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    Summary
    EudraCT Number:2020-004436-21
    Sponsor's Protocol Code Number:WN42636
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-03-11
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2020-004436-21
    A.3Full title of the trial
    A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH GENERALIZED MYASTHENIA GRAVIS
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Patients with Generalized Myasthenia Gravis
    A.4.1Sponsor's protocol code numberWN42636
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/532/2021
    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 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.3.1Product nameSatralizumab
    D.3.2Product code RO5333787
    D.3.4Pharmaceutical form Solution for injection 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.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.3.1Product nameSatralizumab
    D.3.2Product code Ro 533-3787/F01-06
    D.3.4Pharmaceutical form Solution for injection 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboInjection
    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
    Generalized Myasthenia Gravis (gMG)
    E.1.1.1Medical condition in easily understood language
    Myasthenia gravis is a rare neuromuscular disorder that causes weakness in the skeletal muscles. Myasthenia gravis affecting multiple muscle groups throughout the body is called gMG.
    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 10028417
    E.1.2Term Myasthenia gravis
    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
    Evaluate the efficacy of satralizumab versus placebo on function in daily life in the acetylcholine receptor antibody seropositive (AChR+) population
    E.2.2Secondary objectives of the trial
    • Evaluate the efficacy of satralizumab vs placebo on function in daily life in the overall population (OP)
    • Evaluate the efficacy of satralizumab vs placebo in the AChR+ population and OP on function in daily life, QMG, QoL, Fatigue, Clinical status and Disease severity
    • Evaluate the durability of the efficacy of satralizumab vs placebo in the AChR+ population and the OP
    • Evaluate the safety of satralizumab vs placebo
    • Confirm target engagement and pathway inhibition in response to satralizumab
    • Investigate the PK of satralizumab by evaluating plasma exposure over 24 weeks
    • Evaluate the immune response to satralizumab
    • Evaluate the long-term safety and tolerability of satralizumab
    • Assess the efficacy of satralizumab in the AChR + population and OP
    • Assess the effect of satralizumab on steroid/IST/AChEI dose modification in the AChR + population and OP
    • Investigate the pharmacokinetics of satralizumab by evaluating plasma exposure in the OLE
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age >= 12 years (or >= 18 years in France) at time of signing Informed Consent Form
    • Confirmed diagnosis of gMG meeting the following criteria:
    - Documented history of myasthenic weakness
    - MG severity of MGFA Class II, III, or IV (Patients with MG Class IV at participating sites in France are excluded from the study) at screening
    - The confirmation of the diagnosis has to be documented and supported by positive serologic test for one of the three antibody types: anti- AChR, anti- MuSK or anti- LRP4 at screening (antibody status must be confirmed by the central laboratory for all antibody types). For sites in Germany and the Netherlands, confirmation of the diagnosis has to be documented and supported by pre-existing positive serologic test results for one of the three antibody types (anti-AChR, anti-MuSK, or anti-LRP4), which must have been ordered by a health care professional (HCP) for the patient as part of the patient’s historical SOC
    • A total MG-ADL score of >=5 points at screening with more than 50% of this score attributed to non-ocular items
    • Ongoing gMG treatment at a stable dose and not exceeding the maximum protocol allowed doses
    • No contraindication to at least one of the rescue treatments: IVIg, PE, or high dose corticosteroids
    • For female patients of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab
    E.4Principal exclusion criteria
    Exclusion Criteria Related to Myasthenia Gravis (MG):
    • History of thymic cysts, thymoma, thymic carcinoma or other neoplasm of the thymus as defined by the 2015 WHO classification of tumors of the thymus unless deemed cured by adequate treatment with no evidence of recurrence for >=5 years before screening
    • History of thymectomy within 6 months prior to screening
    • Ocular MG (Myasthenia Gravis Foundation of America [MGFA] Class I). In France, MGFA Class IV MG at screening
    • Use of IVIg or subcutaneous immunoglobulin (SCIg) within 6 weeks prior to randomization/Day 1
    • Use of PE within 8 weeks prior to randomization/Day 1
    • Any surgical procedure (except for non-ophthalmic minor surgeries) within 4 weeks prior to screening and planned surgical procedure (except non-ophthalmic minor surgeries) during the study
    E.5 End points
    E.5.1Primary end point(s)
    1. Mean change from baseline in total MG-ADL score (AChR+ population) Week 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. From baseline to Week 24
    E.5.2Secondary end point(s)
    1. Mean change from baseline in total MG-ADL score (OP population)
    2. Mean change from baseline in QMG score, MG-QOL 15r total score and Neuro-QoL Fatigue Subscale total score
    3. Mean change from baseline in total Myasthenia Gravis Composite (MGC) score
    4. Percentage of patients with a >=2-point reduction from baseline in total MG-ADL score
    5. Percentage of patients with a >=3-point reduction from baseline in QMG score
    6. Percentage of patients with a >=3-point reduction from baseline in total MGC score at Week 24
    7. Proportion of patients who have achieved minimal disease manifestation (total MG-ADL score of 0 or 1) at Week 24
    8. Proportion of patients with at least one gMG-related exacerbation and receiving rescue therapy between baseline and Week 24
    9. Proportion of patients receiving rescue therapy between baseline and Week 24
    10. Annualized rate of gMG-related exacerbations
    11. Duration (average number of consecutive months) of meaningful improvement, defined as >=2-point reduction from baseline in total MG-ADL score
    12. Incidence and severity of adverse events, with severity determined according to NCI CTCAE v5.0 grading
    13. Change from baseline in targeted vital signs, ECG results, physical examination findings, targeted clinical laboratory test results, and suicidality
    14. Absolute values and change from baseline in serum levels of biomarkers IL- 6 and sIL- 6R
    15. Serum concentrations of satralizumab (mean and SD of Ctrough) at specified timepoints
    16. Estimates of primary PK parameters (e.g., CL/F and V/F) and secondary PK parameters (e.g., AUC) derived using population-PK modeling
    17. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study
    18. Mean change from active treatment baseline in:
    • MG ADL total score
    • QMG score
    • MGC score
    • MG QOL 15r score
    19. Percentage of responders based on:
    • >= 2-point reduction in total MG ADL score or
    • >= 3-point reduction in QMG score or
    • >= 3-point reduction in total MGC score
    20. Proportion of time and duration that patients show a meaningful improvement, defined as a ≥ 2 point reduction from active treatment baseline in total MG ADL score
    21. Number and severity of gMG-related exacerbations
    22. The proportion of patients who maintain clinical response without increase in symptomatic medication dose and are able to reduce corticosteroid dose or withdraw from corticosteroid or IST during the OLE
    23. Serum concentrations of satralizumab (mean and SD of Ctrough) during the OLE
    24. Estimates of primary PK parameters (e.g., CL/F and V/F) and secondary PK parameters (e.g., AUC) derived using population-PK modeling during the OLE
    25. Prevalence of ADAs at baseline and incidence of ADAs during the OLE
    26. Absolute values and change from baseline in serum levels of PD biomarkers IL- 6 and sIL- 6R
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-3, 8-9. Baseline and Week 24.
    4-7, 18-20. At Week 24.
    10-14, 21-22, 26: Baseline to Week 24.
    15-16, 23-24. At Day 1, Weeks 2, 4, 8, 12, 16, 20, and 24.
    17, 25. Predefined timepoints.
    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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    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 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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    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
    Argentina
    Australia
    Brazil
    Canada
    China
    Japan
    Korea, Republic of
    Russian Federation
    Turkey
    United States
    Czechia
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    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
    LPLV, or the date at which the last data point required for statistical analysis or SFU 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 years4
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    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 Yes
    F.1.1Number of subjects for this age range: 6
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 147
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 32
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 56
    F.4.2.2In the whole clinical trial 185
    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 can continue to receive treatment with satralizumab in the OLE period until commercial availability of satralizumab in patients with gMG, the availability of satralizumab as post-trial access in accordance with local regulation, or until the Sponsor decides to discontinue the development program.
    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 Decision2021-04-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-11
    P. End of Trial
    P.End of Trial StatusOngoing
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