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

October 14, 2005
April 15, 2011
March 2004
October 2008   (final data collection date for primary outcome measure)
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) ] [ Designated as safety issue: Yes ]
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
Not Provided
Complete list of historical versions of study NCT00303329 on ClinicalTrials.gov Archive Site
  • 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) ] [ Designated as safety issue: No ]
    Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.
  • 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) ] [ Designated as safety issue: No ]
    Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.
  • 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) ] [ Designated as safety issue: No ]
    Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.
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Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload

A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.

The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Anemia
  • Hemosiderosis
Drug: Deferasirox
Deferasirox available as 125 mg, 250 mg or 500 mg tablets
Other Name: ICL670
Experimental: Deferasirox
Deferasirox daily oral dose between 5-40 mg/kg/day
Intervention: Drug: Deferasirox
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
184
October 2008
October 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patients completed the planned 12-month core study
  • Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
  • Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation

Exclusion Criteria:

  • Pregnant or breast feeding patients

Other protocol-defined inclusion/exclusion criteria may apply

Both
2 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Belgium,   United Kingdom,   Canada,   France,   Germany,   Italy
 
NCT00303329
CICL670A0108E1
Yes
External Affairs, Novartis Pharmaceuticals
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
April 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP