This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to \<6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.
Who can participate
Age range2 Years – 6 Years
SexALL
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Inclusion criteria
✓. Patients ≥2 to \<6 years old diagnosed with transfusional hemosiderosis
✓. Documented history of red blood cell transfusions
✓. 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.
✓. 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.
✓. 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.
Exclusion criteria
✕. 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.)
✕. Patients continuing on deferoxamine or deferiprone in addition to study treatment. (Patients switching to or continuing on deferasirox are eligible).
✕. Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox.
✕. 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.
✕
What they're measuring
1
Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
. Serum creatinine \> age adjusted ULN measured at any screening visit
✕. 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.
✕. ALT and/or AST \> 2.5 x ULN measured at screening visit 1.
✕. Total bilirubin (TBIL) \>1.5 x ULN measured at screening visit 1.