NCT03372083

Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis

A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 44 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 6 Years
• Healthy volunteers: Not accepted

Summary

This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to \<6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.

Detailed description

The study included a screening period (from Day 0-14) with two visits at least 7 days apart to assess eligibility of patients that were chelation naïve or on a prior iron chelator treatment other than DFX. For Patients on DFX treatment prior to study entry only one screening visit (screening visit 1) were to occur to determine eligibility. Any current chelation therapy except deferasirox were to be discontinued to undergo a 5-day washout period prior to commencing a 24 week treatment period with crushed deferasirox FCT. All patients were to have weekly visits for the first month to monitor renal function. Hepatic function were to be assessed biweekly during the first month. Thereafter, monthly safety assessments were to be performed, including the monitoring of serum ferritin values and trends in order to adapt patient treatment. Eligibility, application of dosing standards and adjustments, as well as safety and serum ferritin assessments as specified in the protocol. The planned duration of treatment was 24 weeks followed by a 30-day safety follow up.

Conditions

• Iron Overload

Interventions

Type	Name	Description
DRUG	Deferasirox	Deferosirox was provided in tablet forms of 90, 180 and 360mg. Tablets were crushed in the home environment and administered by sprinkling the full dose on to soft food to be consumed immediately.

Timeline

• Start date: 2018-01-16
• Primary completion: 2019-12-05
• Completion: 2019-12-05
• First posted: 2017-12-13
• Last updated: 2020-08-25
• Results posted: 2020-08-03

Locations

10 sites across 7 countries: Egypt, Italy, Lebanon, Oman, Thailand, United Arab Emirates, United Kingdom