Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Anemia Clinical Trial

Official title:

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00303329
• Other study ID #: CICL670A0108E1
• Status: Completed
• Phase: Phase 2
• First received: October 14, 2005
• Last updated: April 15, 2011
• Start date: March 2004
• Est. completion date: October 2008

Study information

• Verified date: April 2011
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationBelgium: Institutional Review BoardCanada: Health CanadaFrance: Ministry of HealthGermany: Ethics CommissionItaly: Ethics CommitteeUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 184
• Est. completion date: October 2008
• Est. primary completion date: October 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Beta-Thalassemia
• Hemosiderosis
• Thalassemia

Intervention

Drug:
• Deferasirox
Deferasirox available as 125 mg, 250 mg or 500 mg tablets

Locations

Country	Name	City	State
Belgium	Novartis Investigative Site	Brugge
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Gent
Belgium	Novartis Investigative Site	La Louvière
Belgium	Novartis Investigative Site	Leuven
Canada	Novartis Investigative Site	Montreal
Canada	Novartis Investigative Site	Toronto
France	Novartis Investigative Site	Creteil
France	Novartis Investigative Site	Le Kremlin Bicetre
France	Novartis Investigative Site	Lille
France	Novartis Investigative Site	Troyes
Germany	Novartis Investigative Site	Duesseldorf
Germany	Novartis Investigative Site	Hannover
Germany	Novartis Investigative Site	Ulm
Italy	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
United Kingdom	Novartis Investigative Site	London
United States	Childres's Hospital Boston	Boston	Massachusetts
United States	New York Presbyterian Hospital	New York	New York
United States	Children's Hospital and Research center at Oakland	Oakland	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Stanford Hospital	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	Yes
Secondary	The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study	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.	Core study Baseline to end of extension study (up to 60 months)	No
Secondary	The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study	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.	Core study Baseline to end of extension study (up to 60 months)	No
Secondary	The Absolute Change in Serum Ferritin (µg/L) Levels From Baseline to the End of the Study	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.	Core study Baseline to end of extension study (up to 60 months)	No