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    Summary
    EudraCT Number:2020-003232-24
    Sponsor's Protocol Code Number:AVA-PED-301
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-27
    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 ConcernedGermany - BfArM
    A.2EudraCT number2020-003232-24
    A.3Full title of the trial
    A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia
    A.4.1Sponsor's protocol code numberAVA-PED-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04516967
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/373/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSobi, Inc.
    B.1.3.4CountryUnited States
    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 supportSobi, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSobi, Inc.
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street Address77 4th Ave, 3rd Floor
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post codeMA 02451
    B.5.3.4CountryUnited States
    B.5.4Telephone number+17817867370
    B.5.6E-mailmedinfo.us@sobi.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 Doptelet 20 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderDova Pharmaceuticals Ireland Limited
    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 nameavatrombopag
    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 INNAvatrombopag maleate
    D.3.9.1CAS number 677007-74-8
    D.3.9.2Current sponsor codeER-875039-1
    D.3.9.3Other descriptive nameAVATROMBOPAG MALEATE
    D.3.9.4EV Substance CodeSUB120722
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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.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 nameavatrombopag
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNAvatrombopag maleate
    D.3.9.1CAS number 677007-74-8
    D.3.9.2Current sponsor codeER975039-1
    D.3.9.3Other descriptive nameAVATROMBOPAG MALEATE
    D.3.9.4EV Substance CodeSUB120722
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for oral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Thrombocytopenia in paediatric subjects with immune thrombocytopenia for ≥6 months duration who have had an insufficient response to a previous treatment
    E.1.1.1Medical condition in easily understood language
    Low platelet counts
    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 HLT
    E.1.2Classification code 10043555
    E.1.2Term Thrombocytopenias
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083843
    E.1.2Term Primary immune thrombocytopenia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLGT
    E.1.2Classification code 10035534
    E.1.2Term Platelet disorders
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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 efficacy of avatrombopag is superior to placebo for the treatment of pediatric subjects with ITP of ≥6 months duration who have had an insufficient response to a previous treatment
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of avatrombopag
    • To evaluate the PK and PD of avatrombopag
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For the CORE phase:
    1. Male or female subjects ≥1 and <18 years of age at Screening and Baseline.
    2. Subject and/or subject’s legally authorized representative (LAR) must be able to provide informed consent and/or assent, as applicable.
    3. Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP (Provan, 2019) for ≥6 months duration and has had an insufficient response to a previous treatment, in the opinion of the Investigator.
    4. Subject has an average of 2 platelet counts <30×109/L with no single count >35×109/L.
    The platelet count obtained at the Screening Visit/Visit 1 and 1 other platelet count taken within 28 days on either side of the Screening Visit (may use a historical value collected per standard of care if within 28 days of Screening) will be averaged to obtain the study eligibility platelet count value, which must be <30×109/L. The 2 samples must be obtained ≥24 hours and ≤28 days apart and the results must be available prior to randomization.
    5. Subjects being treated chronically with corticosteroids or azathioprine/6-mercaptopurine must be receiving a stable dose for at least 30 days prior to Day 1/Visit 2 or must have completed these therapies more than 30 days prior to Day 1/Visit 2.
    6. Subjects being treated with mycophenolate mofetil (MMF), cyclosporine (CsA), sirolimus, or danazol must be receiving a stable dose for at least 90 days prior to Day 1/Visit 2 or must have completed these therapies more than 30 days prior to Day 1/Visit 2.
    7. Previous therapy for ITP with immunoglobulins (IVIg and anti-D) or corticosteroid rescue therapy must have been completed at least 14 days prior to Day 1/Visit 2.
    8. Cyclophosphamide and vinca alkaloid regimens must have been completed at least 30 days prior to Day 1/Visit 2.
    9. Splenectomy and rituximab must have been completed at least 90 days prior to Day 1/
    Visit 2.
    10. Previous therapy with any other TPO-RAs (e.g., eltrombopag or romiplostim) or recombinant human TPO must have been completed 28 days prior to Day 1/Visit 2.
    11. Previous therapy with vitamin K antagonists, antifibrinolytic agents, recombinant activated factor VII, heparin, factor Xa inhibitors, direct thrombin inhibitors, desmopressin, or chronic antiplatelet therapy must have been completed within 7 days of Day 1/Visit 2.
    12. Previous therapy with moderate or strong dual inducers or moderate or strong dual inhibitors of cytochrome P450 (CYP)2C9 and CYP3A4 must have been completed within 7 days of Day1/Visit 2.
    13. Platelet transfusion, or receipt of blood products containing platelets must have been completed within 7 days of Day 1/Visit 2. Packed red blood cells (RBCs) are permitted.
    14. Females of childbearing potential must have a negative urine or serum pregnancy test at Screening and Day 1/Visit 2 and must not be breastfeeding.
    15. Female subjects of childbearing potential and who are sexually active and male subjects who are sexually active must agree to use highly effective methods of contraception.
    16. Subject and/or subject’s LAR is willing and able to comply with all aspects of the protocol.

    For the EXTENSION phase:
    1. Subject and/or the LAR must provide consent and/or assent, as applicable, to continue into the open-label Extension Phase. The consent for the Extension Phase will be part of the consent for the Core Phase.
    2. Completed 12 weeks of treatment in the Core Phase or discontinued the Core Phase early due to lack of treatment effects.
    3. Female subjects of childbearing potential and who are sexually active and male subjects who are sexually active must agree to use highly effective methods of contraception.
    4. Subject and/or the subject’s LAR is willing and able to comply with all aspects of the protocol.
    E.4Principal exclusion criteria
    For the CORE phase:
    1. Known secondary ITP.
    2. Body Mass Index (BMI) >30 kg/m2 or >95% for age.
    3. Any history of arterial or venous thrombosis, including partial or complete thrombosis.
    4. Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
    5. History of myelodysplastic syndrome (MDS).
    6. Known history of congenital heart abnormalities or arrhythmias.
    7. History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
    8. Known history of disseminated intravascular coagulation (DIC), hemolytic uremic syndrome (HUS), or thrombotic thrombocytopenic purpura (TTP).
    9. Subjects with Evans syndrome.
    10. Concurrent malignant disease or previous history of myeloid hematologic malignancies.
    11. Hemoglobin (Hgb) levels ≤9 g/dL in ages ≥1 year to <6 years and ≤8 g/dL in ages ≥6 to <18 years.
    12. Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2.
    13. Serum total bilirubin >1.5× the upper limit of normal (ULN) for age, alanine transaminase (ALT) and aspartate aminotransferase (AST) >3× the ULN for age.
    14. Known allergy to avatrombopag or any of its excipients.
    15. Subject is unable to take oral medication or has a malabsorption syndrome, or has known hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption or any other uncontrolled gastrointestinal condition.
    16. Enrollment in another clinical study with any investigational drug or device within 30 days of Day 1/Visit 2 (or 5 half-lives, whichever is longer); however, participation in observational studies within the previous 30 days is permitted.
    17. Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
    18. Considered unable or unwilling to comply with the study protocol requirements.

    For the EXTENSION phase:
    1. Significant safety or tolerability concerns with the subject’s participation, in the opinion of the Investigator.
    2. Subjects requiring the following drugs or procedures at the time of enrollment into the Extension Phase:
    • Rituximab
    • Other TPO-RAs
    • Splenectomy
    • Moderate or strong dual inducers or moderate or strong dual inhibitors of CYP2C9 and CYP3A4.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    Durable platelet response as defined by the proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×109/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase in the absence of rescue medication.

    Alternative Primary Efficacy Endpoint:
    Platelet response as defined by the proportion of subjects for whom at least 2 consecutive platelet assessments are ≥50×109/L over the 12 week Treatment Period in the Core Phase in the absence of rescue medication.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 12 week Treatment Period in the Core Phase
    E.5.2Secondary end point(s)
    • The percentage of weeks subjects have a platelet count ≥50×109/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
    • Platelet response at Day 8 (defined by the proportion of subjects with a platelet count ≥50×109/L at Day 8, in the absence of rescue therapy).
    • The percentage of weeks subjects have a platelet count between ≥50×109/L and ≤150×109/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
    • The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
    • Incidence and severity of bleeding symptoms associated with ITP measured using the WHO Bleeding Scale.
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 12 week Treatment Period in the Core Phase
    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 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) 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 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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    Ukraine
    Russian Federation
    United Kingdom
    United States
    France
    Germany
    Hungary
    Poland
    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 years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months5
    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: 72
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 38
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 28
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 22
    F.4.2.2In the whole clinical trial 72
    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)
    Standard care
    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 Decision2020-11-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-11
    P. End of Trial
    P.End of Trial StatusCompleted
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