Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis
Condition: Iron Overload
MeSH terms: Hemochromatosis; Iron Overload; Hemosiderosis
Intervention: Deferasirox
Intervention description: Deferosirox was provided in tablet forms of 90, 180 and 360mg. Tablets were crushed in the home environment and administered by sprinkling the full dose on to soft food to be consumed immediately.
Intervention MeSH terms: Deferasirox
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 44.0
Sex: All
Minimum age: 2 Years
Maximum age: 6 Years
Healthy volunteers: No
Keywords: Iron Chelation Therapy; Deferasirox; Asunra; Jadenu; Exjade; ICL 670; Pediatric

Summary:

      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.
    

Eligibility criteria:

        Key Inclusion Criteria:

          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.

        Key Exclusion Criteria:

          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.