Safety and Efficacy of Deferasirox in Patients With Trans... | NCT01033747

NCT01033747

Protocol Section

Identification Module

Results Section

Participant Flow Module

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Status Module

Description Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Deferasirox	Experimental	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	Drug: Deferasirox
Deferasirox Crossover	Experimental	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	Drug: Deferasirox

Name	Type	Description	Arm Group Labels	Other Names
Deferasirox	Drug	10 mg/kg or 30 mg/kg orally daily	Deferasirox
Deferasirox

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	Baseline to 7 Years

Secondary Outcomes

Measure	Description	Time Frame
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.

Eligibility Module

Contacts/Locations Module

Name	Affiliation	Role
Novartis Pharmaceuticals	Novartis Pharmaceuticals	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Novartis Investigative Site		Cagliari			Italy
Novartis Investigative Site

Arm/Group Information

ID	Title	Description
FG000	Deferasirox	Deferasirox group consists of all participants who were initially randomized to 10 mg/kg or 20 mg/kg deferasirox orally daily in the main study and remained on deferasirox treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative extension study
FG001	Deferasirox Crossover	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 5 mg to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study

Periods

Title

Milestones

Reasons Not Completed

Overall Study

Type	Comment	Milestone Data
STARTED		FG00051 subjects FG00119 subjects
COMPLETED		FG00014 subjects FG0014 subjects
NOT COMPLETED		FG00037 subjects FG00115 subjects

Type	Comment	Reasons
Adverse Event		FG0009 subjects FG0014 subjects
Abnormal Laboratory Value		FG0001 subjects FG001

Baseline Characteristics Module

Arm/Group Information

ID	Title	Description
BG000	Deferasirox	Deferasirox group consists of all participants who were initially randomized to 10 mg/kg or 20 mg/kg deferasirox orally daily in the main study and remained on deferasirox treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative extension study
BG001

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age Continuous			Mean

Outcome Measures Module

Outcome Measures

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

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.

All participants in the full analysis set treated with deferasirox for more than 3.5 years.

Posted

Mean

Full Range

Percent change

Baseline to 7 Years

ID	Title	Description
OG000	Deferasirox	Deferasirox group consists of all participants who were initially randomized to 10 mg/kg or 20 mg/kg deferasirox orally daily in the main study and remained on deferasirox treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative extension study

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Deferasirox	Deferasirox group consists of all participants who were initially randomized to 10 mg/kg or 20 mg/kg deferasirox orally daily in the main study and remained on the same deferasirox treatment during the comparative prolongation study(NCT00379483)and at the beginning of the 5-year non-comparative extension study

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Extramedullary haemopoiesis	Blood and lymphatic system disorders	MedDRA	Systematic Assessment

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA	Systematic Assessment

Title	Organization	Phone	Extension	Email
Study Director	Novartis Pharmaceuticals	862 778-8300

Condition Browse Module

ID	Term
D019190	Iron Overload
D017086	beta-Thalassemia

ID	Term
D019189	Iron Metabolism Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D013789	Thalassemia

Intervention Browse Module

ID	Term
D001565	Benzoates
D000146	Acids, Carbocyclic
D002264	Carboxylic Acids
D009930	Organic Chemicals