Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment. Provided further assessment of the long-term efficacy and safety of deferasirox in NTDT patients with iron overload (LIC ≥ 5 mg Fe/g liver dw and SF ≥ 300 ng/mL) for up to 260 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
134
Deferasirox dispersible tablets at strengths of 125 mg, 250 mg, and 500 mg were administered by oral daily dosing.
Novartis Investigative Site
Nanning, Guangxi, China
Novartis Investigative Site
Goudi-Athens, GR, Greece
Novartis Investigative Site
Cagliari, CA, Italy
Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment
Time frame: Baseline, 52 weeks
Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw
The percentage of participants with baseline LIC\>15 mg Fe/g dw achieving an LIC \<5 mg Fe/g dw during the study
Time frame: 5 years
Time to Achieving LIC <5 mg Fe/g dw
Time to achieving LIC \<5 mg Fe/g dw for participants with baseline LIC\>15 mg Fe/g dw during the study
Time frame: 5 years
Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period
Time from the target LIC \<3 mg Fe/g dw to the first LIC ≥5 mg Fe/g dw in the follow-up period
Time frame: post-baseline, up to 260 weeks
Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)
The SF-36 is a self-administered questionnaire for adults (from 18 years of age) and contains 36 items which measure: Physical functioning, Role limitation due to physical health problems, Bodily pain, General health perceptions, Vitality, Social functioning, Role limitations due to emotional problems and General mental health . The higher values indicate a better evaluation of health. Range: 0 to 100 \[0 (worst possible health state measured by the questionnaire) to 100 (best possible health state)\].
Time frame: Baseline, 52, 104 & 156 Weeks
Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)
The PedsQL™ is a modular approach to measuring health-related quality of life (HRQOL) in children and adolescents. The 23-item PedsQL™ Generic Core Scales encompass the essential core domains for pediatric HRQOL measurement: 1) Physical Functioning (8 items), 2) Emotional Functioning (5 items), 3) Social Functioning (5 items), and 4) School Functioning (5 items). The Generic Core Scales are designed to enable comparisons across patient and healthy populations. The higher values indicate a better evaluation of health. Range: 0 to 100 \[0 (worst possible health state measured by the questionnaire) to 100 (best possible health state)\].
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Novartis Investigative Site
Milan, MI, Italy
Novartis Investigative Site
Hazmiyeh, Beyrouth, Lebanon
Novartis Investigative Site
Bangkok, Thailand
Novartis Investigative Site
Tunis, Tunisia
Novartis Investigative Site
Adana, Turkey (Türkiye)
Novartis Investigative Site
Istanbul, Turkey (Türkiye)
Novartis Investigative Site
Izmir, Turkey (Türkiye)
...and 1 more locations
Time frame: Baseline, 52, 104 & 156 Weeks
Absolute Change in LIC From Baseline Over Time
Absolute change in serum ferritin from baseline over time up to 260 weeks
Time frame: 24, 52, 76, 104, 128, 156, 180, 208, 232, 260 Weeks
Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)
Correlation between serum ferritin and LIC is assessed using scatter plots with pearson correlation coefficient and simple linear model.
Time frame: Baseline, End of Study (EOS): Week 260 + 30 days follow up
Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)
Correlation for absolute change between LIC and serum ferritin was assessed using scatter plots with pearson correlation coefficient and simple linear model.
Time frame: Week 24, End of Study (EOS): Week 260 + 30 days follow up
Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome
Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment by underlying NTDT syndrome. The 4 underlying disease types: Beta-thalassemia intermedia (N =69), HbE beta-thalassemia (N = 24), Alpha-thalassemia intermedia (HbH disease) (N = 40), Other, specify (N = 1)
Time frame: Baseline, 52 Weeks
Absolute Change in Serum Ferritin From Baseline After 52 Weeks
Absolute change in serum ferritin from baseline after 52 weeks of treatment
Time frame: Baseline, 52 weeks
PK Parameters: AUCtau
The pharmacokinetic parameter, AUCtau was determined using non-compartmental method(s) for deferasirox and its iron complex. AUC=area under the concentration-time curve during a dosing interval at steady state (amount × time × volume).
Time frame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
PK Parameters: Cmax
The pharmacokinetic parameter, Cmax, was determined using non-compartmental method(s) for deferasirox and its iron complex. Cmax (maximum/peak plasma drug concentration after drug administration)=amount × volume
Time frame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
PK Parameters: Tmax
The pharmacokinetic parameter, Tmax, may be determined using non-compartmental method(s) for deferasirox and its iron complex. Tmax=time to reach maximum/peak concentration following drug administration.
Time frame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
Plasma Pharmacokinetics (PK) Deferasirox Concentrations
Blood samples for PK evaluation were collected for a sub-group of patients. The patient had to have been on treatment without dose adjustment or treatment interruption (for any reason) for at least 4 consecutive days prior to scheduled PK sampling visit. If there was a dosage change or interruption within 4 days of the visit, no PK blood samples was collected, and an appropriate comment had to be made on the PK CRF page.
Time frame: Weeks 12 & 24: pre-dose (0hr), 2hr & 4hr post-dose