NCT02993224

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

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 148 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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

Detailed 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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Deferasirox dispersable tablet (DT)	Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country. For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT. For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone). Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water.
DRUG	Deferasirox film coated tablet (FCT)	Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT. Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).

Timeline

• Start date: 2017-07-27
• Primary completion: 2020-01-29
• Completion: 2021-03-11
• First posted: 2016-12-15
• Last updated: 2021-10-04
• Results posted: 2021-10-04

Locations

17 sites across 7 countries: Egypt, Lebanon, Morocco, Saudi Arabia, Thailand, Turkey (Türkiye), Vietnam