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    Summary
    EudraCT Number:2016-002814-29
    Sponsor's Protocol Code Number:CETB115E2403
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-03-29
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-002814-29
    A.3Full title of the trial
    SOAR, Interventional phase II single-arm study to assess efficacy and safety of eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia with follow-up up to 24-months.
    A.3.2Name or abbreviated title of the trial where available
    SOAR
    A.4.1Sponsor's protocol code numberCETB115E2403
    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 AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressLichtstrasse 35
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number +44 61 324 1111
    B.5.5Fax number +44 61 324 8001
    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 Yes
    D.2.1.1.1Trade name REVOLADE
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameEltrombopag
    D.3.2Product code ETB115
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeETB115
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number12.5 to 75
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    First-line severe aplastic anaemia
    E.1.1.1Medical condition in easily understood language
    First-line severe aplastic anaemia
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10002967
    E.1.2Term Aplastic anaemia
    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 evaluate the efficacy of eltrombopag + cyclosporine as first-line therapy on overall hematologic response (partial and complete response) by 6 months
    E.2.2Secondary objectives of the trial
    1: Evaluate the effect of eltrombopag + cyclosporine on overall hematologic response (partial and complete response):
    ● by 3 months
    ● at 12 months
    ● at 24 months
    The following secondary objectives will be assessed by 6 months and 24 months (responders only) as appropriate and will be reported in a cumulative basis
    2: Evaluate the duration of hematologic response
    3: Evaluate the proportion of patients who relapse
    4: Evaluate the clonal evolution to myelodysplasia, paroxysmal nocturnal hemoglobinuria (PNH), and leukemia
    5: Evaluate the red blood cell (RBC) transfusion independence
    6: Evaluate the platelet transfusion independence
    7: Evaluate the longest interval without platelet or RBC transfusion
    8: Evaluate the effect of ETB and CsA on patient symptoms and health related quality of life
    9: Evaluate the safety and tolerability of ETB + CsA
    10: Characterize the PK of ETB when combined with CsA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patient has signed the Informed Consent (ICF) prior to any screening procedures being performed.
    2.Patient is male/female ≥18 years old at the time of informed consent and able to swallow a tablet.
    3.Patient has SAA characterized by:
    a.Bone marrow cellularity <30% (excluding lymphocytes) and
    b.At least two of the following (peripheral blood):
    •Absolute neutrophil count <500/ µL
    •Platelet count <20,000/ µL
    •Absolute reticulocyte count <60,000/ µL
    4.Normal ECG defined as the following as determined via the mean of a triplicate ECG
    • Resting heart rate
    6-<12 years: 60-130 bpm
    12-<18 years: 60-120 bpm
    ≥18 years: 50-90 bpm
    •QTcF at screening <450 msec (male patients), 460 msec (female patients)
    E.4Principal exclusion criteria
    1.Diagnosis of Fanconi anemia.
    2.Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review.
    3.Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
    4. a. Hypersensitivity to eltrombopag or cyclopsorine or their components.
    b. Contraindications to cyclosporine
    5.AST or ALT >3 x ULN.
    6.Serum creatinine, total bilirubin, and alkaline phosphatase >1.5 x ULN.
    7.Patient with liver cirrhosis.
    8.a. Infection not adequately controlled with appropriate therapy.
    8.b. Patients who are human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients are allowed to be enrolled.
    9.Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to consent, be compliant with study
    procedures, tolerate protocol therapy, or patients with less than 3 months of life expectancy.
    10.Patients with cancer who are not considered cure, are on active chemotherapeutic treatment or who take drugs with hematological effects.
    11.Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
    12.Pregnancy statements and contraception requirements:
    Pregnancy or nursing (lactating) women
    Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they are using highly effective methods of contraception during dosing and for 3 months after stopping medication.
    In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
    13.Not able to understand the investigation nature of the study or to give informed consent.
    14.Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
    15.Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
    16.Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that cannot be discontinued or replaced by safe alternative medication per www.qtdrugs.org.
    17. ECOG performance status of ≥2.
    18. Patients under the age of 40 must be referred for consideration of allogeneic bone marrow transplantation (HSCT) if (human leukocyte antigen) HLA matching has been done and a suitable matched sibling donor is available and the patient is willing to undergo transplantation (i.e. patients who do not have a HLA match or are not medically fit, not willing or unable to undergo transplantation will be considered for enrollment).
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective of the study is to evaluate the eltrombopag + cyclosporine as first-line therapy as assessed by the overall hematologic response rate by 6 months. Bayesian approach will be used for analysis of the primary endpoint. The primary analysis will be based on the calculation of observed overall hematologic response rate by 6 months and its posterior distribution using a beta-binomial model. Combination therapy of eltrombopag + cyclosporine will be declared efficacious if the following criteria are met:
    a. Observed hematologic ORR ≥ "clinically meaningful" threshold (30%)
    b. Probability of true ORR "not being clinically meaningful (response ≤ 20%) is less than 10%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Overall hematologic response rate by 6 months
    E.5.2Secondary end point(s)
    Final analysis will be done after all the patients enrolled have completed 24 months of treatment with cyclosporine or discontinued treatment with cyclosporine prior to 24 months.
    E.5.2.1Timepoint(s) of evaluation of this end point
    hematologic response rate by 3 months, at 12 months and at 24 months.
    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 Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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.8.2.4Number of treatment arms in the trial1
    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 EEA10
    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
    Brazil
    Hong Kong
    Hungary
    India
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    Spain
    Thailand
    Turkey
    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
    LVLS
    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 months7
    E.8.9.1In the Member State concerned days21
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 17
    F.4.2.2In the whole clinical trial 50
    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)
    Second treatment line for SAA
    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 Decision2017-03-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-05-30
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