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    Summary
    EudraCT Number:2021-002076-39
    Sponsor's Protocol Code Number:WC42759
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2022-03-08
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-002076-39
    A.3Full title of the trial
    A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE SAFETY AND EFFICACY OF BALOVAPTAN IN PATIENTS WITH ACUTE ISCHEMIC STROKE AT HIGH RISK OF DEVELOPING MALIGNANT CEREBRAL EDEMA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety and Ability of Balovaptan to Reduce the Risk of Severe Brain Swelling in Patients with Acute Ischemic Stroke
    A.4.1Sponsor's protocol code numberWC42759
    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 organisationF.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 nameBalovaptan
    D.3.2Product code RO5285119/F48-01
    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 INNBalovaptan
    D.3.9.1CAS number 1228088-30-9
    D.3.9.2Current sponsor codeRO5285119
    D.3.9.4EV Substance CodeSUB213812
    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 Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Acute Ischemic Stroke (AIS)
    E.1.1.1Medical condition in easily understood language
    AIS is defined by the sudden loss of blood flow to an area of the brain with the resulting loss of neurologic function and can result in permanent damage to the affected area of the brain.
    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 LLT
    E.1.2Classification code 10008107
    E.1.2Term Cerebral edema
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    ● To evaluate the efficacy of balovaptan compared with placebo based on the amount of midline shift (MLS)
    E.2.2Secondary objectives of the trial
    ● To evaluate the efficacy of balovaptan compared with placebo based on global disability, amount of MLS, proportion of patients with surgical decompressive hemicraniectomy (DHC), proportion of patients who received hyperosmolar therapy, impairment of consciousness, neurological impairment, mortality, functional independence, stroke disability and health-related quality of life, hospital length of stay and ICU length of stay
    ● To evaluate the safety of balovaptan compared with placebo
    ● To characterize the balovaptan, M2, and M3 pharmacokinetic (PK) profile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ● Participants who are between the ages >=18 and <=80 years at the time of signing Informed Consent Form
    ● Diagnosis of large vessel occlusion (LVO) in the anterior circulation such that study drug administration can be initiated within 12 hours of last known well (LKW) and at risk of malignant cerebral edema (MCE) development, as defined as follows: Documented occlusion of terminus ICA and/or MCA on CTA or magnetic resonance angiogram AND ASPECTS score <= 5 AND National Institutes of Health Stroke Scale (NIHSS) > 15 for the non-dominant hemisphere and > 20 for the dominant hemisphere
    ● Participants who present with a wake-up stroke (WUS) who are <=8 hours from awakening
    ● Participants with a history of seizures on anti-epileptic medications may be included if they have been on stable doses of those medications for at least 12 weeks prior to LKW, they have not experienced seizures during that time frame, and their anti-epileptic medicines are continued during the study
    ● For women of childbearing potential agreement to remain abstinent or use contraception and agree to refrain from donating eggs, during the treatment period and for at least 28 days after the final dose of balovaptan
    ● No specific contraception methods for males are required
    E.4Principal exclusion criteria
    ● Participants who are >12 hours from LKW at the start of treatment with study drug or >8 hours from awakening with WUS
    ● Any MLS on brain imaging
    ● Evidence of parenchymatous hematoma ([PH]1 or PH2) on baseline imaging (per Heidelberg classification)
    ● Evidence of additional anterior cerebral artery (ACA) infarction
    ● Diagnosis of brain death
    ● Planned surgical decompression prior to randomization
    ● Participants with a known history of a hereditary bleeding disorder which increases bleeding risk
    ● Chronic kidney disease stage III or higher
    ● Hepatic injury
    ● Diagnosis of diabetes insipidus
    ● Participants who have received any prophylactic hyperosmolar therapy
    ● Participants who have received treatment with any other V1a and/or V2 receptor-blocking agent or glyburide
    ● A preexisting medical condition for which the participant is unlikely to survive the next 6 months
    ● Planned limitation or withdrawal of life-sustaining treatment during hospital admission
    ● Participants who are pregnant or breastfeeding, or intending to become pregnant
    E.5 End points
    E.5.1Primary end point(s)
    1. Amount of MLS, as measured in millimeters at the level of the septum pellucidum on NCCT at 72 hours from LKW
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. At 72 hours from LKW
    E.5.2Secondary end point(s)
    1. Global disability, defined as the proportion of participants with modified Rankin Scale-Structured Interview (mRS-SI) score <=4 versus >4 measured at Day 90
    2. Amount of MLS, as measured in millimeters at the level of the septum pellucidum on non-contrast computed tomography (NCCT) at 48 hours and 96-120 hours from LKW
    3. Proportion of patients with surgical DHC performed
    4. Proportion of patients who received hyperosmolar therapy following initiation of study treatment
    5. Impairment of consciousness, as measured by the Glasgow Coma Scale (GCS) score up to Day 4
    6. Neurological impairment, as measured by the NIHSS on hospital Day 4 and Day 90
    7. Mortality at Day 30
    8. Global disability, as measured by the mRS-SI at Day 30
    9. Functional independence, as measured by the FIM at discharge or Day 10 and Day 90
    10. Stroke disability and health-related quality of life, as measured by the Stroke Impact Scale (SIS)-16 at Day 30 and Day 90
    11. Hospital length of stay
    12. ICU length of stay
    13. Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)
    14. Any safety signals seen on brain imaging
    15. Change from baseline in targeted vital signs and ECG
    16. Change from baseline in targeted clinical laboratory test results
    17. Plasma concentration of balovaptan and its major metabolites (M2 and M3) at specified timepoints
    18. Area under the concentration-time curve from Time 0 to 24 hours after a given dose (AUC24hr) of balovaptan and its major metabolites (M2 and M3) as calculated by non-compartmental analysis (NCA)
    19. Maximum observed concentration (Cmax) of balovaptan and its major metabolites (M2 and M3) as calculated by NCA or taken directly from measured concentration
    20. Plasma drug concentration 24 hours after the administration of a given dose (C24hr) of balovaptan and its major metabolites (M2 and M3) as calculated by NCA or taken directly from measured concentration
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. At Day 90
    2. At 48 hours and 96-120 hours from LKW
    3. Up to Day 90
    4. Up to Day 90
    5. Up to Day 4
    6. At Day 4 and Day 90
    7. At Day 30
    8. At Day 30
    9. At discharge or Day 10 and at Day 90
    10. At Day 30 and Day 90
    11-12. Up to Day 90
    13-Screening to Day 90
    14-16. Baseline to Day 90
    17-20. At 1 hour prior to drug administration; at 5 minutes, 2.5, 5, 12 hours after completion of each drug administration and at 24, 48 and 120 hours after completion of the last drug administration
    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 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 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 No
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    United States
    France
    Spain
    United Kingdom
    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 years
    E.8.9.1In the Member State concerned months28
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months28
    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: 49
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 59
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    A legally authorized representative will be used for the consent process if the patient is deemed by the treating physician and/or site investigator to not have decision making capacity due to the nature of their stroke symptoms.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 108
    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)
    Currently, the Sponsor does not have any plans to provide balovaptan or any other study treatments to participants who have completed the study. The Sponsor may evaluate whether to continue providing balovaptan in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product, available at the following
    website: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    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 Decision2022-05-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-06-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-11-17
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