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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7259   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2022-001627-32
    Sponsor's Protocol Code Number:CVAY736Q12301
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-10-31
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-001627-32
    A.3Full title of the trial
    A phase 3 randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to eltrombopag in patients with primary immune thrombocytopenia (ITP) who had an insufficient response or relapsed after first line steroid treatment (VAYHIT2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 3, randomized, double-blind, study of ianalumab (VAY736) versus placebo in addition to eltrombopag in primary immune thrombocytopenia (ITP) patients who failed steroids
    A.3.2Name or abbreviated title of the trial where available
    VAYHIT2
    A.4.1Sponsor's protocol code numberCVAY736Q12301
    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 AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41613241 111
    B.5.5Fax number+41613248 001
    B.5.6E-mailclinicaltrial.enquiries@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 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 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
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level PT
    E.1.2Classification code 10083842
    E.1.2Term Immune thrombocytopenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic 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 demonstrate that the addition of ianalumab (either dose) to standard of care, eltrombopag, prolongs Time to Treatment Failure (TTF) compared to eltrombopag alone in participants with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids
    E.2.2Secondary objectives of the trial
    1. To assess quality, time to and duration of response in each treatment arm
    2. To assess the rate of participants who successfully taper and discontinue eltrombopag in each treatment arm
    3. To assess the incidence and severity of bleeding in each treatment arm
    4. To assess the need for rescue treatments in each treatment group
    5. To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HFRQoL)
    6. To assess B-Cell levels and immunoglobulin (lg) levels
    7. To assess ianalumab pharmacokinetics (PK)
    8. To assess the immunogenicity of ianalumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients aged 18 years and older on the day of signing the informed consent.
    2. A signed informed consent must be obtained prior to participation in the study.
    3. A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy± IVIG.
    4. Patients with platelet count <30 G/L, for whom eltrombopag is clinically indicated as per physician's discretion and with no contraindication to receive eltrombopag.
    Other protocol defined criteria may apply.
    E.4Principal exclusion criteria
    1. ITP patients who received second-line ITP treatments (other than corticosteroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.
    2. Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia
    3. Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
    4. Patients with current or history of life-threatening bleeding
    5. Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAB)-positive. HBcAb positive patients can be enrolled if HBsAg negative, HBV negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given.
    6. Patients with known active or uncontrolled infection requiring systemic treatment during screening period
    7. Patients with hepatic impairment
    8. Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication
    9. Female patients who are pregnant or nursing
    Other protocol defined criteria may apply
    E.5 End points
    E.5.1Primary end point(s)
    Time from randomization date until the first of the following events indicative of treatment failure:
    -platelet count below 30 G/L
    -start of a new ITP treatment
    -need for a rescue treatment
    -ineligibility to taper or inability to discontinue eltrombopag
    -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 participant)
    E.5.2Secondary end point(s)
    1. Percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment
    2. Percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment
    3. Percentage of participants with a best response rate by either response or complete response
    4. Time from randomization to date of first response and time from randomization to date of first complete response
    5. Time from achievement of response to treatment failure
    6. Stable response in each treatment group
    7. Time from achievement of complete response to loss of complete response
    8. Probability to be treatment failure-free (as defined for the primary efficacy endpoint) after the end of treatment period
    9. Percentage of participants reporting bleeding events according to WHO bleeding scale
    10. Number of participants who are in need of rescue treatment in each treatment arm
    11. Percentage of participants who are in need of rescue treatment
    12. The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue in adults.
    13. The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, BotherPhysical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women's Reproductive Health, overall Qol. Each item is rated on a Likert
    type scale. Each scale is scored from O to 100. Higher scores represent better HRQoL
    14. Change from baseline in the frequency (percentage within CD45) and absolute number of CD19+ B-cell counts
    15. Time to B-cell recovery defined as ≥80% of baseline or ≥ 50 cells/µL
    16. Change from baseline in immunoglobulin levels
    17. AUClast: Area under the curve from time zero to the last measurable concentration sampling time (tlast) AUCtau: Area under the curve calculated to the end of a dosing interval
    (tau)
    18. Cmax: Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
    19. Tmax: Time to reach maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
    20. Racc: Accumulation ratio calculated using AUC values obtained after the last and first dose
    21. Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assessing the immunogenicity of ianalumab
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-3,5, 7,9-11,14-16: Randomization to end of study (up to 39 months after randomization of last participant)
    4: Time from randomization up to the longest observed treatment period duration
    6: At 6 months and 1 year
    8: End of planned treatment period
    12,13: From screening (baseline) until end of study (up to 39 months after randomization of last participant)
    17-21: After the first and last dose of study medication
    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 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 trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA28
    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
    Philippines
    Singapore
    Taiwan
    Australia
    China
    India
    Japan
    Korea, Republic of
    Mexico
    Thailand
    Turkey
    United Kingdom
    United States
    Austria
    Belgium
    Czechia
    France
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Romania
    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
    LSLV
    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 months11
    E.8.9.1In the Member State concerned days12
    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: 112
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 38
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 150
    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 Decision2022-10-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-29
    P. End of Trial
    P.End of Trial StatusOngoing
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