randomized, open-label, multicenter, two arm, phase IIstudy to evaluate treatment compliance, efficacy and safetyof an improved deferasirox formulation (granules) inpediatric patients with iron overload

Novartis Healthcare Private Limited2 个研究点分布在 1 个国家目标入组 240 人开始时间: 2017年11月15日最近更新: 2024年8月27日

研究概览

简要总结

This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin over time of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged â‰¥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, and having a treatment goal to reduce iron burden as measured by serum ferritin. Primary objective of the study is to evaluate patient compliance (using stick pack or tablet counts) and change in serum ferritin over time for both formulations of deferasirox, i.e., granules and dispersible tablet (DT) in iron chelation therapy (ICT) naÃ¯ve patients in the core phase. Male and female children and adolescents aged â‰¥ 2 and < 18 years. Written informed consent/assent before any study-specific procedures. Consent will be obtained from patients, parent(s) or legal guardians. Investigators will also obtain consent/assent of patients according to local guidelines.Patients will be randomized to either Deferasirox granules or Deferasirox DT(Exjade) in the core phase.All patients who choose to continue in the extension phase will receive deferasirox granules in the extension phase.The co-primary efficacy endpoints assessed during the core phase will be Compliance measured by stick pack/tablet count.and Change from baseline in serum ferritin at 48 weeks of treatment.Compliance will be measured by stick pack/tablet count: it will be performed by study personnel every 4 weeks during the core phase based on the amount of medication dispensed, returned and reported as lost/wasted by the patient / caregiver. Serum ferritin test will be performed at Screening Visits 1 and 2 and every 4 weeks from week 5 till end of treatment visit.

研究设计

研究类型: Interventional
分配方式: Computer generated randomization
盲法: Open Label

入排标准

年龄范围: 2.00 Year(s) 至 18.00 Year(s)（—）
性别: All

入选标准

•Written informed consent/assent before any study-specific procedures.
•Consent will be obtained from parent(s) or legal guardians.
•Investigators will also obtain assent of patients according to local guidelines.
•Male and female children and adolescents aged between 2 and 18 years.
•Applicable to core phase only.
•Once in the core phase patients can turn 18 years and still be considered eligible, also for participation in the optional extension phase.
•Any transfusion-dependent anemia associated with iron overload requiring iron chelation therapy and with a history of transfusion of approximately 20 PRBC units, and a treatment goal of reduction, not maintenance of iron burden as measured by serum ferritin.
•Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
•Completion of core phase per protocol (For the optional extension phase criteria only).
•Meeting inclusion criteria as defined in the core phase at Visit 17 except for age requirement (For the optional extension phase criteria only).

排除标准

•Creatinine clearance below the contraindication limit in the locally approved prescribing informationCreatinine clearance will be estimated from serum creatinine (using the Schwartz formula) at screening Visit 1 or screening Visit
•Serum creatinine > 1.5 xULN at screening Visit 1 or screening Visit
•ALT or AST greater than 3.0 x ULN at screening visit 1 or screening visit
•Liver disease with severity of Child-Pugh class B or C.
•Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a second morning urine sample at screening Visit 1 or screening Visit
•Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
•Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive).
•Patients with psychiatric or addictive disorders which prevent them from giving their informed consent/assent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol (including use of electronic devices for ePRO).
•Local access to new formulation (granules or FCT) is available for the patient (For the optional extension phase criteria only).
•Meeting any of the exclusion criteria as defined in the core phase (For the optional extension phase criteria only).