LBCTR2019030206已完成4 期
A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets
ovartis Pharma Services Inc.0 个研究点目标入组 3 人开始时间: 2021年2月6日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- N/a: Single Arm Study
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 2 至 6（—）
- 性别
- All
入选标准
- •1.Patients =2 to <6 years old diagnosed with transfusional hemosiderosis
- •2.Documented history of red blood cell transfusions
- •3.Written informed consent/assent before any study-specific procedures. The consent will be obtained from caregiver(s) or patient's legal representative. Investigators will also obtain assent of patients according to local, regional, or national regulations.
- •4.For patients on prior DFX: Serum ferritin (SF) >500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT = 7mg/kg/day.
- •5.For patients on a prior chelator other than DFX (e.g. deferiprone or deferoxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and 2.
排除标准
- •1.Patients that receive more than one iron chelator at the same time as current iron chelation treatment. (Patients who have received combination therapy in their medical history but are currently being treated with a single ICT agent are eligible.)
- •2.Patients continuing on deferoxamine or deferiprone in addition to study treatment.
- •(Patients switching to or continuing on deferasirox are eligible).
- •3.Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox.
- •4.Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void sample urine measured at screening visit 1.
- •5.Serum creatinine > age adjusted ULN measured at any screening visit
- •6.Creatinine clearance below 90 mL/minute measured at any screening visit. Creatinine clearance using the Schwartz formula will be estimated from serum creatinine measured at each respective visit.
- •7.ALT and/or AST > 2.5 x ULN measured at screening visit 1.
- •8.Total bilirubin (TBIL) >1.5 x ULN measured at screening visit 1.
- •9.Patients with significant impaired GI function or GI disease that may significantly alter the absorption of oral deferasirox FCT (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- •10.History of and/or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive.
- •11.Liver disease with severity of Child-Pugh Class B or C.
- •12.History of hypersensitivity to any of the study drug or excipients.
- •13.Patients participating in another clinical trial or receiving an investigational drug.
- •14.Patients with a known history of HIV seropositivity.
- •15.Patients unwilling or unable to comply with the protocol.
- •16.History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
- •17.Significant medical condition interfering with the ability to partake in this study (e.g.
- •uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease: cardiovascular, renal, hepatic, etc.).
- •18.Female patients who reach menarche and they or their caregivers refuse pregnancy testing and/or if there is a positive pregnancy test result.
研究者
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