Overview

This trial is active, not recruiting.

Condition non-transfusion dependent thalassemia
Treatment icl670 deferasirox
Phase phase 4
Sponsor Novartis Pharmaceuticals
Start date December 2012
End date January 2019
Trial size 134 participants
Trial identifier NCT01709838, 2012-000650-64, CICL670E2419

Summary

To assess 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. To provide 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Other)
one arm, deferasirox, LIC based dose titration
icl670 deferasirox Exjade
deferasirox

Primary Outcomes

Measure
Change in liver iron concentration
time frame: baseline, 52 weeks

Secondary Outcomes

Measure
Medical Outcomes Study Short Form-36
time frame: baseline, up to 156 weeks
Pediatric Quality of Life Questionnaires
time frame: baseline, up to 156 weeks
Change in liver iron concentration
time frame: baseline, up to 260 weeks
Correlation between serum ferritin and liver iron concentration
time frame: baseline, up to 260 weeks
Change in serum ferritin
time frame: baseline, up to 260 weeks
Endocrine function laboratory parameters
time frame: baseline, up to 260 weeks
PK parameters
time frame: up to 24 weeks
Adverse events
time frame: up to 260 weeks
Change in liver iron concentration
time frame: every 24 weeks following baseline to week 260
Change in liver iron concentration
time frame: every 24 weeks from week 52 to week 260
Change in liver iron concentration
time frame: every 24 weeks, from baseline to week 260

Eligibility Criteria

Male or female participants at least 10 years old.

Inclusion Criteria: Non-transfusion dependent congenital or chronic anemia inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease)/ Liver iron concentration >/= 5 mg Fe/g dw Serum Ferritin >/= 300 ng/mL - Exclusion Criteria: HbS-beta Thalassemia, anticipated regular transfusion program during the study, blood transfusion 6 months prior to study start, significant proteinuria, creatinine clearance ULN, ALT >5 x ULN, active hepatitis B or C, cirrhosis

Additional Information

Official title An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia
Description To assess the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks. Also, to evaluate the impact of deferasirox on the quality of life for adults and pediatric patients; correlate changes in serum ferritin and LIC, evaluate efficacy changes in NTDT syndrome, evaluate higher doses of deferasirox, assess endocrine function and examine PK parameters.To also assess the efficacy of treatment in patients with very high LIC (>15 mg Fe/g dw) at baseline and those who need re-treatment after having reached the target LIC < 3 mg Fe/g dw during the study.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Novartis.