Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

This study has been completed.

Study NCT00235391 Information provided by Novartis

First Received on October 6, 2005. Last Updated on June 3, 2011 History of Changes

Results First Received: December 17, 2010

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Thalassemia Sickle Cell Disease Diamond Blackfan Anemia Myelofibrosis
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
2 to < 6 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
6 to < 12 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
12 to < 16 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
16 to < 50 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
50 to < 65 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
≥ 65 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.

Participant Flow: Overall Study

	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years
STARTED	97	200	172	1164	43	7
Deferasirox Treatment	97	200	172	1164	43	7
COMPLETED	85	182	148	944	31	3
NOT COMPLETED	12	18	24	220	12	4
Adverse Event	4	5	10	72	5	2
Abnormal laboratory value(s)	3	5	4	27	1	2
Unsatisfactory therapeutic effect	0	2	2	17	0	0
Condition no longer requires treatment	3	0	0	11	1	0
Protocol deviation	0	0	1	11	0	0
Withdrawal by Subject	2	3	3	61	5	0
Lost to Follow-up	0	3	3	16	0	0
Administrative problems	0	0	0	1	0	0
Death	0	0	1	4	0	0

Baseline Characteristics

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Reporting Groups

	Description
2 to < 6 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
6 to < 12 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
12 to < 16 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
16 to < 50 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
50 to < 65 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
≥ 65 Years	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.

Baseline Measures

	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years	Total
Number of Participants [units: participants]	97	200	172	1164	43	7	1683
Age [units: years] Mean ± Standard Deviation	3.48 ± 1.13	8.52 ± 1.64	13.48 ± 1.20	28.91 ± 8.06	54.58 ± 3.92	70.14 ± 3.98	24.27 ± 12.63
Gender [units: participants]
Female	44	101	81	633	32	5	896
Male	53	99	91	531	11	2	787
Baseline Disease Characteristics ^[1] [units: participants]
Beta-Thalassemia Major	74	118	114	905	10	0	1221
Beta-Thalassemia Intermedia	1	22	16	94	20	3	156
Sickle Cell Disease	8	41	31	93	2	1	176
Diamond-Blackfan Anemia	4	7	8	24	0	0	43
Other Diseases	10	12	3	48	11	3	87
Prior Chelation Drug Therapy ^[2] [units: participants]
Deferoxamine	80	167	137	745	31	5	1165
Deferiprone	1	8	11	146	6	1	173
Deferoxamine and Deferiprone	2	16	21	269	6	0	314
Other Chelation Drug	6	6	3	4	0	0	19
Prior Chelatation Drug Information Missing	8	3	0	0	0	1	12
Reason for inadequate prior chelation therapy ^[3] [units: participants]
Therapy non-compliance	57	125	106	659	25	2	974
Therapy contraindication	1	7	6	29	1	1	45
Therapy unacceptable toxicity	11	29	22	131	6	1	200
Therapy poor response	20	34	38	267	8	0	367
Therapy unacceptable discomfort	2	2	0	78	3	2	87
Reason for inadequate therapy information missing	6	3	0	0	0	1	10

[1]	The number of participants with each disease category.
[2]	The number of participants for each chelation drug category.
[3]	The number of participants for each reason for inadequate prior chelation therapy category.

Outcome Measures

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1. Primary:

Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) ]

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2. Secondary:

The Change in Serum Ferritin Values From Baseline Through Completion of the Study [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 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
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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:	NCT00235391 History of Changes
Other Study ID Numbers:	CICL670A2203
Study First Received:	October 6, 2005
Results First Received:	December 17, 2010
Last Updated:	June 3, 2011
Health Authority:	United States: Food and Drug Administration