NCT02435212

Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients With Iron Overload

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 224 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This was a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox dispersible tablet (DT) formulation in children and adolescents aged ≥ 2 and \< 18 years at enrolment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden. Randomization was stratified by age groups (2 to \<10 years, 10 to \<18 years) and prior iron chelation therapy (Yes/ No). There were two study phases which include a 1 year core phase where participants were randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all participants received the granules up to 5 years. Participants who demonstrated benefit to granules or DT in the core phase, and/or expressed the wish to continue in the optional extension phase on granules, were offered this possibility until there was local access to the new formulation (granules or film-coated tablet (FCT)) or up to 5 years, whichever occurred first.

Conditions

• Transfusion-dependent Anemia

Interventions

Type	Name	Description
DRUG	Deferasirox granule formulation	Deferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use and will be administered based on body weight.
DRUG	Deferasirox DT formulation	Deferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use and will be administered based on body weight.

Timeline

• Start date: 2015-10-21
• Primary completion: 2018-05-31
• Completion: 2024-01-15
• First posted: 2015-05-06
• Last updated: 2024-09-19
• Results posted: 2024-09-19

Locations

43 sites across 17 countries: United States, Belgium, Bulgaria, Egypt, France, Hungary, India, Italy, Lebanon, Malaysia, Oman, Panama, Philippines, Russia, Thailand, Tunisia, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study