Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
1 other identifier
interventional
184
7 countries
28
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Mar 2004
Typical duration for phase_2
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 1, 2004
CompletedFirst Submitted
Initial submission to the registry
October 14, 2005
CompletedFirst Posted
Study publicly available on registry
March 16, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2008
CompletedResults Posted
Study results publicly available
May 9, 2011
CompletedMay 9, 2011
April 1, 2011
4.6 years
October 14, 2005
December 15, 2010
April 15, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
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.
Core study Baseline to the end of the study (up to 60 months)
Secondary Outcomes (3)
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
Core study Baseline to end of extension study (up to 60 months)
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Core study Baseline to end of extension study (up to 60 months)
Study Arms (1)
Deferasirox
EXPERIMENTALDeferasirox daily oral dose between 5-40 mg/kg/day
Interventions
Deferasirox available as 125 mg, 250 mg or 500 mg tablets
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Pregnant or breast feeding patients
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
Children's Hospital and Research center at Oakland
Oakland, California, 94609-1809, United States
Stanford Hospital
Stanford, California, 94305, United States
Childres's Hospital Boston
Boston, Massachusetts, 02115, United States
New York Presbyterian Hospital
New York, New York, 10021, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399, United States
Novartis Investigative Site
Bruges, Belgium
Novartis Investigative Site
Brussels, Belgium
Novartis Investigative Site
Ghent, Belgium
Novartis Investigative Site
La Louvière, Belgium
Novartis Investigative Site
Leuven, Belgium
Novartis Investigative Site
Montreal, Canada
Novartis Investigative Site
Toronto, Canada
Novartis Investigative Site
Créteil, France
Novartis Investigative Site
Le Kremlin-Bicêtre, France
Novartis Investigative Site
Lille, France
Novartis Investigative Site
Troyes, France
Novartis Investigative Site
Düsseldorf, Germany
Novartis Investigative Site
Hanover, Germany
Novartis Investigative Site
Ulm, Germany
Novartis Investigative Site
Bologna, Italy
Novartis Investigative Site
Brindisi, Italy
Novartis Investigative Site
Cagliari, Italy
Novartis Investigative Site
Genova, Italy
Novartis Investigative Site
Milan, Italy
Novartis Investigative Site
Pavia, Italy
Novartis Investigative Site
Rome, Italy
Novartis Investigative Site
Torino, Italy
Novartis Investigative Site
London, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
October 14, 2005
First Posted
March 16, 2006
Study Start
March 1, 2004
Primary Completion
October 1, 2008
Study Completion
October 1, 2008
Last Updated
May 9, 2011
Results First Posted
May 9, 2011
Record last verified: 2011-04