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

Primary Purpose

Liver Iron Overload

Status
Completed
Phase
Phase 2
Locations
Italy
Study Type
Interventional
Intervention
Deferasirox
Deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Liver Iron Overload focused on measuring iron overload, iron chelation therapy, B-thalassemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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.

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Deferasirox

Deferasirox Crossover

Arm Description

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 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

Outcomes

Primary Outcome Measures

The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
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

Relative Change in Serum Ferritin From 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.

Full Information

First Posted
October 16, 2009
Last Updated
July 18, 2011
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01033747
Brief Title
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
Official Title
A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
July 2011
Overall Recruitment Status
Completed
Study Start Date
February 2003 (undefined)
Primary Completion Date
January 2008 (Actual)
Study Completion Date
January 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Liver Iron Overload
Keywords
iron overload, iron chelation therapy, B-thalassemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
70 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Arm Description
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
Arm Title
Deferasirox Crossover
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Intervention Description
10 mg/kg or 30 mg/kg orally daily
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Intervention Description
5 mg/kg or 30 mg/kg orally daily
Primary Outcome Measure Information:
Title
The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
Description
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.
Time Frame
Baseline to 7 Years
Secondary Outcome Measure Information:
Title
Relative Change in Serum Ferritin From Baseline to 3.5 Years
Description
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.
Time Frame
Baseline to 3.5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Cagliari
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
Country
Italy
Facility Name
Novartis Investigative Site
City
Milan
Country
Italy
Facility Name
Novartis Investigative Site
City
Torino
Country
Italy

12. IPD Sharing Statement

Learn more about this trial

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

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