A randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance, efficacy and safety of an improved deferasirox formulation (granules) in pediatric patients with iron overload

ovartis Pharma Services Inc.0 个研究点目标入组 23 人开始时间: 2024年7月22日最近更新: 2024年8月19日

适应症

试验速览

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•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 = 2 and < 18 years. [France: Male and female children and adolescent aged = 2 and < 18 years old, however children aged = 2 and = 6years can be enrolled only when deferoxamine treatment is contraindicated or inadequate in these patients as per investigator decision. 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 to reduce iron burden (300mL PRBC = 1 unit in adults whereas 4 ml/kg PRBC is considered 1 unit for children).
•Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
•Patient has to have participated and completed the 48 weeks core phase treatment as per protocol (For optional extension phase eligibility only).

•Creatinine clearance below the contraindication limit in the locally approved prescribing information (using Schwartz formula) at screening visit 1 or screening visit 2.
•Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and or screening Visit 2
•ALT and/or AST > 3.0 x ULN at screening visit 1 or screening visit 2.
•(Criterion no longer applicable, removed as part of Amendment 1): Prior iron chelation therapy.
•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 2.
•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).
•Other protocol-defined Inclusion/Exclusion may apply.

发起方

ovartis Pharma Services Inc.