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    Summary
    EudraCT Number:2022-001672-34
    Sponsor's Protocol Code Number:CVAY736I12301
    National Competent Authority:Romania - National Agency for Medicines and Medical Devices
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2024-10-21
    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 ConcernedRomania - National Agency for Medicines and Medical Devices
    A.2EudraCT number2022-001672-34
    A.3Full title of the trial
    A phase III, randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to first-line corticosteroids in primary immune thrombocytopenia (VAYHIT1)
    Studiu dublu-orb, randomizat, de fază III privind comparația ianalumab (VAY736) față de placebo în asociere cu corticosteroizi de primă linie în trombocitopenia imună primară (VAYHIT1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of ianalumab (VAY736) in addition to first-line corticosteroids in patients with primary immune thrombocytopenia
    A.4.1Sponsor's protocol code numberCVAY736I12301
    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 SponsorNovartis Pharma AG
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma Services Romania S.R.L.
    B.5.2Functional name of contact pointRadu Boroghina - TMO Head Romania
    B.5.3 Address:
    B.5.3.1Street Address2 Gara Herastrau St., Equilibrium Complex, Building 1, Floor 10, Section E10.02, District 2
    B.5.3.2Town/ cityBucharest
    B.5.3.3Post code020334
    B.5.3.4CountryRomania
    B.5.4Telephone number+40213129901
    B.5.5Fax number+40213129907
    B.5.6E-mailradu_dan.boroghina@novartis.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 nameIanalumab
    D.3.2Product code VAY736
    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 INNIanalumab
    D.3.9.2Current sponsor codeVAY736
    D.3.9.4EV Substance CodeSUB193896
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    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
    Primary Immune thrombocytopenia (ITP)
    E.1.1.1Medical condition in easily understood language
    immune thrombocytopenia (ITP)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate that the addition of ianalumab (either dose) to standard first-line corticosteroids prolongs Time to Treatment Failure (TTF) compared to the control arm (placebo + corticosteroids) in participants with primary ITP who responded to corticosteroids (+/-IVIG) prior to randomization
    E.2.2Secondary objectives of the trial
    - To assess quality of response, time to and duration of complete response in each treatment group
    - To assess the incidence and severity of bleeding in each treatment arm
    - To assess the need of rescue treatment in each treatment group
    - To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HRQoL)
    - To assess B-cell and immunoglobulin levels in each treatment group
    - To assess ianalumab pharmacokinetics (PK)
    - To assess the immunogenicity of ianalumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Signed informed consent prior to participation in the study.
    -Male or female participants aged 18 years and older on the day of signing informed consent
    -Primary ITP diagnosed within 3 months before initiating first-line ITP therapy (corticosteroids, IVIG)
    -Platelet count below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG)
    - Response (platelet count >=50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet count measured within 7 days of platelet transfusion will not be considered as response.
    E.4Principal exclusion criteria
    -Evans syndrome or any other cytopenia (patients with anemia related to bleeding iron deficiency are eligible)
    -Current life-threatening bleeding
    -Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up 28 days before randomization/or IVIG given prior to confirmed diagnosis of primary ITP.
    -Prior use of B-cell depleting therapy (e.g., rituximab)
    -Absolute neutrophil count below 1.0 G/L at randomization
    -Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid

    Other protocol-defined Inclusion/Exclusion may apply.
    E.5 End points
    E.5.1Primary end point(s)
    Time from randomization to treatment failure (TTF) defined as platelet count below 30 G/L, need for a rescue treatment, start of a second-line therapy or death.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Randomization to end of study (up to 39 months after randomization of last patient)
    E.5.2Secondary end point(s)
    1. Complete Response (CR) rate in each treatment group
    2. Response (R) rate in each treatment group
    3. Time to response / complete response in each treatment group
    4. Duration of response in each treatment group
    5. Stable response in each treatment group
    6. Proportion of participants with bleeding events overall and by WHO bleeding scale severity
    7. Number and proportion of participants receiving rescue treatment
    8. Cumulative dose/duration of steroids exposure
    9. Change from baseline on T-scores of the PROMIS SF v1.0 Fatigue 13a
    10. Change from baseline in ITP-PAQ domain scores of Symptoms, Fatigue, Bother, Activity
    11. Change from baseline in frequency and absolute number of CD19+ B cell counts
    12. Time to first occurrence of B-cell recovery
    13. Change from baseline in inmmunoglobulins
    14. PK parameters: AUClast, AUCtau
    - PK parameters: Cmax
    - PK parameters: Tmax
    - PK parameters: Accumulation ratio Racc
    15. Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-4, 6-7, 10-12 Randomization to end of study (up to 39 months after randomization of last patient)
    5: At 6 months and 1 year
    7-9 From screening (baseline) till end of study (up to 39 months after randomization of last patient)
    13: After the first and last dose of study medication
    14: Up to end of study (up to 39 months after randomization of last patient)
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    - Patient reported outcomes
    - 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 trial3
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA42
    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
    Malaysia
    Singapore
    Hong Kong
    Australia
    China
    India
    Japan
    Korea, Republic of
    Mexico
    Thailand
    United Kingdom
    United States
    Viet Nam
    Austria
    Belgium
    Czechia
    France
    Germany
    Hungary
    Italy
    Norway
    Romania
    Spain
    Türkiye
    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 years5
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days18
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days15
    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: 169
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 56
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 95
    F.4.2.2In the whole clinical trial 225
    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)
    None
    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 Decision2022-11-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-31
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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