Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload (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: August 22, 2012

Last verified: August 2012

History of Changes

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:	Thalassemia Intermedia Syndrome
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
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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 Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
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 Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
5 mg/kg/Day Placebo	Placebo tablet matching 5 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
10 mg/kg/Day Placebo	Placebo tablet matching 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.

Participant Flow: Overall Study

	5 mg/kg/Day Deferasirox	10 mg/kg/Day Deferasirox	5 mg/kg/Day Placebo	10 mg/kg/Day Placebo
STARTED	55	55	28	28
COMPLETED	48	49	25	26
NOT COMPLETED	7	6	3	2
Adverse Event	2	3	0	1
Withdrawal by Subject	1	2	2	0
Lost to Follow-up	3	1	0	0
Abnormal laboratory value	0	0	0	1
Protocol deviation	1	0	1	0

Baseline Characteristics

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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 Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
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 Content (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
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 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Content (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:

Change in Liver Iron Content (LIC) From Baseline to Week 52 [ Time Frame: Baseline, Week 52 ]

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2. Secondary:

Change in Liver Iron Content (LIC) From Baseline to Week 24 [ Time Frame: Baseline, Week 24 ]

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3. Secondary:

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

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4. Secondary:

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

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5. Secondary:

Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe [ Time Frame: 52 Weeks ]

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6. Secondary:

Change in Liver Iron Content (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24 [ Time Frame: Baseline, Week 24, Week 52 ]

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7. Secondary:

Correlation Between Serum Ferritin and LIC (Liver Iron Content) [ Time Frame: Baseline, 52 weeks ]

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8. Secondary:

Change From Baseline in Hemoglobin at Month 12 [ Time Frame: Baseline, Month 12 ]

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9. Secondary:

Change From Baseline in Transferrin Saturation at Month 12 [ Time Frame: Baseline, Month 12 ]

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10. Secondary:

Change in Liver Iron Content (LIC) in Placebo Patients From Baseline to Week 52 [ Time Frame: Baseline, Week 52 ]

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11. Secondary:

Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results [ Time Frame: 52 Weeks ]

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12. Secondary:

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

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13. Secondary:

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

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14. Secondary:

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

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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
No text entered.

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:	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:	August 22, 2012
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

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