Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48
Who can participate
Age range2 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Prior to any screening procedures were performed, written informed consent/assent must be provided. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators also obtained assent of patients according to local, regional or national guidelines.
✓. Male and female patient aged ≥ 2 years
✓. Deferasirox naïve patient or chelated naive patient or treated by other chelators for at least 6 months, such as:
✓. Deferiprone/ DFP
✓. Deferoxamine /DFO
✓. Combination (DFO + DFP)
✓. Subject was willing to discontinue current iron chelation therapy at least 5 days prior to study day 1 and for the duration of the study
✓. Patients with transfusion-dependent thalassemia (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of \> 1000 ng/ml at screening and if available, LIC \> 3 mg Fe/g dw within 6 months prior to screening
Exclusion criteria
✕. Creatinine clearance below the contraindication limit in the locally approved prescribing information.
✕. Serum creatinine level \> 1.5 x ULN (upper limit of normal)
✕. AST (SGOT) /ALT (SGPT) \> 5 x ULN, unless LIC confirmed as \>10 mg Fe/dw within 6 months prior to screening visit.
What they're measuring
1
Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)
. Significant proteinuria as indicated by a urinary protein/creatinine ratio \> 0.5 mg/mg in a non-first void urine sample.
✕. Patients with significant impaired gastrointestinal (GI) function or GI disease that might 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 or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol
✕. Patients with a known history of HIV seropositivity (Elisa or Western blot).