EUCTR2020-005736-30-IT招募中1 期
A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll). - CDAII-LUSPATERCEPT
FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia0 个研究点目标入组 40 人开始时间: 2023年2月6日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •- Males or female patients >= 18 years of age at the time of signing the informed consent document (ICF).
- •- Subject must understand and voluntarily sign an informed consent form (ICF) prior to any study-related assessments/procedures being conducted.
- •- Biallelic causative mutations in SEC23B gene alone or associated with other gene variants (e.g., globin gene defects).
- •- Monoallelic causative mutations in SEC23B gene in presence of hypoglycosylation of band 3 and/or associated with other gene variants (e.g., globin gene defects).
- •- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
- •- Non-transfusion dependent (NTD) patients, defined as having received < 1/0 unit of RBCs within 8 weeks prior to Cycle 1 Day 1.
- •- Or transfusion dependent patients, defined as requiring ³ 3 units of RBCs every 8 weeks (confirmed over 6 months prior to Cycle 1 Day 1).
- •- Anemia, defined as: (i) mean Hb concentration < 10.9 g/dl of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed during the screening period) in non-transfusion dependent patients, or (ii) transfusion dependent.
- •- Splenectomized or non-splenectomized patients are both eligible.
- •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 35
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. Any clinically significant pulmonary (including pulmonary hypertension), cardiovascular, endocrine, neurologic, hepatic (including cirrhosis and liver failure), gastrointestinal, infectious, immunological (including clinically significant allo- or auto-immunization) or genitourinary disease considered by the investigator as not adequately controlled prior to Cycle 1 Day 1.
- •2. Patients with history of cancer, or suffering from evolutive cancer, or in remission with treatment stopped less than 2 years before.
- •3. Anemia from other causes.
- •4. Symptomatic splenomegaly.
- •5. Thromboembolic events = grade 3 according to the National Cancer lnstitute-Common Terminology Criteria for Adverse Events (NCl-CTCAE) v.4 .0 (current active minor version) within 12 months prior to Cycle 1 Day 1.
- •6. Ejection fraction < 50% by echocardiogram, MUGA or cardiac MRI.
- •7. Uncontrolled hypertension defined as systolic blood pressure (BP) = 150 mm Hg or diastolic BP =95 mm Hg.
- •8. QTc > 450 msec on screening ECG.
- •9. Proteinuria = Grade 2.
- •10. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
- •11. Treatment with another investigational drug or device, or approved therapy for investigational use 28 days prior to Cycle 1 Day 1, or if the half-life of the previous investigational product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
- •12. Transfusion event within 7 days prior to Cycle 1 Day 1.
- •13. Splenectomy within 56 days prior to Cycle 1 Day 1.
- •14. Major surgery (except splenectomy) within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1.
- •15. lron chelation therapy initiated within 56 days prior to Cycle 1 Day 1.
- •16. Cytotoxic agents, systemic corticosteroids, immunosuppressants, or anticoagulant therapy such as warfarin or heparin within 28 days prior to Cycle 1 Day 1 (prophylactic aspirin up to 100 mg/d is permitted).
- •17. Evidence of active hepatitis C (HCV) infection, or active infectious hepatitis B, or known positive human immunodeficiency virus (HIV)
研究者
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