Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

Phase 2

Completed

• Conditions: Liver Iron Overload
• Interventions: Drug: Deferasirox

• Registration Number: NCT01033747
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.

Detailed Description

Not available

Study Timeline

• Study Start: 2003-02
• Primary Completion: 2008-01
• Study Completion: 2008-01
• Study First Submitted: 2009-10-16
• Study First Submitted QC: 2009-12-15
• Study First Posted: 2009-12-16
• Results First Submitted: 2010-12-21
• Status Verified: 2011-07
• Results First Submitted QC: 2011-07-18
• Last Update Submitted: 2011-07-18
• Results First Posted: 2011-08-11
• Last Update Posted: 2011-08-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

• Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
• Patients currently participating in the food-effect sub-study, according to amendment 3.
• Ability to provide written informed consent prior to participation in this non-comparative extension study.
• Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
• Body weight of at least 35 kg.

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Exclusion Criteria

• Pregnant or breastfeeding patients.
• History of non-compliance to medical regimens and patients who are considered potentially unreliable.
• Proteinuria > 300 mg/L second void morning urine.
• Patients with serum creatinine above the upper limit normal.

Other protocol-defined inclusion/exclusion criteria may apply.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study
Deferasirox Crossover	Deferasirox	Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study

Primary Outcome Measures

Name	Time	Method
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox	Baseline to 7 Years	The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.

Secondary Outcome Measures

Name	Time	Method
Relative Change in Serum Ferritin From Baseline to 3.5 Years	Baseline to 3.5 years	The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇮🇹 Torino, Italy