Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet — Jupiter  

Non-transfusion-dependent Thalassemia Clinical Trial

Official title: Open-label, Multicenter, Single Arm, Phase II Study Assessing Treatment Patient Preference for New Deferasirox Formulation (Film-coated Tablet) Compared to the Reference Deferasirox Dispersible Tablet Formulation

Status Completed

Clinical Trial Summary

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

Clinical Trial Description

This was an open-label, multicenter, single arm, phase II study aimed at collecting data on preference for iron chelation therapy in patients with transfusion-dependent thalassemia (TDT) or non-transfusion-dependent thalassemia (NTDT) throughout a 48 week treatment period. Participants who were either chelator-naïve, or who were previously treated with iron chelators (excluding deferasirox) for at least 6 months continuously, were eligible to participate in this study.

The study was divided into 2 phases:

1. Core Phase:

• Screening phase which lasted for a maximum of 4 weeks to determine patient eligibility followed by

• Period 1: Participants were treated with deferasirox DT from Baseline visit Day 1 to Week 24

• Period 2: Participants were treated with deferasirox FCT from Week 25 to Week 48:

At the discretion of the investigator, patients could switch from deferasirox DT to deferasirox FCT at any time during Period 1 of the Core phase, and vice versa, from deferasirox FCT to deferasirox DT at any time during Period 2 of the Core phase. Re-switching treatments was not allowed within each period.

2. Extension Phase:

Participants could continue deferasirox FCT formulation as per the judgment of the investigator, through an extension phase for a maximum of 48 weeks months from the last dose of deferasirox FCT received at the end of period 2 in the Core Phase or until one of the end of study criteria defined is met, whichever came first. Participation in the extension phase was optional.

The end of study was defined as the earliest occurrence of one of the following:

• The patient reached Week 96 in the Extension phase.

• Deferasirox FCT was locally reimbursed for this indication (only applicable for the Extension phase)

• Another clinical study or post-trial access program became available that could continue to provide deferasirox FCT in this patient population and all patients ongoing were eligible to be transferred to that clinical study. ;

Related Conditions & MeSH terms

• Non-transfusion-dependent Thalassemia
• Thalassemia
• Transfusion-dependent Thalassemia

• NCT number: NCT02993224
• Study type: Interventional
• Source: Novartis
• Status: Completed
• Phase: Phase 2
• Start date: July 27, 2017
• Completion date: March 11, 2021