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

Sobi, Inc.0 个研究点目标入组 72 人开始时间: 2020年10月28日最近更新: 2022年2月1日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Sobi, Inc.
入组人数: 72

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

性别: All

入选标准

•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/
•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

排除标准

•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.

研究者

发起方

Sobi, Inc.

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