Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study (THALASSA)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00873041
First received: March 30, 2009
Last updated: May 20, 2013
Last verified: May 2013
Results First Received: June 20, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Non-transfusion Dependent Thalassemia
Interventions: Drug: deferasirox
Drug: placebo

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
There was a 4 week screening period to determine eligibility prior to randomization.

Reporting Groups
  Description
5 mg/kg/Day Deferasirox Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
10 mg/kg/Day Deferasirox Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo/Deferasirox Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.

Participant Flow for 2 periods

Period 1:   Core Study
    5 mg/kg/Day Deferasirox     10 mg/kg/Day Deferasirox     Placebo/Deferasirox  
STARTED     55     55     56  
COMPLETED     48     49     51  
NOT COMPLETED     7     6     5  
Adverse Event                 2                 3                 1  
Withdrawal by Subject                 1                 2                 2  
Lost to Follow-up                 3                 1                 0  
Abnormal laboratory value                 0                 0                 1  
Protocol deviation                 1                 0                 1  

Period 2:   Extension Study
    5 mg/kg/Day Deferasirox     10 mg/kg/Day Deferasirox     Placebo/Deferasirox  
STARTED     41     44     48  
COMPLETED     40     44     46  
NOT COMPLETED     1     0     2  
Adverse Event                 1                 0                 1  
Administrative reasons                 0                 0                 1  



  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
5 mg/kg/Day Deferasirox Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
10 mg/kg/Day Deferasirox Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Total Total of all reporting groups

Baseline Measures
    5 mg/kg/Day Deferasirox     10 mg/kg/Day Deferasirox     Placebo     Total  
Number of Participants  
[units: participants]
  55     55     56     166  
Age, Customized  
[units: Participants]
       
<18 years     6     7     8     21  
Between 18 and 65 years     49     47     48     144  
>=65 years     0     1     0     1  
Gender  
[units: participants]
       
Female     26     26     25     77  
Male     29     29     31     89  



  Outcome Measures
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1.  Primary:   Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52   [ Time Frame: Baseline, Week 52 ]

2.  Primary:   Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study   [ Time Frame: Core Baseline to End of Extension Study (up to 24 months) ]

3.  Secondary:   Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24   [ Time Frame: Baseline, Week 24 ]

4.  Secondary:   Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter   [ Time Frame: Baseline, (Day 286 to End of Study [Day 365]) ]

5.  Secondary:   Core Study: Change in Serum Ferritin Between Baseline and Second Quarter   [ Time Frame: Baseline, (Day 106 to Day 195) ]

6.  Secondary:   Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe   [ Time Frame: 52 Weeks ]

7.  Secondary:   Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24   [ Time Frame: Baseline, Week 24, Week 52 ]

8.  Secondary:   Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)   [ Time Frame: Baseline, 52 weeks ]

9.  Secondary:   Core Study: Change From Baseline in Hemoglobin at Month 12   [ Time Frame: Baseline, Month 12 ]

10.  Secondary:   Core Study: Change From Baseline in Transferrin Saturation at Month 12   [ Time Frame: Baseline, Month 12 ]

11.  Secondary:   Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52   [ Time Frame: Baseline, Week 52 ]

12.  Secondary:   Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results   [ Time Frame: 52 Weeks ]

13.  Secondary:   Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure   [ Time Frame: Baseline, 52 Weeks ]

14.  Secondary:   Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure   [ Time Frame: Baseline, 52 Weeks ]

15.  Secondary:   Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate   [ Time Frame: Baseline, 52 Weeks ]

16.  Secondary:   Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter   [ Time Frame: Core Baseline, Eighth Quarter (last 3 months of the study) ]

17.  Secondary:   Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24   [ Time Frame: Core Baseline, Month 24 ]

18.  Secondary:   Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)   [ Time Frame: Core Baseline, Month 24 ]

19.  Secondary:   Extension Study: Change From Baseline in Hemoglobin at Month 24   [ Time Frame: Core Baseline, Month 24 ]

20.  Secondary:   Extension Study: Change From Baseline in Transferrin Saturation at Month 24   [ Time Frame: Core Baseline, Month 24 ]


  Serious Adverse Events


  Other Adverse Events


  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.


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
No text entered.  


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300


No publications provided by Novartis

Publications automatically indexed to this study:

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00873041     History of Changes
Obsolete Identifiers: NCT01185106
Other Study ID Numbers: CICL670A2209, EudraCT 2007-007000-15
Study First Received: March 30, 2009
Results First Received: June 20, 2012
Last Updated: May 20, 2013
Health Authority: United States: Food and Drug Administration
European Union: European Medicine Agency
Greece: Ministry of Health and Welfare
Italy: The Italian Medicines Agency
Lebanon: Institutional Review Board
Taiwan: Health Authority
Malaysia: Ministry of Health
Thailand: Ministry of Public Health
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency