2023-509401-71-00招募中3 期
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP).
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 53
- 试验地点
- 16
- 主要终点
- 1. (EU and UK) Proportion of adult participants able to achieve platelet counts at or above 50,000/μL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
研究概览
简要总结
To demonstrate the efficacy of rilzabrutinib versus placebo in patients with refractory/relapsed ITP, based on the durability of platelet response during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Patients will be male and female with primary ITP with duration of >6 months in pediatric participants aged 12 to <18 years (pediatric participants aged 10 to <12 years will be enrolled in the EU [EEA countries] only) and duration of >3 months in ages 18 years and above.
- •Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy.
- •An average of 2 platelet counts at least 5 days apart of <30,000/µL during the Screening period and no single platelet count >35,000/µL, within 14 days prior to the first dose of study drug - Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
- •Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants]).
- •Hemoglobin >9 g/dL within 1 week prior to Study Day
- •All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient’s guardian and agree to the schedule of assessments.
排除标准
- •Patients with secondary ITP.
- •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 - Patients who previously received treatment with Bruton’s Tyrosine Kinase (BTK) inhibitors (except rilzabrutinib) within 30 days before the first dose of study drug are not eligible - Patients who previously received rilzabrutinib at any time are not eligible.
- •History of solid organ transplant.
- •Planned surgery in the time frame of the dosing period.
- •Myelodysplastic syndrome.
- •Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study.
- •Pregnant or lactating women.
- •History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer.
- •Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day
- •Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses).
- •Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer.
- •Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1 - Patients treated with rituximab will have normal B-cell counts prior to enrollment.
结局指标
主要结局
1. (EU and UK) Proportion of adult participants able to achieve platelet counts at or above 50,000/μL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
1. (EU and UK) Proportion of adult participants able to achieve platelet counts at or above 50,000/μL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
次要结局
- 4. Proportion of paticipants requiring rescue therapy during the 24-week blinded treatment period.
- 1. Number of weeks with platelet count ≥50,000/μL OR between ≥30,000/μL and <50,000/μL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.
- 2. Number of weeks with platelet counts ≥30,000/μL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.
- 3. Time to first platelet count of ≥50,000/μL OR between ≥30,000/μL and <50,000/μL and doubled from baseline.
- 5. Change from baseline on Item 10 of the ITPPatient Assessment Questionnaire in adult patients (≥18 years) at Week 13.
- 6. (EU and UK) Change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25.
- 7. Proportion of participants who able to achieve stable platelet response, within a period of 24 weeks following initial achievement of the platelet response.
- 8. Frequency and severity of Treatment Emergent Adverse Events.
- 9. Frequency and severity of bleeding TEAEs.
- 10. Plasma concentrations of rilzabrutinib.
- 11. Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult patients (≥18 years).
- 12. Change from baseline in disease-specific QoL as measured by the Kids’ ITP Tools (ITP-KIT) score in pediatric participants.
研究者
Clinical Sciences and Operations
Scientific
Principia Biopharma Inc.
研究点 (16)
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