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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.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00171301
First received: September 12, 2005
Last updated: August 28, 2011
Last verified: August 2011
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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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

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.
Total Total of all reporting groups

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
  Show All Outcome Measures

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 ]

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 ]

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 ]

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 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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.


  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:
unchecked 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.
unchecked 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.


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