Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) - Study Results

Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) (ESCALATOR)

This study has been completed.

Study NCT00171301 Information provided by Novartis

First Received on September 12, 2005. Last Updated on August 28, 2011 History of Changes

Results First Received: January 12, 2011

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:	Beta-thalassemia Major Hemosiderosis Iron Overload Rare Anemia
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
This is an Extension to Core Study CICL670A2402 (NCT00171171). 233 participants completed the core study and entered this extension study.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
2 participants from the 16 and older group did not receive deferasirox. Thus, the 16 and older group comprises of 69 treated participants.

Reporting Groups

	Description
Deferasirox (Between 2 <16 Years )	Participants age 2 years up to 16 years received a daily oral dose of deferasirox. Dose selection was based on the dose last received in the core study. The individual daily doses of deferasirox and the exact amount of tablets (125, 250, or 500 mg) contributing to each dose were calculated by the investigator based on the patient’s body weight.
Deferasirox (16 Years or Older)	Participants age 16 years or older received a daily oral dose of deferasirox. Dose selection was based on the dose last received in the core study. The individual daily doses of deferasirox and the exact amount of tablets (125, 250, or 500 mg) contributing to each dose were calculated by the investigator based on the patient’s body weight.

Participant Flow: Overall Study

	Deferasirox (Between 2 <16 Years )	Deferasirox (16 Years or Older)
STARTED	162	71 ^[1]
Received Deferasirox	162	69
COMPLETED	154	62
NOT COMPLETED	8	9
Death	1	2
Adverse Event	1	2
Lost to Follow-up	6	2
Protocol Violation	0	2
Withdrawal by Subject	0	1

[1]	2 participants in this group never received deferasirox.

Baseline Characteristics

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Reporting Groups

	Description
Deferasirox (Between 2 <16 Years )	Participants age 2 years up to 16 years received a daily oral dose of deferasirox. Dose selection was based on the dose last received in the core study. The individual daily doses of deferasirox and the exact amount of tablets (125, 250, or 500 mg) contributing to each dose were calculated by the investigator based on the patient’s body weight.
Deferasirox (16 Years or Older)	Participants age 16 years or older received a daily oral dose of deferasirox. Dose selection was based on the dose last received in the core study. The individual daily doses of deferasirox and the exact amount of tablets (125, 250, or 500 mg) contributing to each dose were calculated by the investigator based on the patient’s body weight.

Baseline Measures

	Deferasirox (Between 2 <16 Years )	Deferasirox (16 Years or Older)	Total
Number of Participants [units: participants]	162	71	233
Age ^[1] [units: years] Mean ± Standard Deviation	9.5 ± 3.59	21.2 ± 5.82	13.0 ± 6.93
Gender, Customized ^[2] [units: participants]
Female	80	35	115
Male	82	36	118

[1]	Safety Population consisting of all participants who received study drug in the Extension study n= 231.
[2]	Safety Population consisting of all participants who received study drug in the Extension study n=231.

Outcome Measures

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1. Primary:

Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age [ Time Frame: From Core Study Baseline, to Extension End of Study, Up to 3 Years ]

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2. Primary:

Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ]

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3. Secondary:

Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ]

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4. Secondary:

Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC) [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ]

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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
An internal review revealed major Good Clinical Practice violations at 2 sites in Saudi Arabia: 602 for core + extension, 601 for 2-yr extension. Therefore data was excluded (completely for 602 + partly for 601) from analyses.

Results Point of Contact:

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

No publications provided

Responsible Party:	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT00171301 History of Changes
Other Study ID Numbers:	CICL670A2402E1
Study First Received:	September 12, 2005
Results First Received:	January 12, 2011
Last Updated:	August 28, 2011
Health Authority:	Lebanon: Institutional Review Board; Egypt: The Egyptian Society for Medical Ethics; Oman: The Research and Ethics Committee; Saudia Arabia: Clinical Research Committee; Syria: Institutional Review Board