Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

This study has been completed.

Sponsor:

Information provided by:

Novartis

ClinicalTrials.gov Identifier:

NCT00303329

First received: October 14, 2005

Last updated: April 15, 2011

Last verified: April 2011

History of Changes

Results First Received: December 15, 2010

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Anemia Hemosiderosis
Intervention:	Drug: Deferasirox

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

Participant Flow: Overall Study

	β-thalassemia Patients	Rare Anemias Patients
STARTED	85	99
COMPLETED	50	37
NOT COMPLETED	35	62
Adverse Event	7	14
Abnormal Laboratory Values	0	1
Unsatisfactory therapeutic effect	9	2
Condition no longer requires study drug	0	8
Protocol Violation	3	0
Withdrawal by Subject	9	15
Lost to Follow-up	0	1
Administrative problems	1	0
Death	2	12
Stopped at end of core	3	9
Stopped at end of extension 1	1	0

Baseline Characteristics

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Reporting Groups

	Description
β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)
Total	Total of all reporting groups

Baseline Measures

	β-thalassemia Patients	Rare Anemias Patients	Total
Number of Participants [units: participants]	85	99	184
Age [units: years] Mean ± Standard Deviation	24.7 ± 10.03	43.7 ± 26.13	35.0 ± 22.4
Age, Customized [units: participants]
<6 years	2	9	11
Between 6 and 11 years	5	6	11
Between 12 and 15 years	8	5	13
Between 16 and 49 years	69	30	99
Between 50 and 64 years	1	19	20
>=65 years	0	30	30
Gender [units: participants]
Female	43	48	91
Male	42	51	93
Region of Enrollment [units: participants]
United States	15	21	36
France	9	11	20
Canada	12	6	18
Belgium	4	12	16
Germany	2	23	25
United Kingdom	12	0	12
Italy	31	26	57

Outcome Measures

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1. Primary:

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths [ Time Frame: Core study Baseline to the end of the study (up to 60 months) ]

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2. Secondary:

The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study [ Time Frame: Core study Baseline to end of extension study (up to 60 months) ]

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3. Secondary:

The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study [ Time Frame: Core study Baseline to end of extension study (up to 60 months) ]

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4. Secondary:

The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study [ Time Frame: Core study Baseline to end of extension study (up to 60 months) ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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Results Point of Contact:

Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300

No publications provided

Responsible Party:	External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00303329 History of Changes
Other Study ID Numbers:	CICL670A0108E1
Study First Received:	October 14, 2005
Results First Received:	December 15, 2010
Last Updated:	April 15, 2011
Health Authority:	United States: Food and Drug Administration Belgium: Institutional Review Board Canada: Health Canada France: Ministry of Health Germany: Ethics Commission Italy: Ethics Committee United Kingdom: Medicines and Healthcare Products Regulatory Agency

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