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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
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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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
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) ]

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) ]

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) ]

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) ]


  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
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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:
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: 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