CompletedPhase 4

Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia

China, Greece, Italy134 participantsStarted 2012-12-06

Plain-language summary

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.

Who can participate

Age range10 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 \</= 40 ml/min, serum creatinine \> ULN, ALT \>5 x ULN, active hepatitis B or C, cirrhosis Pediatrics Only: A patient's weight of at least 20 kg is required to allow dosing of 5 mg/kg with one tablet of 125 mg

What they're measuring

Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline

Timeframe: Baseline, 52 weeks

Trial details

NCT IDNCT01709838

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-01-03

Results submitted2019-07-17

View on ClinicalTrials.gov More Non-transfusion Dependent Thalassemia trials