Pharma UK Ltd
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameSolaris
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPRoute of administration not applicable
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEculizumab
    D.3.9.1CAS number 219685-50-4
    D.3.9.3Other descriptive nameIgG2/4κ
    D.3.9.4EV Substance CodeAS1
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Atypical Haemolytic Uraemic Syndrome (aHUS)
    E.1.1.1Medical condition in easily understood language
    aHUS is a rare disease caused by a fault in the complement system. The complement system is part of your body’s immune response that attacks bugs.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The principle clinical research objective is to determine the safety of Eculizumab withdrawal in patients with aHUS.
    E.2.2Secondary objectives of the trial
    1. Measure the effectiveness of a monitoring protocol to detect disease relapse following withdrawal of Eculizumab.
    2. Describe the relapse rate after withdrawal of Eculizumab.
    3. Estimate the proportion of patients, currently on long-term treatment with Eculizumab, who can be maintained off treatment.
    4. Describe the period from withdrawal to relapse in those patients who restart treatment.
    5. Measure the change in estimated GFR (calculated by the CKD-EPI or modified Schwartz equations) over the course of the study.
    6. Identify important clinical and laboratory indicators of imminent relapse.

    Heath Economic Objectives

    7. To assess the costs and health outcomes (measured in terms of adverse events and quality-adjusted life years (QALYs)) for patients on standard care (not withdrawing from Eculizumab treatment) over the two-year trial duration.
    8. To assess the costs and health outcomes for patients fully, or partially, withdrawing from Eculizumab treatment, and on a policy of protoco
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    There is a linked qualitative study. The IRAS Project ID for this study is 237008.
    This Qualitative Trial will use structured interviews to find out the experience of a subset of patients who have consented to this clinical trial.
    E.3Principal inclusion criteria
    • Age ≥2+ years of age
    • On Eculizumab treatment for at least 6 months
    • In remission with no evidence of ongoing microangiopathic haemolytic anaemia (MAHA) activity at screening defined by:
    - Platelet count > lower limit of normal as determined by local reference range
    - LDH <x2 upper limit of normal as determined by local lab reference ranges
    • Normal renal function or CKD stages 1-3
    • Absence of decline of renal function confirmed by review of available assessments of renal function for the preceding 6 months by the Chief Investigator and clinical members of the TMG
    • Willing to attend for safety monitoring assessments
    • Willing to travel only to countries that can supply Eculizumab (to be confirmed with co-ordinating centre prior to travel).

    The following criteria must be met by those only wishing to be enrolled in the withdrawal component of the trial.
    • Able to perform or parent/guardian to perform and record self-monitoring urinalysis
    • Sexually active female patients who are of child bearing age must have a negative pregnancy test at screening and consent to use effective contraception for the duration of the study as listed in table 1 in the protocol
    OR fulfil one of the following criteria:
    - Be post-menopausal
    - Have undergone surgical sterilisation
    E.4Principal exclusion criteria
    • Severe non-renal disease manifestations at initial presentation with aHUS, which in the opinion of the Chief Investigator and/or the clinical members of the TMG makes the risk of treatment withdrawal unacceptable.
    • Loss of a previous transplant kidney to recurrent aHUS
    • Transplant recipient with a pathogenic mutation in C3, CFH or CFB
    • Current or planned pregnancy
    • Unable to give informed consent or assent, or unable to obtain parent/guardian consent if under 16 years of age
    • Unable to comply with monitoring protocol
    • Current participation in another clinical trial
    • Haematuria rating of 3+
    • Severe, uncontrolled hypertension (systolic blood pressure >160 mmHg) that is likely to induce at TMA.
    • Current participation in another clinical trial (not including participation in ahus registries)
    E.5 End points
    E.5.1Primary end point(s)
    Number of patients with a Thrombotic Microangiopathy (TMA) related Serious Adverse Event (SAE) defined as any of the following:

    • Irreversible (>3 months) reduction in estimated glomerular filtration rate (eGFR) by ≥20%, not attributable to another cause
    • An episode of AKI attributed to a TMA that requires renal replacement therapy
    • A non-renal manifestation of a TMA that require hospitalisation, cause irreversible organ damage or death.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months after the patient has their baseline visit.
    E.5.2Secondary end point(s)
    1. The effectiveness of a monitoring protocol to detect disease relapse following withdrawal of Eculizumab assessed by:
    i. The proportion of patients who relapse and restart Eculizumab without the development of a TMA-related SAE (section 3.3).
    ii. The time from the first clinical feature (symptom, positive urinalysis or laboratory result) of a relapse of TMA and the re-introduction of Eculizumab treatment.
    2. The relapse rate after withdrawal of Eculizumab as determined by the proportion of patients who relapse after Eculizumab is withdrawn.
    3. Estimate of the proportion of patients, currently on long-term treatment with Eculizumab, who can be maintained off treatment.
    4. Description of the period from withdrawal to relapse in those patients who restart treatment measured from baseline (day 0) to day of re-introduction of treatment or end of the study.
    5. The change in estimated GFR as calculated by the CKD-EPI or modified Schwartz equations over the course of the study from baseline (day 0) to end of the study.
    6. Identification of important clinical and laboratory indicators of imminent relapse by review of reported symptoms, physical signs, urinalysis and laboratory results prior to the diagnosis of a relapse.

    Heath Economic Outcome Measures
    7. Cumulative costs to the NHS and participants, and determinants of costs, over the 24-month follow-up period.
    8. QALYs estimated from responses to the EQ-5D-5L, and SF-36, completed at pre-determined time points, and determinants of QALYs/utilities, over the 24-month follow-up period.
    9. Model-based estimate of the costs and health consequences, with results presented in terms of cost per QALY gained, over the estimated lifetime of patients withdrawing from treatment compared with standard care.
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months after the patient has their baseline visit.
    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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 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 concerned20
    E.8.5The trial involves multiple Member States No
    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
    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 months2
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days30
    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: 20
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 28
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 2
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    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)
    Patients will remain withdrawn or can restart Eculizumab treatment depending on their personal choice and clinical judgement.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation North East and North Cumbria Clinical Research Network
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-03-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-13
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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