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

Purpose

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.

Condition	Intervention	Phase
Anemia Hemosiderosis	Drug: Deferasirox	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload

Resource links provided by NLM:

MedlinePlus related topics: Anemia Iron Thalassemia

Drug Information available for: Deferasirox

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

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.

Secondary Outcome Measures:

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.

Enrollment:	184
Study Start Date:	March 2004
Study Completion Date:	October 2008
Primary Completion Date:	October 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Deferasirox Deferasirox daily oral dose between 5-40 mg/kg/day	Drug: Deferasirox Deferasirox available as 125 mg, 250 mg or 500 mg tablets Other Name: ICL670

Eligibility

Ages Eligible for Study:	2 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00303329

Locations

United States, California
Children's Hospital and Research center at Oakland
Oakland, California, United States, 94609-1809
Stanford Hospital
Stanford, California, United States, 94305
United States, Massachusetts
Childres's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, New York
New York Presbyterian Hospital
New York, New York, United States, 10021
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-4399
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, Belgium
Canada
Novartis Investigative Site
Montreal, Canada
Novartis Investigative Site
Toronto, Canada
France
Novartis Investigative Site
Creteil, France
Novartis Investigative Site
Le Kremlin Bicetre, France
Novartis Investigative Site
Lille, France
Novartis Investigative Site
Troyes, France
Germany
Novartis Investigative Site
Duesseldorf, Germany
Novartis Investigative Site
Hannover, Germany
Novartis Investigative Site
Ulm, Germany
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, Italy
United Kingdom
Novartis Investigative Site
London, United Kingdom

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

No publications provided

Responsible Party:	External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00303329 History of Changes
Other Study ID Numbers:	CICL670A0108E1
Study First Received:	October 14, 2005
Results First Received:	December 15, 2010
Last Updated:	April 15, 2011
Health Authority:	United States: Food and Drug Administration Belgium: Institutional Review Board Canada: Health Canada France: Ministry of Health Germany: Ethics Commission Italy: Ethics Committee United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:

β-thalassemia
rare chronic anemia
iron overload

deferasirox
chronic anemias
transfusional hemosiderosis

Additional relevant MeSH terms:

Anemia
Hemosiderosis
Thalassemia
Iron Overload
Hematologic Diseases
Iron Metabolism Disorders
Metabolic Diseases
Anemia, Hemolytic, Congenital

Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Deferasirox
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013