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    Summary
    EudraCT Number:2021-001088-26
    Sponsor's Protocol Code Number:MN42928
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-02-04
    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 ConcernedGermany - PEI
    A.2EudraCT number2021-001088-26
    A.3Full title of the trial
    SAKURABONSAI: CLINICAL, IMAGING AND BIOMARKER OPEN-LABEL STUDY IN NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD) WITH SATRALIZUMAB AS AN INTERVENTION
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical, Imaging and Biomarker Study in Neuromyelitis Optica Spectrum Disorder (NMOSD) with Satralizumab as an Intervention
    A.4.1Sponsor's protocol code numberMN42928
    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 SponsorF. Hoffmann-La Roche Ltd
    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 supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenentech Inc. c/o F. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.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 Yes
    D.2.1.1.1Trade name Enspryng
    D.2.1.1.2Name of the Marketing Authorisation holderRoche Registration GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/16/1680
    D.3 Description of the IMP
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSatralizumab
    D.3.9.2Current sponsor codeRO5333787
    D.3.9.4EV Substance CodeSUB195082
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    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
    Neuromyelitis Optica Spectrum Disorder (NMOSD)
    E.1.1.1Medical condition in easily understood language
    NMOSD, also known as Devic disease, is a disorder of the brain and spinal cord dominated by inflammation of the optic nerve (optic neuritis) and inflammation of the spinal cord (myelitis).
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10077875
    E.1.2Term Neuromyelitis optica spectrum disorder
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10029322
    E.1.2Term Neuromyelitis optica
    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
    • To describe the efficacy of satralizumab in patients with aquaporin-4 (AQP4) antibody seropositive NMOSD, either treatment naïve or inadequate responders to previous treatment with rituximab (RTX) (or its biosimilar).
    E.2.2Secondary objectives of the trial
    • To describe the evolution of advanced imaging outcomes over time in AQP4 antibody seropositive NMOSD patients treated with satralizumab, either treatment-naïve or inadequate responders to previous treatment with RTX (or its biosimilar)
    • To describe the safety of satralizumab in patients with aquaporin-4 (AQP4) antibody seropositive NMOSD, either treatment naïve or inadequate responders to previous treatment with rituximab (RTX) (or its biosimilars)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age 18 to 74 years, inclusive, at the time of informed consent
    • Have a diagnosis of AQP4 antibody seropositive NMOSD according to the International Panel for NMO Diagnosis (IPND) criteria
    • For women of childbearing potential: agreement to either remain abstinent (refrain from heterosexual intercourse) or to use reliable means of contraception (physical barrier [patient or partner] in conjunction with a spermicidal product, contraceptive pill, patch, injectables, intrauterine device or intrauterine system) during the treatment period and for at least 3 months after the last dose of study drug
    Cohort 1 (treatment-naïve NMOSD patients)
    • Confirmation of NMOSD diagnosis with AQP4+ antibodies
    • Have clinical evidence of at least 1 documented attack or relapse (including first attack) in the last year prior to screening
    • Naïve to maintenance therapy (disease-modifying therapy [DMT] or immunosuppressive therapy [IST])
    Cohort 2 (NMOSD patients with inadequate response to RTX [or its biosimilar])
    • Confirmation of NMOSD diagnosis and AQP4+ antibodies in the disease history of the patient
    • Have a length of disease duration from first symptom of ≤5 years
    • History of ongoing treatment with RTX (or its biosimilar) (at least 2 infusions) for NMOSD with a maximum duration of 6 months since last administration prior to enrolment in the study
    • Ongoing disease activity after last RTX (or its biosimilar) infusion i.e., relapse and/or any new inflammatory event, confirmed by magnetic resonance imaging (MRI) or ophthalmological assessment
    E.4Principal exclusion criteria
    Exclusion criteria for both the cohorts
    • Inability to complete an MRI
    • Participants who are pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the final dose of satralizumab
    • Any surgical procedure (except for minor surgeries) within 4 weeks prior to baseline
    • Evidence of other demyelinating disease, including MS or progressive multifocal leukoencephalopathy (PML)
    • Evidence of serious uncontrolled concomitant diseases that may preclude patient participation
    • Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection or other infection (excluding fungal infections of nail beds or caries dentium) at baseline
    • Infection requiring hospitalization or treatment with intravenous (IV) anti-infective agents within 4 weeks prior to baseline visit
    • Evidence of chronic active hepatitis B
    • Evidence of active tuberculosis (TB)
    • History or laboratory evidence of coagulation disorders
    • Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline
    • Presence or history of malignancy
    • History of drug or alcohol abuse within 1 year prior to baseline
    • History of diverticulitis that, in the Investigator’s opinion, may lead to increased risk of complications such as lower gastrointestinal perforation
    • History of severe allergic reaction to a biologic agent
    • Active suicidal ideation within 6 months prior to screening, or history of suicide attempt within 3 years prior to screening
    • Treatment with any investigational agent within 6 months prior to baseline or 5 drug elimination half-lives of the investigational agent (whichever is longer)

    Cohort 1 (treatment-naïve NMOSD patients)
    • Any previous treatment with IL-6 inhibitory therapy (e.g., tocilizumab), alemtuzumab, total body irradiation, stem-cell therapy, or bone marrow transplantation
    • Any previous treatment with eculizumab, belimumab, natalizumab, glatiramer acetate, fingolimod, teriflunomide, dimethyl fumarate, siponimod, or ozanimod
    • Any previous treatment with anti-CD4, cladribine or mitoxantrone
    • Any previous treatment with B-cell depleting agents
    • Any previous treatment with immunosuppressants

    Cohort 2 (NMOSD patients with inadequate response to RTX)
    • Discontinued RTX (or biosimilar) treatment due to any other reason than inadequate response to treatment
    • The following laboratory abnormalities at screening:
    - White blood cells (WBC) < 3.0 × 10^3/μL
    - Absolute neutrophil count (ANC) < 2.0 × 10^3/μL
    - Absolute lymphocyte count (ALC) < 0.5 × 10^3/μL
    - Platelet count < 10 × 10^4/μL
    - Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 1.5 times the upper limit of normal (ULN)
    - Positive serum beta human chorionic gonadotropin (hCG) measured at screening or positive urine dipstick pregnancy test prior to initiation of study treatment
    E.5 End points
    E.5.1Primary end point(s)
    1. Clinical measures related to disease activity and progression over 96 weeks
    2. Proportion of relapse-free patients
    3. Annualized relapse rate (ARR)
    4. Time to first relapse (TFR)
    5. Severity of relapses (hospitalization; use of corticosteroids; rescue therapy; need for plasma exchange; residual disability)
    6. Mean change from baseline in expanded disability status scale (EDSS) score over the course of the study
    7. Time to onset of confirmed disability progression (CDP) sustained for at least 12 weeks and 24 weeks
    8. Change from baseline in the Symbol Digital Modalities Test (SDMT) over the course of the study
    9. Change in high-contrast (100%) and low contrast (2.5%) visual acuity using appropriate high-and low-contrast letter acuity (LCLA) charts over the course of the study
    10. Change in Visual Functioning Questionnaire-25 (VFQ-25) over the course of the study
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-5. Up to Week 96
    6-7. Baseline (Day -28 to Day -1) to Week 96
    8-10. Baseline (Day -28 to Day -1) to Week 96
    E.5.2Secondary end point(s)
    1. Count, volume and regional distribution of T2-weighted fluid-attenuated inversion recovery (FLAIR) hyperintense lesions, including new and enlarging lesions of the cerebrum, optic nerves, optic chiasm, area postrema, brainstem and cerebellum; Fazekas scoring
    2. Contrast-enhancing T1-weighted lesions (CEL) of the cerebrum, optic nerves, optic chiasm, area postrema, brainstem and cerebellum; optionally meningeal enhancement
    3. Diffusion abnormalities of brain and optic nerves
    4. Microbleeds of brain and optic nerves
    5. Cerebral perfusion alterations
    6. Global and regional brain volume loss including basal ganglia, cerebellum and upper cervical spinal cord. Regional brain volume will be assessed only at screening, Week 48 and Week 96
    7. New and persisting short T1 inversion recovery (STIR)/proton density (PD) hyperintense lesions and T1-weighted contrast enhancement: Qualitative neuroradiological assessment
    8. Quantitative T1 mapping (magnetization-prepared rapid gradient echo sequence [MP2RAGE])
    9. T2*/R* for iron concentration estimation
    10. Quantitative diffusion/diffusion tensor imaging (DTI)
    11. Change in the retinal nerve fiber layer (RNFL) thickness
    12. Change in the ganglion cell plus inner plexiform (GCIP) layer thickness
    13. Incidence and severity of adverse events (AEs), serious AEs (SAEs) and AEs of special interest (AESIs)
    14. Change from Baseline in vital signs, physical examinations, and clinical laboratory tests
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-5. Up to Week 96
    6. At Screening, Weeks 48 and 96
    7-10. Up to Week 96
    11-12. Baseline (Day -28 to Day -1) to Week 96
    13. Up to Week 96
    14. Baseline (Day -28 to Day -1) to Week 96
    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 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) 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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    Germany
    India
    Italy
    Korea, Republic of
    Turkey
    United States
    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
    The end of this study is defined as the date when the last patient, last visit (LPLV) occurs or the date at which the last data point required for statistical analysis or safety follow-up is received from the last patient, whichever occurs later.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 95
    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 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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 100
    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 who complete the 96-week treatment period and choose not to continue on satralizumab treatment outside this study, or who discontinue treatment early and choose not to continue on satralizumab treatment outside this study, will be followed up for 12 weeks after the last dose of satralizumab.
    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 Decision2022-02-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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