EUCTR2017-004012-19-BG进行中（未招募）1 期
An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of Rilzabrutinib (PRN1008), an Oral BTK Inhibitor, in Patients with Relapsed Immune Thrombocytopenia
Principia Biopharma, a Sanofi Company0 个研究点目标入组 83 人开始时间: 2017年12月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 83
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
- •1. Male and female patients, aged 18 to 80 years old (Czech Republic
- •and Norway only: aged 18 to 65 years old)
- •2. Immune-related ITP (both primary and secondary)
- •3. Refractory or relapsed patients with no available and approved
- •therapeutic options with a platelet count of count <30,000/µL on two
- •occasions no sooner than 7 days apart in the 15 days before treatment begins
- •4. A history of response (two or more platelet counts = 50,000/µL with
- •an increase of = 20,000/µL) to at least one prior line of therapy (with
- •splenectomy being considered a line of therapy)
- •5. Adequate hematologic, hepatic, and renal function (absolute
- •neutrophil count =1.5 × 109/L, hemoglobin [Hgb] >9 g/dL, AST/ALT =
- •1.5 × ULN, albumin =3 g/dL, total bilirubin =1.5 × ULN, estimated
- •glomerular filtration rate [eGFR] > 60 mL/min (Cockcroft and Gault
- •method) (C1D1 pre-dose may be checked up to Day -3 prior to C1D1)
- •6. Female patients who are of reproductive potential must agree for the duration of active treatment in the study to use a highly effective means of contraception (hormonal contraception methods that inhibits ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal ligation, vasectomized partner, sexual abstinence when this is in line with the preferred and usual lifestyle of the patient). Unless surgically sterile, postmenopausal females should have menopause confirmed by FSH testing
- •7. Able to provide written informed consent and agreeable to the
- •schedule of assessments
- •1. Male or female patients, aged 18 to 80 years old
- •2. Patients with immune-related ITP (both primary and secondary) as
- •defined by current guidelines with at least 3 months duration
- •3. Patients who had a response (achievement of platelet count =
- •50,000/µL) to IVIg/anti-D or corticosteroid that was not sustained and failed at least one other ITP therapy (that was not IVIg or
- •corticosteroid)
- •4. Patients with a platelet count of < 30,000/µL on two occasions no less than 7 days apart in the 15 days before treatment begins, and no
- •platelet count above 35,000/µL on Study Day 1.
- •5. Patients with adequate hematologic, hepatic, and renal function
- •(absolute neutrophil count =1.5 × 109/L, Hgb >9 g/dL, AST/ALT =1.5 ×
- •ULN, albumin =3 g/dL, total bilirubin =1.5 × ULN, eGFR >50 mL/min
- •(Cockcroft and Gault method) (pre-dose may be checked up to Day -3)
- •6. Female patients who are of reproductive potential must agree for the duration of active treatment in the study to use a highly effective means of contraception (hormonal contraception methods that inhibits ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal ligation, vasectomized partner, or true abstinence; when this is in line with the preferred and usual lifestyle of the patient). Unless surgically sterile, postmenopausal females should have menopause confirmed by FSH testing.
- •7. Able to provide written informed consent and agreeable to the
- •schedule of assessments
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 67
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 16
排除标准
- •1. Pregnant or lactating women
- •2. ECG findings of QTcF > 450 msec (males) or > 470 msec (females), poorly controlled atrial fibrillation (i.e., symptomatic patients or a ventricular rate above 100 beats/min on ECG), or other clinically significant abnormalities
- •3. History of current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the trial, with the exception of non-melanoma skin cancer
- •4. Transfusion with blood or blood products or plasmapheresis within 2 weeks before Day 1
- •5. Change in corticosteroid and/or TPO agonist dose within 2 weeks prior to Day 1 (more than 10% variation from Day 1 daily doses)
- •6. Use of rescue medications other than corticosteroids or TPO in Exclusion Criterion #5 in the two weeks before Day 1
- •7. Immunosuppressant drugs other than corticosteroids – these drugs should be discontinued for at least 14 days before Day 1
- •8. Treatment with rituximab or splenectomy within the 3 months prior to Day 1
- •9. Ongoing need for the use of proton pump inhibitor drugs such as omeprazole and esomeprazole (it is acceptable to change patient to H2 receptor blocking drugs prior to Day 1)
- •10. Concomitant use of known strong-to-moderate inducers or inhibitors of CYP3A within 3 days or 5 half-lives (whichever is longer) of Day 1
- •11. Use of CYP3A-sensitive substrate drugs with a narrow therapeutic index within 3 days or 5 half-lives (whichever is longer) of study drug dosing including, but not limited to, alfentanil, astemizole, cisapride, cyclosporine, dihydroergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, tacrolimus, or terfenadine
- •12. Planned or concomitant use of any anticoagulants and platelet aggregation inhibiting drugs such as aspirin, non-steroidal anti-inflammatory
- •drugs NSAIDs, thienopyridenes (within 14 days of planned dosing through end of follow-up
- •13. Has received any investigational drug within the 30 days before receiving the first dose of study medication, or at least 5 times elimination half-life of the drug(whichever is longer); patient should not be using an investigational device at the time of dosing
- •14. Current drug or alcohol abuse
- •15. Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel resection that would preclude adequate study drug absorption
- •16. History of solid organ transplant
- •17. Positive for screening for HIV, hepatitis B (surface antigen and core antibodies unrelated to vaccination), or hepatitis C (anti-HCV antibody confirmed with HCV RNA)
- •18. History of serious infections requiring intravenous therapy within the last 3 months before Day 1
- •19. Clinically significant cognitive dysfunction (= Grade 1) or medical history suggestive of increased risk for cognitive dysfunction during the study
- •20. Live vaccine within 28 days prior to Day 1 or plan to receive one during the study
- •21. Planned surgery in the time frame of the dosing period
- •22. Any other clinically significant disease, condition, or medical history that, in the opinion of the Investigator, would interfere with patient safety, study evaluations, and/or study procedures
- •1. Pregnant or lactating women
- •2. ECG findings of QTcF > 450 msec (males) or > 470 msec (females),
- •poorly controlled atrial fibrillation (i.e., symptomatic patients or a
- •ventricular rate above 100 beats/min on ECG), or other clinically
- •significant abnormalities
- •3. History (within 5 years of SD1) or current, active malignancy requiring or likely to requir
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